menu
    • menu
    [{"orgOrder":0,"company":"Visirna Therapeutics HK Limited","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"HONG KONG","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"VSA001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Visirna Therapeutics HK Limited","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Visirna Therapeutics HK Limited \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Visirna Therapeutics HK Limited \/ Undisclosed"},{"orgOrder":0,"company":"Visirna Therapeutics HK Limited","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"HONG KONG","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"VSA003","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Visirna Therapeutics HK Limited","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Visirna Therapeutics HK Limited \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Visirna Therapeutics HK Limited \/ Undisclosed"},{"orgOrder":0,"company":"Taiho Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"TAS-205","moa":"15-LOX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Taiho Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Taiho Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Taiho Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Taiho Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"TAS-205","moa":"15-LOX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Taiho Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Taiho Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Taiho Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Taiho Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"TAS-205","moa":"15-LOX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Taiho Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Taiho Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Taiho Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Boston Medical Center","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Bortezomib","moa":"26S proteasome","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Boston Medical Center","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boston Medical Center \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Boston Medical Center \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Memorial Sloan Kettering Cancer Center","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Bortezomib","moa":"26S proteasome","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Memorial Sloan Kettering Cancer Center","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Memorial Sloan Kettering Cancer Center \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Memorial Sloan Kettering Cancer Center \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Gabrail Cancer Center Research","sponsor":"Sanofi | National Cancer Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Bortezomib","moa":"26S proteasome","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Gabrail Cancer Center Research","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gabrail Cancer Center Research \/ Sanofi | National Cancer Institute","highestDevelopmentStatusID":"6","companyTruncated":"Gabrail Cancer Center Research \/ Sanofi | National Cancer Institute"},{"orgOrder":0,"company":"Criterium","sponsor":"Amgen Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Carfilzomib","moa":"26S proteasome","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Criterium","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Criterium \/ Amgen Inc","highestDevelopmentStatusID":"6","companyTruncated":"Criterium \/ Amgen Inc"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Ixazomib Citrate","moa":"26S proteasome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Ixazomib Citrate","moa":"26S proteasome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Icahn School of Medicine at Mount Sinai","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Ixazomib Citrate","moa":"26S proteasome","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Icahn School of Medicine at Mount Sinai","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Icahn School of Medicine at Mount Sinai \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Icahn School of Medicine at Mount Sinai \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Leadiant Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2011","type":"Inapplicable","leadProduct":"Elapegademase","moa":"2'-deoxyadenosine | Adenosine||2\\'-deoxyadenosine | Adenosine","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Leadiant Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Leadiant Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Leadiant Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Nitisinone","moa":"4-hydroxyphenylpyruvate dioxygenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Nitisinone","moa":"4-hydroxyphenylpyruvate dioxygenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Nitisinone","moa":"4-hydroxyphenylpyruvate dioxygenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Parexel","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Nitisinone","moa":"4-hydroxyphenylpyruvate dioxygenase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cycle Pharmaceuticals \/ Parexel","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Parexel"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Parexel","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Nitisinone","moa":"4-hydroxyphenylpyruvate dioxygenase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cycle Pharmaceuticals \/ Parexel","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Parexel"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Parexel","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Nitisinone","moa":"4-hydroxyphenylpyruvate dioxygenase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cycle Pharmaceuticals \/ Parexel","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Parexel"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Nitisinone","moa":"4-hydroxyphenylpyruvate dioxygenase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cycle Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Oakrum Pharma, LLC","sponsor":"Eton Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Acquisition","leadProduct":"Nitisinone","moa":"4-hydroxyphenylpyruvate dioxygenase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Oakrum Pharma, LLC","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Oakrum Pharma, LLC \/ Eton Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Oakrum Pharma, LLC \/ Eton Pharmaceuticals"},{"orgOrder":0,"company":"Analog Pharma","sponsor":"Dipharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Nitisinone","moa":"4-hydroxyphenylpyruvate dioxygenase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Analog Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Analog Pharma \/ Dipharma","highestDevelopmentStatusID":"15","companyTruncated":"Analog Pharma \/ Dipharma"},{"orgOrder":0,"company":"Sutphin Drugs","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Nitisinone","moa":"4-hydroxyphenylpyruvate dioxygenase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sutphin Drugs","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sutphin Drugs \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Sutphin Drugs \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Givosiran","moa":"5-aminolevulinate synthase mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Givosiran","moa":"5-aminolevulinate synthase mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Givosiran","moa":"5-aminolevulinate synthase mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Givosiran","moa":"5-aminolevulinate synthase mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2017","type":"Inapplicable","leadProduct":"Givosiran","moa":"5-aminolevulinate synthase mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2016","type":"Inapplicable","leadProduct":"Givosiran","moa":"5-aminolevulinate synthase mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"Givosiran","moa":"5-aminolevulinate synthase mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"Givosiran","moa":"5-aminolevulinate synthase mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Givosiran","moa":"5-aminolevulinate synthase mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Medison Pharma","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Oligonucleotide","year":"2023","type":"Expanded Collaboration","leadProduct":"Givosiran Sodium","moa":"5-aminolevulinate synthase mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Medison Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Medison Pharma \/ Alnylam Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Medison Pharma \/ Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Alcobra","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Metadoxine","moa":"5-HT2B receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alcobra","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Alcobra \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alcobra \/ Undisclosed"},{"orgOrder":0,"company":"Medivation","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Latrepirdine","moa":"5-HT6 inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medivation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Medivation \/ Pfizer Inc","highestDevelopmentStatusID":"10","companyTruncated":"Medivation \/ Pfizer Inc"},{"orgOrder":0,"company":"Unity Health Toronto","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Tobramycin","moa":"70S ribosome","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Unity Health Toronto","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Unity Health Toronto \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Unity Health Toronto \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibiotic","year":"2014","type":"Inapplicable","leadProduct":"Tobramycin","moa":"70S ribosome","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Antibiotic","year":"2010","type":"Inapplicable","leadProduct":"Tobramycin","moa":"70S ribosome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Chiesi Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Undisclosed"},{"orgOrder":0,"company":"Viatris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2018","type":"Inapplicable","leadProduct":"Tobramycin","moa":"70S ribosome","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Viatris","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Viatris \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Viatris \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibiotic","year":"2013","type":"Inapplicable","leadProduct":"Tobramycin","moa":"70S ribosome","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Pari","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Antibiotic","year":"2013","type":"Inapplicable","leadProduct":"Tobramycin","moa":"70S ribosome","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pari","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Pari \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Pari \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibiotic","year":"2013","type":"Inapplicable","leadProduct":"Tobramycin","moa":"70S ribosome","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibiotic","year":"2014","type":"Inapplicable","leadProduct":"Tobramycin","moa":"70S ribosome","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nebulizer","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Antibiotic","year":"2010","type":"Inapplicable","leadProduct":"Tobramycin","moa":"70S ribosome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Chiesi Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Undisclosed"},{"orgOrder":0,"company":"University of Milan","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Antibiotic","year":"2012","type":"Inapplicable","leadProduct":"Tobramycin","moa":"70S ribosome","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"University of Milan","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Milan \/ Chiesi Group","highestDevelopmentStatusID":"11","companyTruncated":"University of Milan \/ Chiesi Group"},{"orgOrder":0,"company":"Erempharma","sponsor":"University of Lyon | Epidemiologie Pharmacologie Investigation Clinique Information medicale Mere Enfant | Clininfo | Hospices Civils de Lyon","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Antibiotic","year":"2011","type":"Inapplicable","leadProduct":"Tobramycin","moa":"70S ribosome","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Erempharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Erempharma \/ University of Lyon | Epidemiologie Pharmacologie Investigation Clinique Information medicale Mere Enfant | Clininfo | Hospices Civils de Lyon","highestDevelopmentStatusID":"7","companyTruncated":"Erempharma \/ University of Lyon | Epidemiologie Pharmacologie Investigation Clinique Information medicale Mere Enfant | Clininfo | Hospices Civils de Lyon"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Sanofi","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Blackstone Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Collaboration","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Blackstone Life Sciences","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Blackstone Life Sciences"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"NICE","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ NICE","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ NICE"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Cystic Fibrosis Foundation | ECFS-Clinical Trial Network","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Ataluren","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Cystic Fibrosis Foundation | ECFS-Clinical Trial Network","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Cystic Fibrosis Foundation | ECFS-Clinical Trial Network"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"ELX-02","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"ELX-02","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"ELX-02","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"ELX-02","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"ELX-02","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"SGS Life Sciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"ELX-02","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eloxx Pharmaceuticals \/ SGS Life Sciences","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ SGS Life Sciences"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"SGS Life Sciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"ELX-02","moa":"80S Ribosome","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eloxx Pharmaceuticals \/ SGS Life Sciences","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ SGS Life Sciences"},{"orgOrder":0,"company":"Adverum Biotechnologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"ADVM-043","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Adverum Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Adverum Biotechnologies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Adverum Biotechnologies \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"ARC-AAT","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"ARC-AAT","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Deep Track Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Private Placement","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Deep Track Capital","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Deep Track Capital"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"VX-634","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"VX-634","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"VX-814","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"VX-814","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Z Factor Limited","sponsor":"Hammersmith Medicines Research | Centessa Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"ZF874","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Z Factor Limited","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Z Factor Limited \/ Hammersmith Medicines Research | Centessa Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Z Factor Limited \/ Hammersmith Medicines Research | Centessa Pharmaceuticals"},{"orgOrder":0,"company":"Z Factor","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"ZF874","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Z Factor","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Z Factor \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Z Factor \/ Undisclosed"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"ZF874","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Centessa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Centessa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Centessa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"AM-Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Ilofotase Alpha","moa":"A2AR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AM-Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AM-Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AM-Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AM-Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Ilofotase Alpha","moa":"A2AR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AM-Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AM-Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AM-Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Adrenas Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"AAV BBP-631","moa":"AAV","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Adrenas Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Adrenas Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Adrenas Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jerry R. Mendell","sponsor":"Duchenne Alliance | Milo Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"rAAV1.CMV.huFollistatin344","moa":"AAV1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jerry R. Mendell","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Jerry R. Mendell \/ Duchenne Alliance | Milo Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Jerry R. Mendell \/ Duchenne Alliance | Milo Therapeutics"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"National Heart, Lung, and Blood Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"rAAV1-CB-hAAT","moa":"AAV1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beacon Therapeutics \/ National Heart, Lung, and Blood Institute","highestDevelopmentStatusID":"8","companyTruncated":"Beacon Therapeutics \/ National Heart, Lung, and Blood Institute"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-103","moa":"AAV5","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-103","moa":"AAV5","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-103","moa":"AAV5","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Digna Biotech","sponsor":"Porphyria Centre Sweden | University of Navarra | Uniqure | Nationales Centrum f\u00fcr Tumorerkrankungen","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"rAAV2\/5-PBGD Vector","moa":"AAV5","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Digna Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Digna Biotech \/ Porphyria Centre Sweden | University of Navarra | Uniqure | Nationales Centrum f\u00fcr Tumorerkrankungen","highestDevelopmentStatusID":"6","companyTruncated":"Digna Biotech \/ Porphyria Centre Sweden | University of Navarra | Uniqure | Nationales Centrum f\u00fcr Tumorerkrankungen"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"AskBio009","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Rebisufligene Etisparvovec","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Rebisufligene Etisparvovec","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Rebisufligene Etisparvovec","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Rebisufligene Etisparvovec","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Rebisufligene Etisparvovec","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"UX111","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Abeona Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Abeona Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"UX111","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Abeona Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Abeona Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Acquisition","leadProduct":"UX111","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Abeona Therapeutics \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Abeona Therapeutics \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Abeona Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2016","type":"Inapplicable","leadProduct":"UX111","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Abeona Therapeutics","highestDevelopmentStatusID":"9","companyTruncated":"Ultragenyx Pharmaceutical \/ Abeona Therapeutics"},{"orgOrder":0,"company":"Lantu Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Vesemnogene Lantuparvovec","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lantu Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Lantu Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lantu Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"AAVB-039","moa":"ABCA4 protein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AAVantgarde Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2012","type":"Inapplicable","leadProduct":"SAR422459","moa":"ABCR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2011","type":"Inapplicable","leadProduct":"SAR422459","moa":"ABCR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Oxford Biomedica","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Licensing Agreement","leadProduct":"SAR422459","moa":"ABCR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Oxford Biomedica","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oxford Biomedica \/ Sanofi","highestDevelopmentStatusID":"8","companyTruncated":"Oxford Biomedica \/ Sanofi"},{"orgOrder":0,"company":"Shenzhen Beike Bio-Technology Co., Ltd.","sponsor":"The Second Affiliated Hospital of Kunming Medical University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2012","type":"Inapplicable","leadProduct":"Human Umbilical Cord Derived Mesenchymal Stem Cell","moa":"ACE2 receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Shenzhen Beike Bio-Technology Co., Ltd.","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Shenzhen Beike Bio-Technology Co., Ltd. \/ The Second Affiliated Hospital of Kunming Medical University","highestDevelopmentStatusID":"7","companyTruncated":"Shenzhen Beike Bio-Technology Co., Ltd. \/ The Second Affiliated Hospital of Kunming Medical University"},{"orgOrder":0,"company":"Eisai","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Donepezil","moa":"Acetylcholinesterase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eisai","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eisai \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eisai \/ Undisclosed"},{"orgOrder":0,"company":"Annovis Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Buntanetap","moa":"ACHE","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Annovis Bio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Annovis Bio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Annovis Bio \/ Undisclosed"},{"orgOrder":0,"company":"Enzyvant","sponsor":"Aceragen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Acquisition","leadProduct":"Recombinant Human Acid Ceramidase","moa":"Acid ceramidase","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Enzyvant","amount2":0.23000000000000001,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Enzyvant \/ Aceragen","highestDevelopmentStatusID":"5","companyTruncated":"Enzyvant \/ Aceragen"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"SPR001","moa":"ACTH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Spruce Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"SPR001","moa":"ACTH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Spruce Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"H. Lundbeck AS","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Lu AG13909","moa":"ACTH receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"H. Lundbeck AS","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"H. Lundbeck AS \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"H. Lundbeck AS \/ Undisclosed"},{"orgOrder":0,"company":"Dana-Farber Cancer Institute","sponsor":"Brigham and Women's Hospital | Boston Children's Hospital | Washington University School of Medicine | Medical College of Wisconsin | Johns Hopkins University | La Jolla Institute for Allergy & Immunology | National Heart, Lung, and Blood Institute | Aste","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Regadenoson Monohydrate","moa":"Adenosine A2a receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Dana-Farber Cancer Institute","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dana-Farber Cancer Institute \/ Brigham and Women's Hospital | Boston Children's Hospital | Washington University School of Medicine | Medical College of Wisconsin | Johns Hopkins University | La Jolla Institute for Allergy & Immunology | National Heart, Lung, and Blood Institute | Aste","highestDevelopmentStatusID":"6","companyTruncated":"Dana-Farber Cancer Institute \/ Brigham and Women's Hospital | Boston Children's Hospital | Washington University School of Medicine | Medical College of Wisconsin | Johns Hopkins University | La Jolla Institute for Allergy & Immunology | National Heart, Lung, and Blood Institute | Aste"},{"orgOrder":0,"company":"Versiti","sponsor":"Medical College of Wisconsin | La Jolla Institute for Allergy & Immunology | University of Illinois, Chicago | Oregon Health and Science University | Dana-Farber Cancer Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Regadenoson Monohydrate","moa":"Adenosine A2a receptor","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Versiti","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Versiti \/ Medical College of Wisconsin | La Jolla Institute for Allergy & Immunology | University of Illinois, Chicago | Oregon Health and Science University | Dana-Farber Cancer Institute","highestDevelopmentStatusID":"1","companyTruncated":"Versiti \/ Medical College of Wisconsin | La Jolla Institute for Allergy & Immunology | University of Illinois, Chicago | Oregon Health and Science University | Dana-Farber Cancer Institute"},{"orgOrder":0,"company":"Palo Biofarma","sponsor":"Hospital de la Santa Creu i Sant Pau","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"PBF-999","moa":"Adenosine A2a receptor | Phosphodiesterase 10A","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Palo Biofarma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Palo Biofarma \/ Hospital de la Santa Creu i Sant Pau","highestDevelopmentStatusID":"6","companyTruncated":"Palo Biofarma \/ Hospital de la Santa Creu i Sant Pau"},{"orgOrder":0,"company":"Hospital de la Santa Creu i Sant Pau","sponsor":"Palo Biofarma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"PBF-999","moa":"Adenosine A2a receptor | Phosphodiesterase 10A","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Hospital de la Santa Creu i Sant Pau","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hospital de la Santa Creu i Sant Pau \/ Palo Biofarma","highestDevelopmentStatusID":"6","companyTruncated":"Hospital de la Santa Creu i Sant Pau \/ Palo Biofarma"},{"orgOrder":0,"company":"University of California, Los Angeles","sponsor":"National Institute of Allergy and Infectious Diseases | National Human Genome Research Institute | National Heart, Lung, and Blood Institute | Orchard Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2013","type":"Inapplicable","leadProduct":"OTL-101","moa":"Adenosine deaminase (ADA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"University of California, Los Angeles","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"University of California, Los Angeles \/ National Institute of Allergy and Infectious Diseases | National Human Genome Research Institute | National Heart, Lung, and Blood Institute | Orchard Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"University of California, Los Angeles \/ National Institute of Allergy and Infectious Diseases | National Human Genome Research Institute | National Heart, Lung, and Blood Institute | Orchard Therapeutics"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Droxidopa","moa":"Adrenergic receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NYU Langone Health \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NYU Langone Health \/ Undisclosed"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Dexmedetomidine Hydrochloride","moa":"Adrenergic receptor alpha-2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Film, Soluble","sponsorNew":"NYU Langone Health \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NYU Langone Health \/ Undisclosed"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Dexmedetomidine Hydrochloride","moa":"Adrenergic receptor alpha-2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Film, Soluble","sponsorNew":"NYU Langone Health \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NYU Langone Health \/ Undisclosed"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Winthrop University Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Guanfacine","moa":"Adrenergic receptor alpha-2","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NYU Langone Health \/ Winthrop University Hospital","highestDevelopmentStatusID":"11","companyTruncated":"NYU Langone Health \/ Winthrop University Hospital"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"Johns Hopkins University | Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2010","type":"Inapplicable","leadProduct":"Sodium Chloride","moa":"Adrenergic receptor Alpha-2 (ADRA2)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"University of North Carolina","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nebulizer","sponsorNew":"University of North Carolina \/ Johns Hopkins University | Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"6","companyTruncated":"University of North Carolina \/ Johns Hopkins University | Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Constant Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Talfirastide","moa":"AGTR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Constant Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Constant Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Constant Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vaderis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"VAD044","moa":"AKT kinase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vaderis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vaderis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vaderis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vaderis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"VAD044","moa":"AKT kinase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vaderis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vaderis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vaderis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vaderis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"VAD044","moa":"AKT kinase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vaderis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vaderis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vaderis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vaderis Therapeutics","sponsor":"Medicxi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"VAD044","moa":"AKT kinase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vaderis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vaderis Therapeutics \/ Medicxi","highestDevelopmentStatusID":"7","companyTruncated":"Vaderis Therapeutics \/ Medicxi"},{"orgOrder":0,"company":"Vascarta","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Plant Extract\/Herbal","year":"2025","type":"Inapplicable","leadProduct":"Curcumin","moa":"Albendazole monooxygenase (CYP3A4)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vascarta","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Vascarta \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vascarta \/ Undisclosed"},{"orgOrder":0,"company":"Vascarta","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Plant Extract\/Herbal","year":"2025","type":"Inapplicable","leadProduct":"Curcumin","moa":"Albendazole monooxygenase (CYP3A4)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vascarta","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vascarta \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vascarta \/ Undisclosed"},{"orgOrder":0,"company":"Vascarta","sponsor":"National Heart, Lung, and Blood Institute","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Plant Extract\/Herbal","year":"2025","type":"Agreement","leadProduct":"Curcumin","moa":"Albendazole monooxygenase (CYP3A4)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vascarta","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Vascarta \/ National Heart, Lung, and Blood Institute","highestDevelopmentStatusID":"6","companyTruncated":"Vascarta \/ National Heart, Lung, and Blood Institute"},{"orgOrder":0,"company":"Nivalis Therapeutics","sponsor":"Medidata Solutions","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Cavosonstat","moa":"Alcohol dehydrogenase class III","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nivalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nivalis Therapeutics \/ Medidata Solutions","highestDevelopmentStatusID":"8","companyTruncated":"Nivalis Therapeutics \/ Medidata Solutions"},{"orgOrder":0,"company":"Nivalis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Cavosonstat","moa":"Alcohol dehydrogenase class III","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nivalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nivalis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nivalis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nivalis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Cavosonstat","moa":"Alcohol dehydrogenase class III","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nivalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nivalis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nivalis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nivalis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"N6022","moa":"Alcohol dehydrogenase class III","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nivalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nivalis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nivalis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nivalis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"N91115","moa":"Alcohol dehydrogenase class III","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nivalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nivalis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nivalis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nivalis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"N91115","moa":"Alcohol dehydrogenase class III","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nivalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nivalis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nivalis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"National Heart, Lung, and Blood Institute","sponsor":"Quercis Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Isoquercitrin","moa":"Aldo-keto reductase family 1 member B1; Angiotensin-converting enzyme","graph1":"Genetic Disease","graph2":"Phase II","graph3":"National Heart, Lung, and Blood Institute","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Heart, Lung, and Blood Institute \/ Quercis Pharma","highestDevelopmentStatusID":"8","companyTruncated":"National Heart, Lung, and Blood Institute \/ Quercis Pharma"},{"orgOrder":0,"company":"Jeffrey Zwicker","sponsor":"Quercegen Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Isoquercitrin","moa":"Aldo-keto reductase family 1 member B1; Angiotensin-converting enzyme","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Jeffrey Zwicker","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jeffrey Zwicker \/ Quercegen Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Jeffrey Zwicker \/ Quercegen Pharmaceuticals"},{"orgOrder":0,"company":"Maggie's Pearl","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Epalrestat","moa":"Aldose reductase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Maggie's Pearl","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Maggie's Pearl \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Maggie's Pearl \/ Undisclosed"},{"orgOrder":0,"company":"Diagonal Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"DIAG723","moa":"ALK-1 receptor","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Diagonal Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Diagonal Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Diagonal Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"ALXN1850","moa":"Alkaline phosphatase tissue-nonspecific (ALPL)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"ALXN1850","moa":"Alkaline phosphatase tissue-nonspecific (ALPL)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"ALXN1850","moa":"Alkaline phosphatase tissue-nonspecific (ALPL)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"ALXN1850","moa":"Alkaline phosphatase tissue-nonspecific (ALPL)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Sanford Burnham Prebys","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Amino Acid","year":"2022","type":"Inapplicable","leadProduct":"N-Acetyl-D-Leucine","moa":"Alkaline phosphatase, tissue-nonspecific isozyme","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Daiichi Sankyo \/ Sanford Burnham Prebys","highestDevelopmentStatusID":"8","companyTruncated":"Daiichi Sankyo \/ Sanford Burnham Prebys"},{"orgOrder":0,"company":"Grifols International","sponsor":"Grifols International","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"Alpha-1 Proteinase Inhibitor","moa":"Alpha-1 antitrypsin||Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Grifols International \/ Grifols International","highestDevelopmentStatusID":"10","companyTruncated":"Grifols International \/ Grifols International"},{"orgOrder":0,"company":"Grifols International","sponsor":"Grifols International","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"Alpha-1 Proteinase Inhibitor","moa":"Alpha-1 antitrypsin||Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Grifols International \/ Grifols International","highestDevelopmentStatusID":"10","companyTruncated":"Grifols International \/ Grifols International"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Alpha-1 Proteinase Inhibitor","moa":"Alpha-1 antitrypsin||Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Alpha-1 Proteinase Inhibitor","moa":"Alpha-1 antitrypsin||Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"Alpha-1 Proteinase Inhibitor","moa":"Alpha-1 antitrypsin||Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Alpha-1 Proteinase Inhibitor","moa":"Alpha-1 antitrypsin||Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"Alpha-1 Proteinase Inhibitor","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Alpha-1 Proteinase Inhibitor","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"DCR-A1AT","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dicerna Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Dicerna Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dicerna Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"INBRX-101","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inhibrx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Inhibrx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inhibrx \/ Undisclosed"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"INBRX-101","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inhibrx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Inhibrx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inhibrx \/ Undisclosed"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"INBRX-101","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inhibrx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Inhibrx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inhibrx \/ Undisclosed"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"INBRX-101","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inhibrx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Inhibrx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inhibrx \/ Undisclosed"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"INBRX-101","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inhibrx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Inhibrx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inhibrx \/ Undisclosed"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Acquisition","leadProduct":"INBRX-101","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inhibrx","amount2":2.2000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":2.2000000000000002,"dosageForm":"Infusion","sponsorNew":"Inhibrx \/ Sanofi","highestDevelopmentStatusID":"8","companyTruncated":"Inhibrx \/ Sanofi"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Acquisition","leadProduct":"INBRX-101","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inhibrx","amount2":2.2000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":2.2000000000000002,"dosageForm":"Infusion","sponsorNew":"Inhibrx \/ Sanofi","highestDevelopmentStatusID":"8","companyTruncated":"Inhibrx \/ Sanofi"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"INBRX-101","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inhibrx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Inhibrx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inhibrx \/ Undisclosed"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"INBRX-101","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inhibrx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Inhibrx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inhibrx \/ Undisclosed"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"INBRX-101","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inhibrx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Inhibrx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inhibrx \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"OctaAlpha1","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"VX-864","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"VX-864","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A||Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Blackstone Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Financing","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Capsule","sponsorNew":"Amicus Therapeutics \/ Blackstone Life Sciences","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Blackstone Life Sciences"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"ARYA Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Acquisition","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0.59999999999999998,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.59999999999999998,"dosageForm":"Capsule","sponsorNew":"Amicus Therapeutics \/ ARYA Sciences","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ ARYA Sciences"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Protalix BioTherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Chiesi Group \/ Protalix BioTherapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Protalix BioTherapeutics"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Protalix BioTherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Chiesi Group \/ Protalix BioTherapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Protalix BioTherapeutics"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Protalix BioTherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chiesi Group \/ Protalix BioTherapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Protalix BioTherapeutics"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Chiesi Group","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Chiesi Group"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Chiesi Group","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Chiesi Group"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Chiesi Group","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Chiesi Group"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Chiesi Group","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Chiesi Group"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Psagot Investment House","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2020","type":"Financing","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Psagot Investment House","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Psagot Investment House"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Chiesi Group","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Chiesi Group"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Chiesi Group","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Chiesi Group"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix BioTherapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Protalix BioTherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Viking Global Investors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Series C Financing","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Viking Global Investors","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Viking Global Investors"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"AVR-RD-01","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AVR-RD-01","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AVR-RD-01","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AVR-RD-01","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AVR-RD-01","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-01","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AVR-RD-01","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-01","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Public Offering","leadProduct":"AVR-RD-01","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Morgan Stanley","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Morgan Stanley"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AVR-RD-01","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"ST-920","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"CalciMedica","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"GB-501","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CalciMedica","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CalciMedica \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"CalciMedica \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"San Raffaele-Telethon Institute for Gene Therapy","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ San Raffaele-Telethon Institute for Gene Therapy","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ San Raffaele-Telethon Institute for Gene Therapy"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Oxford Biomedica","sponsor":"Orchard Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oxford Biomedica","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Oxford Biomedica \/ Orchard Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Oxford Biomedica \/ Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Generium","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Antibody-protein Conjugate","year":"2024","type":"Inapplicable","leadProduct":"Verenafusp Alfa","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Generium \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Generium \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"RGX-111","moa":"Alpha-L-iduronidase (IDUA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"RGX-111","moa":"Alpha-L-iduronidase (IDUA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"RGX-111","moa":"Alpha-L-iduronidase (IDUA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"RGX-111","moa":"Alpha-L-iduronidase (IDUA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2016","type":"Inapplicable","leadProduct":"SB-318","moa":"Alpha-L-iduronidase (IDUA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AM-Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Ilofotase Alfa","moa":"ALPI","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AM-Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AM-Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AM-Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"GS-5737","moa":"Amiloride-sensitive sodium channel, ENaC","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gilead Sciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Gilead Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"GS-5737","moa":"Amiloride-sensitive sodium channel, ENaC","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gilead Sciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Gilead Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Parion Sciences","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Idrevloride","moa":"Amiloride-sensitive sodium channel, ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Parion Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Parion Sciences \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Parion Sciences \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Parion Sciences","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"P-1037","moa":"Amiloride-sensitive sodium channel, ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Parion Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Parion Sciences \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Parion Sciences \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"Cofirasersen","moa":"Amiloride-sensitive sodium channel, ENaC mRNA","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nebulizer","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Synlogic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"SYNB1020","moa":"Ammonia","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Synlogic \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Synlogic \/ Undisclosed"},{"orgOrder":0,"company":"Versantis","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"VS-01","moa":"Ammonia clearance","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Versantis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Versantis \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Versantis \/ Undisclosed"},{"orgOrder":0,"company":"NephroDI Therapeutics","sponsor":"Xontogeny, LLC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Financing","leadProduct":"NDI-5033","moa":"AMPK","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NephroDI Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NephroDI Therapeutics \/ Xontogeny, LLC","highestDevelopmentStatusID":"4","companyTruncated":"NephroDI Therapeutics \/ Xontogeny, LLC"},{"orgOrder":0,"company":"Poxel","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"PXL770","moa":"AMPK","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Poxel","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Poxel \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Poxel \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"Chimeric Fibril-Reactive Monoclonal Antibody 11-1F4","moa":"Amyloid","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Life Molecular Imaging","sponsor":"pharmtrace","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Florbetaben","moa":"Amyloid","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Life Molecular Imaging","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Life Molecular Imaging \/ pharmtrace","highestDevelopmentStatusID":"6","companyTruncated":"Life Molecular Imaging \/ pharmtrace"},{"orgOrder":0,"company":"AC Immune","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Vaccine","year":"2020","type":"Inapplicable","leadProduct":"ACI-24","moa":"Amyloid beta A4 protein (APP)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AC Immune","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AC Immune \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AC Immune \/ Undisclosed"},{"orgOrder":0,"company":"AC Immune","sponsor":"National Institute on Aging | Alzheimer's Disease Cooperative Study | LuMind IDSC Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Vaccine","year":"2016","type":"Inapplicable","leadProduct":"ACI-24","moa":"Amyloid beta A4 protein (APP)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AC Immune","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AC Immune \/ National Institute on Aging | Alzheimer's Disease Cooperative Study | LuMind IDSC Foundation","highestDevelopmentStatusID":"8","companyTruncated":"AC Immune \/ National Institute on Aging | Alzheimer's Disease Cooperative Study | LuMind IDSC Foundation"},{"orgOrder":0,"company":"C.T. Development America","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Eprodisate Disodium","moa":"Amyloid fibril formation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"C.T. Development America","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"C.T. Development America \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"C.T. Development America \/ Undisclosed"},{"orgOrder":0,"company":"Proclara Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"NPT189","moa":"Amyloid-beta\/Tau","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Proclara Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proclara Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Proclara Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Proclara Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"NPT189","moa":"Amyloid-beta\/Tau","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Proclara Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proclara Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Proclara Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Life Molecular Imaging","sponsor":"pharmtrace klinische Entwicklung","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Radiolabeled Compound","year":"2022","type":"Inapplicable","leadProduct":"18-F Florbetaben","moa":"Amyloid-beta-plaque","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Life Molecular Imaging","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Life Molecular Imaging \/ pharmtrace klinische Entwicklung","highestDevelopmentStatusID":"10","companyTruncated":"Life Molecular Imaging \/ pharmtrace klinische Entwicklung"},{"orgOrder":0,"company":"Cleveland Clinic","sponsor":"Eli Lilly","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Radiolabeled Compound","year":"2017","type":"Inapplicable","leadProduct":"18-F Florbetapir","moa":"Amyloid-beta-plaque","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Cleveland Clinic","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Cleveland Clinic \/ Eli Lilly","highestDevelopmentStatusID":"11","companyTruncated":"Cleveland Clinic \/ Eli Lilly"},{"orgOrder":0,"company":"AccurEdit Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ART001","moa":"Amyloidogenic TTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AccurEdit Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AccurEdit Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AccurEdit Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AccurEdit Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ART001","moa":"Amyloidogenic TTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AccurEdit Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AccurEdit Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AccurEdit Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AccurEdit Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ART001","moa":"Amyloidogenic TTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AccurEdit Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AccurEdit Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AccurEdit Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Akcea Therapeutics","sponsor":"Ionis Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2017","type":"Inapplicable","leadProduct":"AKCEA-ANGPTL3-LRx","moa":"Angiopoietin like 3 (ANGPTL3) mRNA | Asialoglycoprotein receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Akcea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Akcea Therapeutics \/ Ionis Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Akcea Therapeutics \/ Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Akcea Therapeutics","sponsor":"Ionis Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"AKCEA-ANGPTL3-LRx","moa":"Angiopoietin like 3 (ANGPTL3) mRNA | Asialoglycoprotein receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Akcea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Akcea Therapeutics \/ Ionis Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Akcea Therapeutics \/ Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Evinacumab","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Collaboration","leadProduct":"Evinacumab","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Regeneron Pharmaceuticals","amount2":0.089999999999999997,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Infusion","sponsorNew":"Regeneron Pharmaceuticals \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Regeneron Pharmaceuticals \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Evinacumab","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Evinacumab","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Evinacumab","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Evinacumab","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Evinacumab","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Evinacumab","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Evinacumab","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2014","type":"Inapplicable","leadProduct":"REGN1500","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2014","type":"Inapplicable","leadProduct":"REGN1500","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"ARO-ANG","moa":"ANGPTL3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Beijing Suncadia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"SHR-1918","moa":"ANGPTL3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Beijing Suncadia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beijing Suncadia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Beijing Suncadia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Beijing Suncadia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"SHR-1918","moa":"ANGPTL3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Beijing Suncadia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beijing Suncadia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Beijing Suncadia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Beijing Suncadia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"SHR-1918","moa":"ANGPTL3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Beijing Suncadia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beijing Suncadia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Beijing Suncadia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Zodasiran","moa":"ANGPTL3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"ETD002","moa":"Anoctamin-1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Enterprise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Enterprise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Hansa Biopharma","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Enzyme","year":"2020","type":"Licensing Agreement","leadProduct":"Imlifidase","moa":"Antibodies","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Hansa Biopharma","amount2":0.40999999999999998,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.40999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Hansa Biopharma \/ Sarepta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Hansa Biopharma \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"Baliforsen","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Donidalorsen","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Donidalorsen","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Donidalorsen","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Donidalorsen","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Donidalorsen","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Donidalorsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Donidalorsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Donidalorsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Donidalorsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Donidalorsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Donidalorsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Donidalorsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Otsuka Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Licensing Agreement","leadProduct":"Donidalorsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Otsuka Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Otsuka Pharmaceutical"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Otsuka Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Licensing Agreement","leadProduct":"Donidalorsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Otsuka Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Otsuka Pharmaceutical"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Donidalorsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Donidalorsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Ionis Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Eplontersen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AstraZeneca \/ Ionis Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"AstraZeneca \/ Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Eplontersen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Olezarsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Licensing Agreement","leadProduct":"Olezarsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"European Commission","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"QR-010","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"ProQR Therapeutics \/ European Commission","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ European Commission"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"European Commission","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"QR-010","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"ProQR Therapeutics \/ European Commission","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ European Commission"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2017","type":"Inapplicable","leadProduct":"WVE-120101","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"WVE-120101","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2017","type":"Inapplicable","leadProduct":"WVE-120102","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"WVE-120102","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"WVE-120102","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"ALN-AT3SC","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Duke University","sponsor":"Eisai Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Tedelparin","moa":"Antithrombin-III","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Duke University","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Duke University \/ Eisai Inc","highestDevelopmentStatusID":"8","companyTruncated":"Duke University \/ Eisai Inc"},{"orgOrder":0,"company":"Cumberland Pharmaceuticals","sponsor":"Vanderbilt University Medical Center","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Ifetroban Sodium","moa":"antithrombotics (thromboxane A2 receptor antagonists)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cumberland Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cumberland Pharmaceuticals \/ Vanderbilt University Medical Center","highestDevelopmentStatusID":"8","companyTruncated":"Cumberland Pharmaceuticals \/ Vanderbilt University Medical Center"},{"orgOrder":0,"company":"BlackfinBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"BFB-101","moa":"AP4B1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BlackfinBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BlackfinBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BlackfinBio \/ Undisclosed"},{"orgOrder":0,"company":"BlackfinBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"BFB-101","moa":"AP4B1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BlackfinBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BlackfinBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BlackfinBio \/ Undisclosed"},{"orgOrder":0,"company":"Mammoth Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"MB-111","moa":"APOC-3","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Mammoth Biosciences","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mammoth Biosciences \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Mammoth Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Visirna","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Oligonucleotide","year":"2025","type":"Licensing Agreement","leadProduct":"Plozasiran","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Visirna","amount2":0.40000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.40000000000000002,"dosageForm":"Injection","sponsorNew":"Visirna \/ Sanofi","highestDevelopmentStatusID":"10","companyTruncated":"Visirna \/ Sanofi"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Plozasiran","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Plozasiran","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Plozasiran","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Plozasiran","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Plozasiran","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Plozasiran","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Plozasiran","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Plozasiran","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Plozasiran","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Sixth Street","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Financing","leadProduct":"Plozasiran","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Sixth Street","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Sixth Street"},{"orgOrder":0,"company":"Cerenis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"CER-001","moa":"Apolipoprotein A-I (APOA1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cerenis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cerenis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cerenis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Olezarsen","moa":"Apolipoprotein C-III mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Olezarsen","moa":"Apolipoprotein C-III mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Olezarsen","moa":"Apolipoprotein C-III mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Olezarsen","moa":"Apolipoprotein C-III mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Olezarsen","moa":"Apolipoprotein C-III mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Olezarsen","moa":"Apolipoprotein C-III mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Theratechnologies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Licensing Agreement","leadProduct":"Olezarsen","moa":"Apolipoprotein C-III mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Theratechnologies","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Theratechnologies"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Olezarsen","moa":"Apolipoprotein C-III mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Olezarsen","moa":"Apolipoprotein C-III mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Akcea Therapeutics","sponsor":"Ionis Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2016","type":"Inapplicable","leadProduct":"Volanesorsen","moa":"Apolipoprotein C-III mRNA 3'UTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Akcea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Akcea Therapeutics \/ Ionis Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Akcea Therapeutics \/ Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Akcea Therapeutics","sponsor":"CaligorRx, Inc.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"Volanesorsen","moa":"Apolipoprotein C-III mRNA 3'UTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Akcea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Akcea Therapeutics \/ CaligorRx, Inc.","highestDevelopmentStatusID":"10","companyTruncated":"Akcea Therapeutics \/ CaligorRx, Inc."},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Akcea Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"Volanesorsen","moa":"Apolipoprotein C-III mRNA 3'UTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ Akcea Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Akcea Therapeutics"},{"orgOrder":0,"company":"Calithera Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"CB-280","moa":"Arginase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Calithera Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Calithera Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Calithera Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Calithera Biosciences","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Funding","leadProduct":"CB-280","moa":"Arginase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Calithera Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Calithera Biosciences \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"6","companyTruncated":"Calithera Biosciences \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Calithera Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"CB-280","moa":"Arginase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Calithera Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Calithera Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Calithera Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Calithera Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"CB-280","moa":"Arginase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Calithera Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Calithera Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Calithera Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Calithera Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"CB-280","moa":"Arginase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Calithera Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Calithera Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Calithera Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Calithera Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"CB-280","moa":"Arginase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Calithera Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Calithera Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Calithera Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"National Taiwan University Hospital","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"TAIWAN","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"AAV2-hAADC","moa":"Aromatic-L-amino-acid decarboxylase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"National Taiwan University Hospital","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"National Taiwan University Hospital \/ PTC Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"National Taiwan University Hospital \/ PTC Therapeutics"},{"orgOrder":0,"company":"National Taiwan University Hospital","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"TAIWAN","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"AAV2-hAADC","moa":"Aromatic-L-amino-acid decarboxylase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"National Taiwan University Hospital","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"National Taiwan University Hospital \/ PTC Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"National Taiwan University Hospital \/ PTC Therapeutics"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Mannitol","moa":"Aspergillus Mannitol 2-dehydrogenase (Asperg M2DH)","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"University of North Carolina","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of North Carolina \/ Chiesi Group","highestDevelopmentStatusID":"11","companyTruncated":"University of North Carolina \/ Chiesi Group"},{"orgOrder":0,"company":"Pharmaxis","sponsor":"Aptar Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Miscellaneous","year":"2021","type":"Agreement","leadProduct":"Mannitol","moa":"Aspergillus Mannitol 2-dehydrogenase (Asperg M2DH)","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pharmaxis","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"Pharmaxis \/ Aptar Pharma","highestDevelopmentStatusID":"15","companyTruncated":"Pharmaxis \/ Aptar Pharma"},{"orgOrder":0,"company":"Syntara Limited","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Mannitol","moa":"Aspergillus Mannitol 2-dehydrogenase (Asperg M2DH)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Syntara Limited","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Syntara Limited \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Syntara Limited \/ Undisclosed"},{"orgOrder":0,"company":"Syntara Limited","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Mannitol","moa":"Aspergillus Mannitol 2-dehydrogenase (Asperg M2DH)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Syntara Limited","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Syntara Limited \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Syntara Limited \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"ARO-ATXN2","moa":"Ataxin-2","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Baylor College of Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2021","type":"Inapplicable","leadProduct":"Citrulline","moa":"Atherogenic","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Baylor College of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baylor College of Medicine \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Baylor College of Medicine \/ Undisclosed"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"Hawaii Pacific Neuroscience","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"nL-ATN1-002","moa":"ATN1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ Hawaii Pacific Neuroscience","highestDevelopmentStatusID":"7","companyTruncated":"n-Lorem Foundation \/ Hawaii Pacific Neuroscience"},{"orgOrder":0,"company":"Esperion Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Bempedoic Acid","moa":"||ATP-citrate synthase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Esperion Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Esperion Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Esperion Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2012","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Divestment","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0.11,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2014","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Icon Plc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Icon Plc","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Icon Plc"},{"orgOrder":0,"company":"Neurotech Pharmaceuticals","sponsor":"University of California, San Francisco","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2012","type":"Inapplicable","leadProduct":"NT-501","moa":"atropine derivatives","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Neurotech Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Neurotech Pharmaceuticals \/ University of California, San Francisco","highestDevelopmentStatusID":"8","companyTruncated":"Neurotech Pharmaceuticals \/ University of California, San Francisco"},{"orgOrder":0,"company":"Prothena","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"PRX004","moa":"ATTR protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Prothena","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prothena \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Prothena \/ Undisclosed"},{"orgOrder":0,"company":"Prothena","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2021","type":"Acquisition","leadProduct":"PRX004","moa":"ATTR protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Prothena","amount2":1.2,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":1.2,"dosageForm":"Infusion","sponsorNew":"Prothena \/ Novo Nordisk","highestDevelopmentStatusID":"6","companyTruncated":"Prothena \/ Novo Nordisk"},{"orgOrder":0,"company":"Prothena","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"PRX004","moa":"ATTR protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Prothena","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prothena \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Prothena \/ Undisclosed"},{"orgOrder":0,"company":"Prothena","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"PRX004","moa":"ATTR protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Prothena","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Prothena \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Prothena \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"TAK-667","moa":"Aurora kinase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"IMMvention Therapeutix","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Partnership","leadProduct":"IMM\u2013004","moa":"BACH1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"IMMvention Therapeutix","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IMMvention Therapeutix \/ Novo Nordisk","highestDevelopmentStatusID":"4","companyTruncated":"IMMvention Therapeutix \/ Novo Nordisk"},{"orgOrder":0,"company":"Insmed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2011","type":"Inapplicable","leadProduct":"Amikacin","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Insmed","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Insmed \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Insmed \/ Undisclosed"},{"orgOrder":0,"company":"Insmed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2011","type":"Inapplicable","leadProduct":"Amikacin","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Insmed","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Insmed \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Insmed \/ Undisclosed"},{"orgOrder":0,"company":"Insmed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2024","type":"Inapplicable","leadProduct":"Amikacin","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Insmed","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nebulizer","sponsorNew":"Insmed \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Insmed \/ Undisclosed"},{"orgOrder":0,"company":"Insmed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2019","type":"Inapplicable","leadProduct":"Amikacin","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Insmed","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Insmed \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Insmed \/ Undisclosed"},{"orgOrder":0,"company":"Seattle Children\u2019s Hospital","sponsor":"National Heart, Lung, and Blood Institute | Cystic Fibrosis Foundation | CF Therapeutics Development Network Coordinating Center","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2016","type":"Inapplicable","leadProduct":"Azithromycin","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Seattle Children\u2019s Hospital","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Seattle Children\u2019s Hospital \/ National Heart, Lung, and Blood Institute | Cystic Fibrosis Foundation | CF Therapeutics Development Network Coordinating Center","highestDevelopmentStatusID":"11","companyTruncated":"Seattle Children\u2019s Hospital \/ National Heart, Lung, and Blood Institute | Cystic Fibrosis Foundation | CF Therapeutics Development Network Coordinating Center"},{"orgOrder":0,"company":"Nabriva Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Lefamulin Acetate","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nabriva Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nabriva Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nabriva Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nabriva Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Lefamulin Acetate","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nabriva Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nabriva Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nabriva Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nabriva Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Lefamulin Acetate","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nabriva Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nabriva Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nabriva Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nabriva Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Lefamulin Acetate","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nabriva Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nabriva Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nabriva Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nabriva Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Lefamulin Acetate","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nabriva Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nabriva Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nabriva Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"National Eye Institute","sponsor":"The Emmes Company, LLC","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Minocycline Hydrochloride","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"National Eye Institute","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Eye Institute \/ The Emmes Company, LLC","highestDevelopmentStatusID":"7","companyTruncated":"National Eye Institute \/ The Emmes Company, LLC"},{"orgOrder":0,"company":"University of Southern California","sponsor":"Merck & Co","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Tedizolid","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"University of Southern California","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Southern California \/ Merck & Co","highestDevelopmentStatusID":"11","companyTruncated":"University of Southern California \/ Merck & Co"},{"orgOrder":0,"company":"Clarametyx Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"CMTX-101","moa":"Bacterial biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Clarametyx Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Clarametyx Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Clarametyx Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Clarametyx Biosciences","sponsor":"KAMRA Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Funding","leadProduct":"CMTX-101","moa":"Bacterial biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Clarametyx Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Clarametyx Biosciences \/ KAMRA Fund","highestDevelopmentStatusID":"7","companyTruncated":"Clarametyx Biosciences \/ KAMRA Fund"},{"orgOrder":0,"company":"Aradigm","sponsor":"Grifols International","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2020","type":"Acquisition","leadProduct":"Ciprofloxacin","moa":"Bacterial DNA gyrase | Topoisomerase IV","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aradigm","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Inhalation","sponsorNew":"Aradigm \/ Grifols International","highestDevelopmentStatusID":"8","companyTruncated":"Aradigm \/ Grifols International"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"CF Therapeutics Development Network Coordinating Center | Seattle Children\u2019s Hospital | Washington University School of Medicine | University of Washington | University of Colorado, Denver | Baylor College of Medicine | University of Alabama at Birmingham","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2011","type":"Inapplicable","leadProduct":"Rifampicin","moa":"Bacterial DNA-directed RNA polymerase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of North Carolina","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of North Carolina \/ CF Therapeutics Development Network Coordinating Center | Seattle Children\u2019s Hospital | Washington University School of Medicine | University of Washington | University of Colorado, Denver | Baylor College of Medicine | University of Alabama at Birmingham","highestDevelopmentStatusID":"8","companyTruncated":"University of North Carolina \/ CF Therapeutics Development Network Coordinating Center | Seattle Children\u2019s Hospital | Washington University School of Medicine | University of Washington | University of Colorado, Denver | Baylor College of Medicine | University of Alabama at Birmingham"},{"orgOrder":0,"company":"Bausch Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Antibiotic","year":"2021","type":"Inapplicable","leadProduct":"Rifaximin","moa":"Bacterial DNA-directed RNA polymerase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bausch Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bausch Health \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bausch Health \/ Undisclosed"},{"orgOrder":0,"company":"Bausch Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Antibiotic","year":"2020","type":"Inapplicable","leadProduct":"Rifaximin","moa":"Bacterial DNA-directed RNA polymerase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bausch Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bausch Health \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bausch Health \/ Undisclosed"},{"orgOrder":0,"company":"Aridis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"AR-501","moa":"Bacterial growth","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aridis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aridis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aridis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Aridis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Gallium Citrate","moa":"Bacterial growth","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aridis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"Aridis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aridis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Aridis Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Funding","leadProduct":"Gallium Citrate","moa":"Bacterial growth","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aridis Pharmaceuticals","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Aerosol","sponsorNew":"Aridis Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"8","companyTruncated":"Aridis Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Aridis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Gallium Citrate","moa":"Bacterial growth","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aridis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"Aridis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aridis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Aridis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Gallium Citrate","moa":"Bacterial growth","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aridis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"Aridis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aridis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Aridis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Gallium Citrate","moa":"Bacterial growth","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aridis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"Aridis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aridis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Aridis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Gallium Citrate","moa":"Bacterial growth","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aridis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"Aridis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aridis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"University of Florida","sponsor":"Tyler's Hope for a Dystonia Cure, Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2011","type":"Inapplicable","leadProduct":"Ampicillin","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"University of Florida","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"University of Florida \/ Tyler's Hope for a Dystonia Cure, Inc","highestDevelopmentStatusID":"6","companyTruncated":"University of Florida \/ Tyler's Hope for a Dystonia Cure, Inc"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"Salus Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2010","type":"Inapplicable","leadProduct":"Aztreonam","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Gilead Sciences \/ Salus Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Gilead Sciences \/ Salus Pharmaceuticals"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2010","type":"Inapplicable","leadProduct":"Aztreonam Lysine","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Gilead Sciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Gilead Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2011","type":"Inapplicable","leadProduct":"Aztreonam Lysine","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Gilead Sciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Gilead Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2011","type":"Inapplicable","leadProduct":"Aztreonam Lysine","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nebulizer","sponsorNew":"Gilead Sciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Gilead Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Hartford Hospital","sponsor":"Shionogi | Keystone Bioanalytical | Indiana University Health Methodist Hospital | University of Pittsburgh Medical Center | University of Texas System","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Cefiderocol","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Hartford Hospital","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hartford Hospital \/ Shionogi | Keystone Bioanalytical | Indiana University Health Methodist Hospital | University of Pittsburgh Medical Center | University of Texas System","highestDevelopmentStatusID":"11","companyTruncated":"Hartford Hospital \/ Shionogi | Keystone Bioanalytical | Indiana University Health Methodist Hospital | University of Pittsburgh Medical Center | University of Texas System"},{"orgOrder":0,"company":"University College Cork","sponsor":"Queen's University, Belfast | Universit\u00e9 Paris Cit\u00e9 | University of Dundee | University of Washington | University Hospital Heidelberg | Teagasc | Clininfo | GABO:mi | Papworth Hospital NHS Foundation Trust | KU Leuven | Assistance Publique \u2013 H\u00f4pitaux de ","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Ceftazidime","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University College Cork","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University College Cork \/ Queen's University, Belfast | Universit\u00e9 Paris Cit\u00e9 | University of Dundee | University of Washington | University Hospital Heidelberg | Teagasc | Clininfo | GABO:mi | Papworth Hospital NHS Foundation Trust | KU Leuven | Assistance Publique \u2013 H\u00f4pitaux de ","highestDevelopmentStatusID":"1","companyTruncated":"University College Cork \/ Queen's University, Belfast | Universit\u00e9 Paris Cit\u00e9 | University of Dundee | University of Washington | University Hospital Heidelberg | Teagasc | Clininfo | GABO:mi | Papworth Hospital NHS Foundation Trust | KU Leuven | Assistance Publique \u2013 H\u00f4pitaux de "},{"orgOrder":0,"company":"Hartford Hospital","sponsor":"Merck & Co | Q2 Solutions | Connecticut Children's Medical Center | St. Christopher's Hospital for Children | UT Southwestern Medical Center | University of Pittsburgh Medical Center | Indiana University Health Methodist Hospital | James Whitcomb Riley Ho","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2022","type":"Inapplicable","leadProduct":"Imipenem","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Hartford Hospital","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hartford Hospital \/ Merck & Co | Q2 Solutions | Connecticut Children's Medical Center | St. Christopher's Hospital for Children | UT Southwestern Medical Center | University of Pittsburgh Medical Center | Indiana University Health Methodist Hospital | James Whitcomb Riley Ho","highestDevelopmentStatusID":"11","companyTruncated":"Hartford Hospital \/ Merck & Co | Q2 Solutions | Connecticut Children's Medical Center | St. Christopher's Hospital for Children | UT Southwestern Medical Center | University of Pittsburgh Medical Center | Indiana University Health Methodist Hospital | James Whitcomb Riley Ho"},{"orgOrder":0,"company":"Unity Health Toronto","sponsor":"Sunnybrook Health Sciences Centre | University of Toronto","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Antibiotic","year":"2010","type":"Inapplicable","leadProduct":"Meropenem","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Unity Health Toronto","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Unity Health Toronto \/ Sunnybrook Health Sciences Centre | University of Toronto","highestDevelopmentStatusID":"1","companyTruncated":"Unity Health Toronto \/ Sunnybrook Health Sciences Centre | University of Toronto"},{"orgOrder":0,"company":"Axovia Therapeutics","sponsor":"A Race Against Blindness","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Funding","leadProduct":"AXV101","moa":"BBS1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Axovia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Axovia Therapeutics \/ A Race Against Blindness","highestDevelopmentStatusID":"4","companyTruncated":"Axovia Therapeutics \/ A Race Against Blindness"},{"orgOrder":0,"company":"Axovia Therapeutics","sponsor":"A Race Against Blindness","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Funding","leadProduct":"AXV-101","moa":"BBS1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Axovia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Axovia Therapeutics \/ A Race Against Blindness","highestDevelopmentStatusID":"4","companyTruncated":"Axovia Therapeutics \/ A Race Against Blindness"},{"orgOrder":0,"company":"Actinium pharmaceuticals","sponsor":"Columbia University","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Radiolabeled Compound","year":"2024","type":"Collaboration","leadProduct":"131-I Apamistamab","moa":"BC8","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Actinium pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Actinium pharmaceuticals \/ Columbia University","highestDevelopmentStatusID":"6","companyTruncated":"Actinium pharmaceuticals \/ Columbia University"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"CRISPR Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"CTX001","moa":"B-cell lymphoma\/leukemia 11A (BCL11A)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ CRISPR Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ CRISPR Therapeutics"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"CRISPR Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"CTX001","moa":"B-cell lymphoma\/leukemia 11A (BCL11A)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ CRISPR Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ CRISPR Therapeutics"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"DFT383","moa":"BCL-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Zentalis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"ZN-d5","moa":"BCL-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Zentalis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zentalis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Zentalis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zentalis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"ZN-D5","moa":"BCL-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Zentalis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zentalis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Zentalis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"IASO Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Equecabtagene Autoleucel","moa":"BCMA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"IASO Bio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IASO Bio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"IASO Bio \/ Undisclosed"},{"orgOrder":0,"company":"Nexcella","sponsor":"Immix Biopharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Immix Biopharma","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Immix Biopharma"},{"orgOrder":0,"company":"Immix Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Immix Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Immix Biopharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Immix Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Nexcella","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Undisclosed"},{"orgOrder":0,"company":"Immix Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Immix Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Immix Biopharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Immix Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Immix Biopharma","sponsor":"Titan Partners Group LLC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Immix Biopharma","amount2":0.02,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Infusion","sponsorNew":"Immix Biopharma \/ Titan Partners Group LLC","highestDevelopmentStatusID":"8","companyTruncated":"Immix Biopharma \/ Titan Partners Group LLC"},{"orgOrder":0,"company":"Immix Biopharma","sponsor":"Titan Partners Group LLC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Immix Biopharma","amount2":0.02,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Infusion","sponsorNew":"Immix Biopharma \/ Titan Partners Group LLC","highestDevelopmentStatusID":"8","companyTruncated":"Immix Biopharma \/ Titan Partners Group LLC"},{"orgOrder":0,"company":"Immix Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Immix Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Immix Biopharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Immix Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Nexcella","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Undisclosed"},{"orgOrder":0,"company":"Nexcella","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Undisclosed"},{"orgOrder":0,"company":"Nexcella","sponsor":"Cirm","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Funding","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Cirm","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Cirm"},{"orgOrder":0,"company":"Immix Biopharma","sponsor":"Cirm","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Funding","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Immix Biopharma","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Infusion","sponsorNew":"Immix Biopharma \/ Cirm","highestDevelopmentStatusID":"8","companyTruncated":"Immix Biopharma \/ Cirm"},{"orgOrder":0,"company":"Immix Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Immix Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Immix Biopharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Immix Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Immix Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Immix Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Immix Biopharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Immix Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Immix Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Immix Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Immix Biopharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Immix Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Nexcella","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Cyclophosphamide","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Undisclosed"},{"orgOrder":0,"company":"Nexcella","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Undisclosed"},{"orgOrder":0,"company":"Immix Biopharma","sponsor":"Goose Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Financing","leadProduct":"NXC-201","moa":"BCMA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Immix Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Immix Biopharma \/ Goose Capital","highestDevelopmentStatusID":"8","companyTruncated":"Immix Biopharma \/ Goose Capital"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"General Atlantic","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Private Placement","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ General Atlantic","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ General Atlantic"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Softgel Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Softgel Capsule","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pharvaris \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Undisclosed"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Viking Global Investors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Series C Financing","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pharvaris","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Pharvaris \/ Viking Global Investors","highestDevelopmentStatusID":"6","companyTruncated":"Pharvaris \/ Viking Global Investors"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"OPGx-BEST1","moa":"BEST1 gene","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Opus Genetics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Opus Genetics \/ Undisclosed"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Salbutamol Sulphate","moa":"Beta-2 adrenergic receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"NYU Langone Health \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"NYU Langone Health \/ Undisclosed"},{"orgOrder":0,"company":"Unity Health Toronto","sponsor":"University of California, San Francisco | The Hospital for Sick Children | University of Toronto | Sunnybrook Health Sciences Centre | Toronto Metropolitan University | National Institute of Neurological Disorders and Stroke","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Timolol Maleate","moa":"Beta-2 adrenergic receptor | Beta-1 adrenergic receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Unity Health Toronto","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Drops","sponsorNew":"Unity Health Toronto \/ University of California, San Francisco | The Hospital for Sick Children | University of Toronto | Sunnybrook Health Sciences Centre | Toronto Metropolitan University | National Institute of Neurological Disorders and Stroke","highestDevelopmentStatusID":"7","companyTruncated":"Unity Health Toronto \/ University of California, San Francisco | The Hospital for Sick Children | University of Toronto | Sunnybrook Health Sciences Centre | Toronto Metropolitan University | National Institute of Neurological Disorders and Stroke"},{"orgOrder":0,"company":"Viralgen","sponsor":"Sio Gene Therapies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell & Gene Therapy","year":"2020","type":"Partnership","leadProduct":"AXO-AAV-GM1","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Viralgen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Viralgen \/ Sio Gene Therapies","highestDevelopmentStatusID":"7","companyTruncated":"Viralgen \/ Sio Gene Therapies"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AXO-AAV-GM1","moa":"||Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sio Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sio Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AXO-AAV-GM1","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sio Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sio Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AXO-AAV-GM1","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sio Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sio Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AXO-AAV-GM1","moa":"||Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sio Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sio Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"National Human Genome Research Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AXO-AAV-GM1","moa":"||Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sio Gene Therapies \/ National Human Genome Research Institute","highestDevelopmentStatusID":"7","companyTruncated":"Sio Gene Therapies \/ National Human Genome Research Institute"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"GEMMA Biotherapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Licensing Agreement","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0.13,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ GEMMA Biotherapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ GEMMA Biotherapeutics"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Public Offering","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0.13,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ J.P. Morgan"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"SRP-9003","moa":"Beta-sarcoglycan (SGCB)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SRP-9003","moa":"Beta-sarcoglycan (SGCB)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"SRP-9003","moa":"Beta-sarcoglycan (SGCB)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Atabecestat","moa":"Beta-secretase 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Akashi Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Halofuginone","moa":"Bifunctional aminoacyl-tRNA synthetase (EPRS)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Akashi Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Akashi Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Akashi Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Processa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Halofuginone","moa":"Bifunctional aminoacyl-tRNA synthetase (EPRS)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Processa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Processa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Processa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Processa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Halofuginone","moa":"Bifunctional aminoacyl-tRNA synthetase (EPRS)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Processa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Processa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Processa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Enyo Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vonafexor","moa":"Bile acid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enyo Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Enyo Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Enyo Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Enyo Pharma","sponsor":"OrbiMed Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2024","type":"Series C Financing","leadProduct":"Vonafexor","moa":"Bile acid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enyo Pharma","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Tablet","sponsorNew":"Enyo Pharma \/ OrbiMed Advisors","highestDevelopmentStatusID":"8","companyTruncated":"Enyo Pharma \/ OrbiMed Advisors"},{"orgOrder":0,"company":"Enyo Pharma","sponsor":"Vesalius Biocapital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2025","type":"Series C Financing","leadProduct":"Vonafexor","moa":"Bile acid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enyo Pharma","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Tablet","sponsorNew":"Enyo Pharma \/ Vesalius Biocapital","highestDevelopmentStatusID":"8","companyTruncated":"Enyo Pharma \/ Vesalius Biocapital"},{"orgOrder":0,"company":"Spinogenix","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"SPG601","moa":"BK","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spinogenix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Spinogenix \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spinogenix \/ Undisclosed"},{"orgOrder":0,"company":"Spinogenix","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"SPG601","moa":"BK","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spinogenix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Spinogenix \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spinogenix \/ Undisclosed"},{"orgOrder":0,"company":"Spinogenix","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SPG601","moa":"BK","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spinogenix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pill","sponsorNew":"Spinogenix \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spinogenix \/ Undisclosed"},{"orgOrder":0,"company":"Spinogenix","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SPG601","moa":"BK","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spinogenix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pill","sponsorNew":"Spinogenix \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spinogenix \/ Undisclosed"},{"orgOrder":0,"company":"Spinogenix","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SPG601","moa":"BK","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spinogenix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pill","sponsorNew":"Spinogenix \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spinogenix \/ Undisclosed"},{"orgOrder":0,"company":"Spinogenix","sponsor":"Avance Clinical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SPG601","moa":"BK","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spinogenix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Spinogenix \/ Avance Clinical","highestDevelopmentStatusID":"8","companyTruncated":"Spinogenix \/ Avance Clinical"},{"orgOrder":0,"company":"Kaerus Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"KER-0193","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Kaerus Bioscience","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kaerus Bioscience \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Kaerus Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Kaerus Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"KER-0193","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Kaerus Bioscience","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kaerus Bioscience \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Kaerus Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Kaerus Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"KER-0193","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Kaerus Bioscience","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kaerus Bioscience \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Kaerus Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Servier","sponsor":"Kaerus Bioscience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2025","type":"Acquisition","leadProduct":"KER-0193","moa":"BK channel","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Servier","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Servier \/ Kaerus Bioscience","highestDevelopmentStatusID":"8","companyTruncated":"Servier \/ Kaerus Bioscience"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"Massachusetts General Hospital","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Massachusetts General Hospital","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Massachusetts General Hospital \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"Massachusetts General Hospital \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Glenmark Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Glenmark Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Glenmark Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Glenmark Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Aurobindo Pharma Limited","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Aurobindo Pharma Limited","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aurobindo Pharma Limited \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Aurobindo Pharma Limited \/ Undisclosed"},{"orgOrder":0,"company":"Alembic Pharmaceuticals Limited","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alembic Pharmaceuticals Limited","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alembic Pharmaceuticals Limited \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alembic Pharmaceuticals Limited \/ Undisclosed"},{"orgOrder":0,"company":"Fresenius Kabi AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Fresenius Kabi AG","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Fresenius Kabi AG \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Fresenius Kabi AG \/ Undisclosed"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Cycle Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Slayback Pharma","sponsor":"Sandoz B2B","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Licensing Agreement","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Slayback Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Slayback Pharma \/ Sandoz B2B","highestDevelopmentStatusID":"15","companyTruncated":"Slayback Pharma \/ Sandoz B2B"},{"orgOrder":0,"company":"Adalvo","sponsor":"AmbioPharm","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"MALTA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Adalvo","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Adalvo \/ AmbioPharm","highestDevelopmentStatusID":"15","companyTruncated":"Adalvo \/ AmbioPharm"},{"orgOrder":0,"company":"Resverlogix","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Apabetalone","moa":"Bromodomain and extra-terminal motif (BET)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Resverlogix","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Resverlogix \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Resverlogix \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Human","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Human","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Human","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Human","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Pharming","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Recombinant","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pharming \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Undisclosed"},{"orgOrder":0,"company":"Pharming","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Recombinant","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pharming \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Undisclosed"},{"orgOrder":0,"company":"Pharming","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Recombinant","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pharming \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Recombinant","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"Pharming","sponsor":"NewBridge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Protein","year":"2021","type":"Agreement","leadProduct":"Conestat Alpha","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Pharming \/ NewBridge Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Pharming \/ NewBridge Pharmaceuticals"},{"orgOrder":0,"company":"IMMUNOe Research Centers","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Protein","year":"2018","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Recombinant","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"IMMUNOe Research Centers","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"IMMUNOe Research Centers \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"IMMUNOe Research Centers \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Human C1 Esterase Inhibitor","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"OCTA-C1-INH","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Pharming","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"OTL-105","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Orchard Therapeutics","amount2":0.20999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Pharming","highestDevelopmentStatusID":"4","companyTruncated":"Orchard Therapeutics \/ Pharming"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"BMN 331","moa":"C1-INH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARO-C3","moa":"C3 receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Silence Therapeutics","sponsor":"Mallinckrodt Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2022","type":"Collaboration","leadProduct":"SLN501","moa":"C3 receptor","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Silence Therapeutics","amount2":2.02,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":2.02,"dosageForm":"Undisclosed","sponsorNew":"Silence Therapeutics \/ Mallinckrodt Pharmaceuticals","highestDevelopmentStatusID":"5","companyTruncated":"Silence Therapeutics \/ Mallinckrodt Pharmaceuticals"},{"orgOrder":0,"company":"IntraBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Amino Acid","year":"2024","type":"Inapplicable","leadProduct":"N-Acetyl-L-Leucine","moa":"Calcium channel","graph1":"Genetic Disease","graph2":"Phase III","graph3":"IntraBio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules for Suspension","sponsorNew":"IntraBio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"IntraBio \/ Undisclosed"},{"orgOrder":0,"company":"Calcilytix Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Encaleret","moa":"calcium receptor antagonists","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Calcilytix Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Calcilytix Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Calcilytix Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Encaleret","moa":"calcium receptor antagonists","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Encaleret","moa":"calcium receptor antagonists","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Calcilytix Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Encaleret","moa":"calcium receptor antagonists","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BridgeBio Pharma \/ Calcilytix Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Calcilytix Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Encaleret","moa":"calcium receptor antagonists","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Cad-1883","moa":"Calcium-activated potassium channel KCa2 (KCNN)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Rimtuzalcap","moa":"Calcium-sensitive potassium ion channel PAM","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Kither Biotech","sponsor":"Avance Clinical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"KIT2014","moa":"cAMP","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Kither Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Kither Biotech \/ Avance Clinical","highestDevelopmentStatusID":"6","companyTruncated":"Kither Biotech \/ Avance Clinical"},{"orgOrder":0,"company":"Kither Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"KIT2014","moa":"cAMP","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Kither Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Kither Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Kither Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Kither Biotech","sponsor":"Claris Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Peptide, Unconjugated","year":"2022","type":"Series B Financing","leadProduct":"KIT2014","moa":"cAMP","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Kither Biotech","amount2":0.02,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Inhalation","sponsorNew":"Kither Biotech \/ Claris Ventures","highestDevelopmentStatusID":"6","companyTruncated":"Kither Biotech \/ Claris Ventures"},{"orgOrder":0,"company":"Northwell Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2015","type":"Inapplicable","leadProduct":"Alemtuzumab","moa":"CAMPATH-1 antigen","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Northwell Health","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Northwell Health \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Northwell Health \/ Undisclosed"},{"orgOrder":0,"company":"Hackensack Meridian Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2013","type":"Inapplicable","leadProduct":"Alemtuzumab","moa":"CAMPATH-1 antigen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Hackensack Meridian Health","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Hackensack Meridian Health \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Hackensack Meridian Health \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Moderna Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"VX-522","moa":"cAMP-dependent chloride channel (CFTR)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"Vertex Pharmaceuticals \/ Moderna Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Vertex Pharmaceuticals \/ Moderna Therapeutics"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"VX-522","moa":"cAMP-dependent chloride channel (CFTR)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2021","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor||CB1\/CB2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2018","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor||CB1\/CB2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2019","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor||CB1\/CB2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2021","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor||CB1\/CB2 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Universit\u00e9 de Sherbrooke","sponsor":"Canadian Institutes of Health Research | Jazz Pharmaceuticals | Centre de recherche du Centre hospitalier universitaire de Sherbrooke","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Controlled Substance","year":"2024","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Universit\u00e9 de Sherbrooke","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Universit\u00e9 de Sherbrooke \/ Canadian Institutes of Health Research | Jazz Pharmaceuticals | Centre de recherche du Centre hospitalier universitaire de Sherbrooke","highestDevelopmentStatusID":"8","companyTruncated":"Universit\u00e9 de Sherbrooke \/ Canadian Institutes of Health Research | Jazz Pharmaceuticals | Centre de recherche du Centre hospitalier universitaire de Sherbrooke"},{"orgOrder":0,"company":"Universit\u00e9 de Sherbrooke","sponsor":"Canadian Institutes of Health Research | Jazz Pharmaceuticals | Centre de recherche du Centre hospitalier universitaire de Sherbrooke","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Controlled Substance","year":"2024","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Universit\u00e9 de Sherbrooke","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Universit\u00e9 de Sherbrooke \/ Canadian Institutes of Health Research | Jazz Pharmaceuticals | Centre de recherche du Centre hospitalier universitaire de Sherbrooke","highestDevelopmentStatusID":"8","companyTruncated":"Universit\u00e9 de Sherbrooke \/ Canadian Institutes of Health Research | Jazz Pharmaceuticals | Centre de recherche du Centre hospitalier universitaire de Sherbrooke"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Harmony Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2023","type":"Acquisition","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20000000000000001,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Harmony Biosciences","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Harmony Biosciences"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2023","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2020","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Harmony Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2023","type":"Acquisition","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20000000000000001,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Harmony Biosciences","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Harmony Biosciences"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Lincoln Park Capital Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Agreement","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Lincoln Park Capital Fund","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Lincoln Park Capital Fund"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2025","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Harmony Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Harmony Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2020","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2021","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2020","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2020","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2020","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2020","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2020","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Radius Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2016","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Radius Health","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Radius Health \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Radius Health \/ Undisclosed"},{"orgOrder":0,"company":"Radius Health","sponsor":"Benuvia Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2018","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Radius Health","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Radius Health \/ Benuvia Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Radius Health \/ Benuvia Therapeutics"},{"orgOrder":0,"company":"Benuvia Therapeutics","sponsor":"Radius Health","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2021","type":"Acquisition","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Benuvia Therapeutics","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Solution","sponsorNew":"Benuvia Therapeutics \/ Radius Health","highestDevelopmentStatusID":"8","companyTruncated":"Benuvia Therapeutics \/ Radius Health"},{"orgOrder":0,"company":"Radius Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Radius Health","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Radius Health \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Radius Health \/ Undisclosed"},{"orgOrder":0,"company":"Radius Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2021","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Radius Health","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Radius Health \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Radius Health \/ Undisclosed"},{"orgOrder":0,"company":"Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal","sponsor":"GW Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Controlled Substance","year":"2011","type":"Inapplicable","leadProduct":"Tetrahydrocannabinol","moa":"Cannabinoid CB1 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal \/ GW Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal \/ GW Pharmaceutical"},{"orgOrder":0,"company":"Montefiore Medical Center","sponsor":"Foundation for Prader-Willi Research | GW Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Cannabidivarin","moa":"cannabinol derivatives","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Montefiore Medical Center","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Montefiore Medical Center \/ Foundation for Prader-Willi Research | GW Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Montefiore Medical Center \/ Foundation for Prader-Willi Research | GW Pharmaceutical"},{"orgOrder":0,"company":"Corbus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Lenabasum","moa":"cannabinol derivatives","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Corbus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Corbus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Corbus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Corbus Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Lenabasum","moa":"cannabinol derivatives","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Corbus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Corbus Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"8","companyTruncated":"Corbus Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Corbus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Lenabasum","moa":"cannabinol derivatives","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Corbus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Corbus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Corbus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Corbus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Lenabasum","moa":"cannabinol derivatives","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Corbus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Corbus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Corbus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Corbus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Lenabasum","moa":"cannabinol derivatives","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Corbus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Corbus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Corbus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Torrent Pharmaceuticals Limited","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Dichlorphenamide","moa":"Carbonic anhydrase I | Carbonic anhydrase II | Carbonic anhydrase XII | Carbonic anhydrase IV","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Torrent Pharmaceuticals Limited","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Torrent Pharmaceuticals Limited \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Torrent Pharmaceuticals Limited \/ Undisclosed"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Torrent Pharmaceuticals Limited","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Dichlorphenamide","moa":"Carbonic anhydrase I | Carbonic anhydrase II | Carbonic anhydrase XII | Carbonic anhydrase IV","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cycle Pharmaceuticals \/ Torrent Pharmaceuticals Limited","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Torrent Pharmaceuticals Limited"},{"orgOrder":0,"company":"Strongbridge Biopharma","sponsor":"Xeris Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Acquisition","leadProduct":"Dichlorphenamide","moa":"Carbonic anhydrase I | Carbonic anhydrase II | Carbonic anhydrase XII | Carbonic anhydrase IV","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Strongbridge Biopharma","amount2":0.27000000000000002,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.27000000000000002,"dosageForm":"Tablet","sponsorNew":"Strongbridge Biopharma \/ Xeris Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Strongbridge Biopharma \/ Xeris Pharmaceuticals"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SGT-501","moa":"CASQ2 | RYR2","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SGT-501","moa":"CASQ2\/RYR2","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Boston University","sponsor":"Corino Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Tolcapone","moa":"Catechol O-methyltransferase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Boston University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boston University \/ Corino Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Boston University \/ Corino Therapeutics"},{"orgOrder":0,"company":"Corino Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tolcapone","moa":"Catechol O-methyltransferase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Corino Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Corino Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Corino Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Hospital de Clinicas de Porto Alegre","sponsor":"Fundo de Incentivo \u00e0 Pesquisa do Hospital de Cl\u00ednicas de Porto Alegre | Associa\u00e7\u00e3o de Amigos da Hematologia | Ajinomoto Company","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BRAZIL","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2010","type":"Inapplicable","leadProduct":"L-Arginine","moa":"Cationic amino acid transporter 3; Antizyme inhibitor 2; Argininosuccinate lyase; Nitric oxide synthase, inducible; High affinity cationic amino acid transporter 1; Arginase-2, mitochondrial; Cationic amino acid transporter 4; Argininosuccinate synthase; Nitric oxide synthase 3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Hospital de Clinicas de Porto Alegre","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hospital de Clinicas de Porto Alegre \/ Fundo de Incentivo \u00e0 Pesquisa do Hospital de Cl\u00ednicas de Porto Alegre | Associa\u00e7\u00e3o de Amigos da Hematologia | Ajinomoto Company","highestDevelopmentStatusID":"10","companyTruncated":"Hospital de Clinicas de Porto Alegre \/ Fundo de Incentivo \u00e0 Pesquisa do Hospital de Cl\u00ednicas de Porto Alegre | Associa\u00e7\u00e3o de Amigos da Hematologia | Ajinomoto Company"},{"orgOrder":0,"company":"Gabrail Cancer Center Research","sponsor":"Children's Healthcare of Atlanta | National Center for Complementary and Integrative Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2015","type":"Inapplicable","leadProduct":"L-Arginine","moa":"Cationic amino acid transporter 3; Antizyme inhibitor 2; Argininosuccinate lyase; Nitric oxide synthase, inducible; High affinity cationic amino acid transporter 1; Arginase-2, mitochondrial; Cationic amino acid transporter 4; Argininosuccinate synthase; Nitric oxide synthase 3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Gabrail Cancer Center Research","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gabrail Cancer Center Research \/ Children's Healthcare of Atlanta | National Center for Complementary and Integrative Health","highestDevelopmentStatusID":"8","companyTruncated":"Gabrail Cancer Center Research \/ Children's Healthcare of Atlanta | National Center for Complementary and Integrative Health"},{"orgOrder":0,"company":"Emerald Health Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2020","type":"Inapplicable","leadProduct":"Cannabigerol","moa":"CB1 receptor","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Emerald Health Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Emerald Health Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Emerald Health Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Inversago Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"INV-101","moa":"CB1 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Inversago Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Inversago Pharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Inversago Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inversago Pharma","sponsor":"Forbion","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2020","type":"Series B Financing","leadProduct":"INV-101","moa":"CB1 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Inversago Pharma","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Tablet","sponsorNew":"Inversago Pharma \/ Forbion","highestDevelopmentStatusID":"6","companyTruncated":"Inversago Pharma \/ Forbion"},{"orgOrder":0,"company":"Inversago Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"INV-101","moa":"CB1 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Inversago Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Inversago Pharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Inversago Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inversago Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Monlunabant","moa":"CB1 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Inversago Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inversago Pharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Inversago Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inversago Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Monlunabant","moa":"CB1 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Inversago Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inversago Pharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Inversago Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Radius Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"RAD011","moa":"CB1\/2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Radius Health","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Radius Health \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Radius Health \/ Undisclosed"},{"orgOrder":0,"company":"Radius Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2021","type":"Inapplicable","leadProduct":"RAD011","moa":"CB1\/2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Radius Health","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Radius Health \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Radius Health \/ Undisclosed"},{"orgOrder":0,"company":"CytoDyn","sponsor":"Amarex Clinical Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Leronlimab","moa":"C-C chemokine receptor type 5","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CytoDyn","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CytoDyn \/ Amarex Clinical Research","highestDevelopmentStatusID":"8","companyTruncated":"CytoDyn \/ Amarex Clinical Research"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Magenta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2020","type":"Collaboration","leadProduct":"MGTA-117","moa":"CD117","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beam Therapeutics \/ Magenta Therapeutics","highestDevelopmentStatusID":"5","companyTruncated":"Beam Therapeutics \/ Magenta Therapeutics"},{"orgOrder":0,"company":"Medexus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"APVO101","moa":"CD19\/CD3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medexus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Medexus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Medexus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biotest","sponsor":"Syneos Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"BT595","moa":"CD20","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biotest","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biotest \/ Syneos Health","highestDevelopmentStatusID":"10","companyTruncated":"Biotest \/ Syneos Health"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Calico Life Sciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"ABBV-CLS-628","moa":"CD3\/CLDN18.2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AbbVie Inc \/ Calico Life Sciences","highestDevelopmentStatusID":"8","companyTruncated":"AbbVie Inc \/ Calico Life Sciences"},{"orgOrder":0,"company":"RoslinCT","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Collaboration","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"RoslinCT","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"RoslinCT \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"RoslinCT \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Lonza Group","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2024","type":"Agreement","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Lonza Group","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lonza Group \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Lonza Group \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Immunogen","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Licensing Agreement","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Immunogen","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"Infusion","sponsorNew":"Immunogen \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Immunogen \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"CRISPR Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ CRISPR Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ CRISPR Therapeutics"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CRISPR Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CRISPR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CRISPR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Collaboration","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Charles River Laboratories, Inc \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Charles River Laboratories, Inc \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Licensing Agreement","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Editas Medicine \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"NHS England","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Agreement","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ NHS England","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ NHS England"},{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CRISPR Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CRISPR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CRISPR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CRISPR Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CRISPR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CRISPR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CRISPR Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CRISPR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CRISPR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"CRISPR Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ CRISPR Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ CRISPR Therapeutics"},{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CRISPR Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CRISPR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CRISPR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CRISPR Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CRISPR Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"CRISPR Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Collaboration","leadProduct":"Exagamglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CRISPR Therapeutics","amount2":1.1000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":1.1000000000000001,"dosageForm":"Infusion","sponsorNew":"CRISPR Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"CRISPR Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2014","type":"Inapplicable","leadProduct":"Lovotibeglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Bluebird Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Lovotibeglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Bluebird Bio","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Bluebird Bio"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Medicaid","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Agreement","leadProduct":"Lovotibeglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Medicaid","highestDevelopmentStatusID":"15","companyTruncated":"Bluebird Bio \/ Medicaid"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Lovotibeglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Hercules Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Financing","leadProduct":"Lovotibeglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Hercules Capital","highestDevelopmentStatusID":"15","companyTruncated":"Bluebird Bio \/ Hercules Capital"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Lovotibeglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Lovotibeglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Lovotibeglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Lovotibeglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Financing","leadProduct":"Lovotibeglogene Autotemcel","moa":"CD34","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Bluebird Bio","amount2":0.10000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"15","companyTruncated":"Bluebird Bio \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Gamida Cell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Cell & Gene Therapy","year":"2012","type":"Inapplicable","leadProduct":"Omidubicel","moa":"CD34","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Gamida Cell","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gamida Cell \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Gamida Cell \/ Undisclosed"},{"orgOrder":0,"company":"New York Medical College","sponsor":"UCSF Benioff Children's Hospital Oakland | Medical College of Wisconsin | Washington University School of Medicine | Tufts Medical Center | University of California, San Francisco | University of California, Los Angeles | Miltenyi Biomedicine | Ann & Robe","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"CD34 selected T-cell depleted allogeneic SCT","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase II","graph3":"New York Medical College","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"New York Medical College \/ UCSF Benioff Children's Hospital Oakland | Medical College of Wisconsin | Washington University School of Medicine | Tufts Medical Center | University of California, San Francisco | University of California, Los Angeles | Miltenyi Biomedicine | Ann & Robe","highestDevelopmentStatusID":"8","companyTruncated":"New York Medical College \/ UCSF Benioff Children's Hospital Oakland | Medical College of Wisconsin | Washington University School of Medicine | Tufts Medical Center | University of California, San Francisco | University of California, Los Angeles | Miltenyi Biomedicine | Ann & Robe"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"EDIT-301","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"DRI Healthcare Trust","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Financing","leadProduct":"Renizgamglogene Autogedtemcel","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ DRI Healthcare Trust","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ DRI Healthcare Trust"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Renizgamglogene Autogedtemcel","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Azzur Group","sponsor":"Editas Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Agreement","leadProduct":"Renizgamglogene Autogedtemcel","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Azzur Group","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Azzur Group \/ Editas Medicine","highestDevelopmentStatusID":"7","companyTruncated":"Azzur Group \/ Editas Medicine"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Azzur Group","sponsor":"Editas Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Partnership","leadProduct":"Renizgamglogene Autogedtemcel","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Azzur Group","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Azzur Group \/ Editas Medicine","highestDevelopmentStatusID":"7","companyTruncated":"Azzur Group \/ Editas Medicine"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Renizgamglogene Autogedtemcel","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Sorrento Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"STI-6129","moa":"CD38","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sorrento Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sorrento Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sorrento Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sorrento Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"STI-6129","moa":"CD38","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sorrento Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sorrento Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sorrento Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jiayin Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"EXG110","moa":"CD39","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jiayin Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Jiayin Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jiayin Biotech \/ Undisclosed"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Etentamig","moa":"CD3E","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AbbVie Inc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AbbVie Inc \/ Undisclosed"},{"orgOrder":0,"company":"Columbia University","sponsor":"Actinium pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Radiolabeled Compound","year":"2025","type":"Inapplicable","leadProduct":"131-I Apamistmab","moa":"CD45","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Columbia University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Columbia University \/ Actinium pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Columbia University \/ Actinium pharmaceuticals"},{"orgOrder":0,"company":"Expression Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"CD68-ET3-LV Gene Therapy","moa":"CD68","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Expression Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Expression Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Expression Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spexis","sponsor":"SPRIM Global Investments","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibiotic","year":"2023","type":"Partnership","leadProduct":"Colistimethate Sodium","moa":"Cell membrane","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Spexis","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Spexis \/ SPRIM Global Investments","highestDevelopmentStatusID":"10","companyTruncated":"Spexis \/ SPRIM Global Investments"},{"orgOrder":0,"company":"Spexis","sponsor":"SPRIM Global Investments","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibiotic","year":"2023","type":"Financing","leadProduct":"Colistimethate Sodium","moa":"Cell membrane","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Spexis","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Spexis \/ SPRIM Global Investments","highestDevelopmentStatusID":"10","companyTruncated":"Spexis \/ SPRIM Global Investments"},{"orgOrder":0,"company":"Spexis","sponsor":"SPRIM Global Investments","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibiotic","year":"2023","type":"Financing","leadProduct":"Colistimethate Sodium","moa":"Cell membrane","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Spexis","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Spexis \/ SPRIM Global Investments","highestDevelopmentStatusID":"10","companyTruncated":"Spexis \/ SPRIM Global Investments"},{"orgOrder":0,"company":"National Jewish Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"Colistin Sulfate","moa":"Cell outer membrane","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"National Jewish Health","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Jewish Health \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"National Jewish Health \/ Undisclosed"},{"orgOrder":0,"company":"Joseph Kuti","sponsor":"Cumberland Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Telavancin","moa":"Cell outer membrane","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Joseph Kuti","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Joseph Kuti \/ Cumberland Pharmaceuticals","highestDevelopmentStatusID":"11","companyTruncated":"Joseph Kuti \/ Cumberland Pharmaceuticals"},{"orgOrder":0,"company":"National Taiwan University Hospital","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"TAIWAN","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Eliglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"National Taiwan University Hospital","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"National Taiwan University Hospital \/ Sanofi","highestDevelopmentStatusID":"1","companyTruncated":"National Taiwan University Hospital \/ Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Eliglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Eliglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Eliglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Eliglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Eliglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Eliglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Eliglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Eliglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Eliglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Eliglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Lucerastat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Idorsia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Idorsia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Idorsia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Lucerastat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Idorsia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Idorsia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Idorsia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Lucerastat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Idorsia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Idorsia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Idorsia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Lucerastat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Idorsia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Idorsia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Idorsia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Lucerastat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Idorsia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Idorsia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Idorsia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"National Taiwan University Hospital","sponsor":"Johnson & Johnson","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"TAIWAN","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"National Taiwan University Hospital","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Taiwan University Hospital \/ Johnson & Johnson","highestDevelopmentStatusID":"1","companyTruncated":"National Taiwan University Hospital \/ Johnson & Johnson"},{"orgOrder":0,"company":"Assistance Publique \u2013 H\u00f4pitaux de Paris","sponsor":"Johnson & Johnson | Centres de Ressources et de Comp\u00e9tences de la Mucoviscidose","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Assistance Publique \u2013 H\u00f4pitaux de Paris","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Johnson & Johnson | Centres de Ressources et de Comp\u00e9tences de la Mucoviscidose","highestDevelopmentStatusID":"9","companyTruncated":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Johnson & Johnson | Centres de Ressources et de Comp\u00e9tences de la Mucoviscidose"},{"orgOrder":0,"company":"Breckenridge Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Breckenridge Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Breckenridge Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Breckenridge Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AT-GTX-501","moa":"Ceroid-lipofuscinosis neuronal protein 6 (CLN6)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AT-GTX-501","moa":"Ceroid-lipofuscinosis neuronal protein 6 (CLN6)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"4D-710","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"4D-710","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"4D-710","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"4D-710","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"4D-710","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Novotech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Novotech","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Novotech"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"The Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Funding","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ The Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ The Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Funding","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"OXB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"BI 3720931","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Boehringer Ingelheim GmbH \/ OXB","highestDevelopmentStatusID":"7","companyTruncated":"Boehringer Ingelheim GmbH \/ OXB"},{"orgOrder":0,"company":"Porosome Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"CF PT-101","moa":"CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Porosome Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Porosome Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Porosome Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Deutivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Deutivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Concert Pharmaceuticals","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Agreement","leadProduct":"Deutivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Concert Pharmaceuticals","amount2":0.25,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.25,"dosageForm":"Tablet","sponsorNew":"Concert Pharmaceuticals \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Concert Pharmaceuticals \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Deutivacaftor","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Hercules Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Financing","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Hercules Capital","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Hercules Capital"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zikani Therapeutics","sponsor":"Eloxx Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Acquisition","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zikani Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Injection","sponsorNew":"Zikani Therapeutics \/ Eloxx Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Zikani Therapeutics \/ Eloxx Pharmaceuticals"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Funding","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"The Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Funding","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ The Cystic Fibrosis Foundation","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ The Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"H.C. Wainwright & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ H.C. Wainwright & Co","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ H.C. Wainwright & Co"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Exaluren Sulfate","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Galapagos","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"GLPG1837","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Galapagos","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Galapagos \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Galapagos \/ Undisclosed"},{"orgOrder":0,"company":"Galapagos","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"GLPG1837","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Galapagos","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Galapagos \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Galapagos \/ Undisclosed"},{"orgOrder":0,"company":"Galapagos","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"GLPG2451","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Galapagos","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Galapagos \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Galapagos \/ Undisclosed"},{"orgOrder":0,"company":"Galapagos","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"GLPG2737","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Galapagos","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Galapagos \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Galapagos \/ Undisclosed"},{"orgOrder":0,"company":"Galapagos","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"GLPG2737","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Galapagos","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Galapagos \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Galapagos \/ Undisclosed"},{"orgOrder":0,"company":"Galapagos","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"GLPG2737","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Galapagos","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Galapagos \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Galapagos \/ Undisclosed"},{"orgOrder":0,"company":"Galapagos","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"GLPG2737","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Galapagos","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Galapagos \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Galapagos \/ Undisclosed"},{"orgOrder":0,"company":"Royal College of Surgeons, Ireland","sponsor":"Imperial College London | University College Dublin | University of Limerick | Cystic Fibrosis Registry of Ireland | Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation | Medizinische Hochschule Brandenb","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Kaftrio","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Royal College of Surgeons, Ireland","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Royal College of Surgeons, Ireland \/ Imperial College London | University College Dublin | University of Limerick | Cystic Fibrosis Registry of Ireland | Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation | Medizinische Hochschule Brandenb","highestDevelopmentStatusID":"11","companyTruncated":"Royal College of Surgeons, Ireland \/ Imperial College London | University College Dublin | University of Limerick | Cystic Fibrosis Registry of Ireland | Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation | Medizinische Hochschule Brandenb"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"KB407","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Krystal Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"KB407","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Krystal Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"KB407","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Krystal Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"KB407","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Krystal Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"KB407","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Krystal Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"KB407","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Krystal Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Novotech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"KB407","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nebulizer","sponsorNew":"Krystal Biotech \/ Novotech","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Novotech"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"KB407","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nebulizer","sponsorNew":"Krystal Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2017","type":"Inapplicable","leadProduct":"MRT5005","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"MRT5005","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"MRT5005","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"RCT223","moa":"CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spirovant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Doxorubicin","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Spirovant Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Spirovant Sciences \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Spirovant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Spirovant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"SPIRO-2101","moa":"CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Spirovant Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Spirovant Sciences \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Spirovant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"SpliSense","sponsor":"Biotel Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Oligonucleotide","year":"2021","type":"Series B Financing","leadProduct":"SPL84-23","moa":"CFTR","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"SpliSense","amount2":0.029999999999999999,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Inhalation","sponsorNew":"SpliSense \/ Biotel Limited","highestDevelopmentStatusID":"5","companyTruncated":"SpliSense \/ Biotel Limited"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Vanzacaftor","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"NHS England","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"Deutivacaftor","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ NHS England","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ NHS England"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"VX-118","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"VX-152","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"VX-668","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"VX-670","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"VX-670","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SION-109","moa":"CFTR ICL4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sionna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sionna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sionna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"SION-451","moa":"CFTR NBD1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sionna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Sionna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sionna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"SION-719","moa":"CFTR NBD1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sionna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sionna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sionna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"SpliSense","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"SPL23-ASO","moa":"CFTR RNA","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SpliSense","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"SpliSense \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"SpliSense \/ Undisclosed"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"RST-001","moa":"Channelrhodopsin-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AbbVie Inc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AbbVie Inc \/ Undisclosed"},{"orgOrder":0,"company":"Tavanta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"TAVT-135","moa":"Chloride ion","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Tavanta Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tavanta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Tavanta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Ursodeoxycholic Acid","moa":"Bile acid receptor FXR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Childhood Liver Disease Network","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Ursodeoxycholic Acid","moa":"Bile acid receptor FXR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mirum Pharmaceuticals \/ Childhood Liver Disease Network","highestDevelopmentStatusID":"8","companyTruncated":"Mirum Pharmaceuticals \/ Childhood Liver Disease Network"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Ovid Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Soticlestat","moa":"Cholesterol 24-hydroxylase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Takeda Pharmaceutical \/ Ovid Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Takeda Pharmaceutical \/ Ovid Therapeutics"},{"orgOrder":0,"company":"ZyVersa Therapeutics, Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"Cholesterol and lipid accumulation","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ZyVersa Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ZyVersa Therapeutics, Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"ZyVersa Therapeutics, Inc \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2011","type":"Inapplicable","leadProduct":"Sebelipase Alfa","moa":"Cholesterol ester | Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2011","type":"Inapplicable","leadProduct":"Sebelipase Alfa","moa":"Cholesterol ester | Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"NMD PHARMA \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NMD PHARMA \/ Undisclosed"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"NMD PHARMA \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NMD PHARMA \/ Undisclosed"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"NMD PHARMA \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NMD PHARMA \/ Undisclosed"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"NMD PHARMA \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NMD PHARMA \/ Undisclosed"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"NMD PHARMA \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NMD PHARMA \/ Undisclosed"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Organovo Holdings","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2023","type":"Series B Financing","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NMD PHARMA","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Tablet","sponsorNew":"NMD PHARMA \/ Organovo Holdings","highestDevelopmentStatusID":"8","companyTruncated":"NMD PHARMA \/ Organovo Holdings"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"NMD PHARMA \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NMD PHARMA \/ Undisclosed"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NMD PHARMA \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"NMD PHARMA \/ Undisclosed"},{"orgOrder":0,"company":"University of L\u2019Aquila","sponsor":"SiSaf Ltd","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Oligonucleotide","year":"2021","type":"Collaboration","leadProduct":"SIS-101-ADO","moa":"CLCN7","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"University of L\u2019Aquila","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of L\u2019Aquila \/ SiSaf Ltd","highestDevelopmentStatusID":"5","companyTruncated":"University of L\u2019Aquila \/ SiSaf Ltd"},{"orgOrder":0,"company":"SiSaf","sponsor":"CSSi LifeSciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"SIS-101-ADO","moa":"CLCN7","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"SiSaf","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SiSaf \/ CSSi LifeSciences","highestDevelopmentStatusID":"5","companyTruncated":"SiSaf \/ CSSi LifeSciences"},{"orgOrder":0,"company":"SiSaf","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"SIS-101-ADO","moa":"CLCN7","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"SiSaf","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SiSaf \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"SiSaf \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Eftrenonacog Alpha","moa":"Coagulation factor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Medicure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Pyridoxal Phosphate","moa":"Coagulation factor IIa (F2)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medicure","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Medicure \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Medicure \/ Undisclosed"},{"orgOrder":0,"company":"Medicure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Pyridoxal Phosphate","moa":"Coagulation factor IIa (F2)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medicure","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Medicure \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Medicure \/ Undisclosed"},{"orgOrder":0,"company":"Medicure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Pyridoxal Phosphate","moa":"Coagulation factor IIa (F2)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medicure","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Medicure \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Medicure \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"AAV5-hFIXco-Padua","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"AAV5-hFIXco-Padua","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Albutrepenonacog Alfa","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Albutrepenonacog Alfa","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"CSL Behring","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ CSL Behring","highestDevelopmentStatusID":"15","companyTruncated":"Uniqure \/ CSL Behring"},{"orgOrder":0,"company":"Uniqure","sponsor":"CSL Behring","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Uniqure","amount2":2.0499999999999998,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":2.0499999999999998,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ CSL Behring","highestDevelopmentStatusID":"15","companyTruncated":"Uniqure \/ CSL Behring"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"CSL Behring","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2023","type":"Licensing Agreement","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Uniqure","amount2":2.0499999999999998,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":2.0499999999999998,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ CSL Behring","highestDevelopmentStatusID":"15","companyTruncated":"Uniqure \/ CSL Behring"},{"orgOrder":0,"company":"Uniqure","sponsor":"HealthCare Royalty","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2023","type":"Divestment","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Uniqure","amount2":0.40000000000000002,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.40000000000000002,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ HealthCare Royalty","highestDevelopmentStatusID":"15","companyTruncated":"Uniqure \/ HealthCare Royalty"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"CSL Behring","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Licensing Agreement","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":2.0499999999999998,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ CSL Behring","highestDevelopmentStatusID":"15","companyTruncated":"Uniqure \/ CSL Behring"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Orsini Specialty Pharmacy","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Partnership","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Orsini Specialty Pharmacy","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Orsini Specialty Pharmacy"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"Fidanacogene elaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Fidanacogene elaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Fidanacogene elaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Fidanacogene elaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Labcorp","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Fidanacogene elaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Labcorp","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Labcorp \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Labcorp \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Fidanacogene elaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Fidanacogene elaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"IntraBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"IB1001","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase II","graph3":"IntraBio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IntraBio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"IntraBio \/ Undisclosed"},{"orgOrder":0,"company":"Medexus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"IB1001","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medexus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Medexus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Medexus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANGENE CORPORATION","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"IB1001","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CANGENE CORPORATION","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CANGENE CORPORATION \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CANGENE CORPORATION \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Nonacog Beta Pegol","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"PF-06838435","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Recombinant Factor IX","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2015","type":"Inapplicable","leadProduct":"SPK-9001","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2015","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orsini Specialty Pharmacy \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Arup Laboratories","sponsor":"Medicover","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Partnership","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Arup Laboratories","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arup Laboratories \/ Medicover","highestDevelopmentStatusID":"15","companyTruncated":"Arup Laboratories \/ Medicover"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Mim8","moa":"Coagulation factor IX (F9)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Mim8","moa":"Coagulation factor IX (F9)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Mim8","moa":"Coagulation factor IX (F9)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Mim8","moa":"Coagulation factor IX (F9)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Mim8","moa":"Coagulation factor IX (F9)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Mim8","moa":"Coagulation factor IX (F9)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2016","type":"Inapplicable","leadProduct":"Sb-Fix","moa":"Coagulation factor IX (F9)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chugai Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Chugai Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chugai Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Chugai Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chugai Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Chugai Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chugai Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Chugai Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2015","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"Indiana Hemophilia &Thrombosis Center, Inc.","sponsor":"Genentech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Indiana Hemophilia &Thrombosis Center, Inc.","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Indiana Hemophilia &Thrombosis Center, Inc. \/ Genentech","highestDevelopmentStatusID":"11","companyTruncated":"Indiana Hemophilia &Thrombosis Center, Inc. \/ Genentech"},{"orgOrder":0,"company":"Tulane University","sponsor":"Genentech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tulane University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tulane University \/ Genentech","highestDevelopmentStatusID":"6","companyTruncated":"Tulane University \/ Genentech"},{"orgOrder":0,"company":"University of Washington","sponsor":"Genentech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of Washington","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"University of Washington \/ Genentech","highestDevelopmentStatusID":"8","companyTruncated":"University of Washington \/ Genentech"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genentech \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"Margaret Ragni","sponsor":"Health Resources and Services Administration","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Margaret Ragni","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Margaret Ragni \/ Health Resources and Services Administration","highestDevelopmentStatusID":"10","companyTruncated":"Margaret Ragni \/ Health Resources and Services Administration"},{"orgOrder":0,"company":"Gabrail Cancer Center Research","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Gabrail Cancer Center Research","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gabrail Cancer Center Research \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Gabrail Cancer Center Research \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Chia Tai Tianqing Pharmaceutical Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Eptacog Alfa","moa":"Coagulation factor VII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chia Tai Tianqing Pharmaceutical Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed"},{"orgOrder":0,"company":"Aryogen Pharmed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRAN","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Eptacog Alfa","moa":"Coagulation factor VII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Aryogen Pharmed","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aryogen Pharmed \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Aryogen Pharmed \/ Undisclosed"},{"orgOrder":0,"company":"Aryogen Pharmed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRAN","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Eptacog Alfa","moa":"Coagulation factor VII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Aryogen Pharmed","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Aryogen Pharmed \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Aryogen Pharmed \/ Undisclosed"},{"orgOrder":0,"company":"Sinocelltech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"SCT800","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sinocelltech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sinocelltech \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sinocelltech \/ Undisclosed"},{"orgOrder":0,"company":"Sinocelltech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"SCT800","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sinocelltech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sinocelltech \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sinocelltech \/ Undisclosed"},{"orgOrder":0,"company":"Zhengzhou Gensciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Efmoroctocog Alfa","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zhengzhou Gensciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zhengzhou Gensciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zhengzhou Gensciences \/ Undisclosed"},{"orgOrder":0,"company":"Zhengzhou Gensciences","sponsor":"Jiangsu Gensciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Efmoroctocog Alfa","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zhengzhou Gensciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zhengzhou Gensciences \/ Jiangsu Gensciences","highestDevelopmentStatusID":"10","companyTruncated":"Zhengzhou Gensciences \/ Jiangsu Gensciences"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"BAY81-8973","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Damoctocog Alfa Pegol","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Damoctocog Alfa Pegol","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Damoctocog Alfa Pegol","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Damoctocog Alfa Pegol","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Efmoroctocog Alfa","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Swedish Orphan Biovitrum AB \/ Sanofi","highestDevelopmentStatusID":"11","companyTruncated":"Swedish Orphan Biovitrum AB \/ Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Efmoroctocog Alfa","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Collaboration","leadProduct":"Giroctocogene fitelparvovec","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sangamo Therapeutics","amount2":2.7200000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":2.7200000000000002,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Biogen","highestDevelopmentStatusID":"10","companyTruncated":"Sangamo Therapeutics \/ Biogen"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Sangamo Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Giroctocogene fitelparvovec","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Sangamo Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Sangamo Therapeutics"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Sangamo Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Giroctocogene fitelparvovec","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Sangamo Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Sangamo Therapeutics"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Termination","leadProduct":"Giroctocogene fitelparvovec","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sangamo Therapeutics","amount2":0.13,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"10","companyTruncated":"Sangamo Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Giroctocogene fitelparvovec","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Sangamo Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Giroctocogene fitelparvovec","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Sangamo Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Sangamo Therapeutics"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Sangamo Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Giroctocogene fitelparvovec","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Sangamo Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Sangamo Therapeutics"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Sangamo Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Giroctocogene fitelparvovec","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Sangamo Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Sangamo Therapeutics"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Lonoctocog Alfa","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Moroctocog Alfa","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Moroctocog Alfa","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"N8-GP","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Octocog Alfa","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Octocog Alfa","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"Pegylated Recombinant Factor Viii","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Baxalta Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Pegylated Recombinant Factor Viii","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Pegylated Recombinant Factor Viii","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Baxalta Pharmaceutical \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"SB-525","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Institute of Hematology & Blood Diseases Hospital, China","sponsor":"Sinocelltech Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"SCT800","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Institute of Hematology & Blood Diseases Hospital, China","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Institute of Hematology & Blood Diseases Hospital, China \/ Sinocelltech Ltd.","highestDevelopmentStatusID":"11","companyTruncated":"Institute of Hematology & Blood Diseases Hospital, China \/ Sinocelltech Ltd."},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Protein","year":"2018","type":"Inapplicable","leadProduct":"Turoctocog Alfa","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BAY2599023","moa":"Coagulation factor VIII (F8)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Bayer AG \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"OBI-1","moa":"Coagulation factor VIII (F8)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"OBI-1","moa":"Coagulation factor VIII (F8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"OBI-1","moa":"Coagulation factor VIII (F8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"SPK-8011","moa":"Coagulation factor VIII (F8)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spark Therapeutics, Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Spark Therapeutics, Inc \/ Undisclosed"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2016","type":"Inapplicable","leadProduct":"SPK-8011","moa":"Coagulation factor VIII (F8)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spark Therapeutics, Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Spark Therapeutics, Inc \/ Undisclosed"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"SPK-8011","moa":"Coagulation factor VIII (F8)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spark Therapeutics, Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Spark Therapeutics, Inc \/ Undisclosed"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SPK-8011","moa":"Coagulation factor VIII (F8)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spark Therapeutics, Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Spark Therapeutics, Inc \/ Undisclosed"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"SPK-8011","moa":"Coagulation factor VIII (F8)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spark Therapeutics, Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Spark Therapeutics, Inc \/ Undisclosed"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SPK-8011","moa":"Coagulation factor VIII (F8)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spark Therapeutics, Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Spark Therapeutics, Inc \/ Undisclosed"},{"orgOrder":0,"company":"Nirmish Shah","sponsor":"Bristol Myers Squibb","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"HPAPI","year":"2014","type":"Inapplicable","leadProduct":"Apixaban","moa":"Coagulation factor X","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Nirmish Shah","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nirmish Shah \/ Bristol Myers Squibb","highestDevelopmentStatusID":"10","companyTruncated":"Nirmish Shah \/ Bristol Myers Squibb"},{"orgOrder":0,"company":"Prime Therapeutics","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Agreement","leadProduct":"Antihemophilic Factor Recombinant","moa":"Coagulation factor X; Coagulation factor IX; von Willebrand factor; Phytanoyl-CoA dioxygenase, peroxisomal; Asialoglycoprotein receptor 2; Endoplasmic reticulum chaperone BiP; Calreticulin; Calnexin; Protein ERGIC-53; Prolow-density lipoprotein receptor-r||Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Prime Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Prime Therapeutics \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Prime Therapeutics \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Antihemophilic Factor, Recombinant","moa":"Coagulation factor X; Coagulation factor IX; von Willebrand factor; Phytanoyl-CoA dioxygenase, peroxisomal; Asialoglycoprotein receptor 2; Endoplasmic reticulum chaperone BiP; Calreticulin; Calnexin; Protein ERGIC-53; Prolow-density lipoprotein receptor-related protein 1; Multiple coagulation factor deficiency protein 2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Antihemophilic Factor, Recombinant","moa":"Coagulation factor X; Coagulation factor IX; von Willebrand factor; Phytanoyl-CoA dioxygenase, peroxisomal; Asialoglycoprotein receptor 2; Endoplasmic reticulum chaperone BiP; Calreticulin; Calnexin; Protein ERGIC-53; Prolow-density lipoprotein receptor-related protein 1; Multiple coagulation factor deficiency protein 2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Bayer AG","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Antihemophilic Factor, Recombinant","moa":"Coagulation factor X; Coagulation factor IX; von Willebrand factor; Phytanoyl-CoA dioxygenase, peroxisomal; Asialoglycoprotein receptor 2; Endoplasmic reticulum chaperone BiP; Calreticulin; Calnexin; Protein ERGIC-53; Prolow-density lipoprotein receptor-related protein 1; Multiple coagulation factor deficiency protein 2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Bayer AG","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Bayer AG"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Coagulation Factor IX","moa":"Coagulation factor Xa (F10)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Unity Health Toronto","sponsor":"The Ottawa Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Thrombin","moa":"Coagulation factor XI; Coagulation factor XIII A chain; Coagulation factor XIII B chain; Fibrinogen alpha chain; Fibrinogen beta chain; Fibrinogen gamma chain; Coagulation factor V; Coagulation factor VIII; Proteinase-activated receptor 1; Proteinase-activated receptor 4","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Unity Health Toronto","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"Unity Health Toronto \/ The Ottawa Hospital","highestDevelopmentStatusID":"1","companyTruncated":"Unity Health Toronto \/ The Ottawa Hospital"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"CSL312","moa":"Coagulation factor XII (F12)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"CSL312","moa":"Coagulation factor XII (F12)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"CSL312","moa":"Coagulation factor XII (F12)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Annexon Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"ANX005","moa":"Complement C1s component (C1S)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Annexon Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Annexon Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Annexon Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Annexon Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"ANX005","moa":"Complement C1s component (C1S)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Annexon Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Annexon Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Annexon Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Annexon Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"ANX005","moa":"Complement C1s component (C1S)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Annexon Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Annexon Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Annexon Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Crovalimab","moa":"Complement C5","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Crovalimab","moa":"Complement C5","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Pozelimab","moa":"Complement C5","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Pozelimab","moa":"Complement C5","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"ALXN1820","moa":"Complement factor P (CFP)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"FibroGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2015","type":"Inapplicable","leadProduct":"Pamrevlumab","moa":"Connective tissue growth factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"FibroGen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"FibroGen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"FibroGen \/ Undisclosed"},{"orgOrder":0,"company":"FibroGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Deflazacort","moa":"||Connective tissue growth factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"FibroGen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"FibroGen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"FibroGen \/ Undisclosed"},{"orgOrder":0,"company":"FibroGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Deflazacort","moa":"||Connective tissue growth factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"FibroGen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"FibroGen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"FibroGen \/ Undisclosed"},{"orgOrder":0,"company":"FibroGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Deflazacort","moa":"||Connective tissue growth factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"FibroGen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"FibroGen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"FibroGen \/ Undisclosed"},{"orgOrder":0,"company":"FibroGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Deflazacort","moa":"||Connective tissue growth factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"FibroGen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"FibroGen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"FibroGen \/ Undisclosed"},{"orgOrder":0,"company":"FibroGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Deflazacort","moa":"||Connective tissue growth factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"FibroGen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"FibroGen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"FibroGen \/ Undisclosed"},{"orgOrder":0,"company":"FibroGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Pamrevlumab","moa":"Connective tissue growth factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"FibroGen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"FibroGen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"FibroGen \/ Undisclosed"},{"orgOrder":0,"company":"FibroGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Pamrevlumab","moa":"Connective tissue growth factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"FibroGen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"FibroGen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"FibroGen \/ Undisclosed"},{"orgOrder":0,"company":"FibroGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Pamrevlumab","moa":"Connective tissue growth factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"FibroGen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"FibroGen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"FibroGen \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"Monopar Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Public Offering","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Monopar Therapeutics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Tablet","sponsorNew":"Monopar Therapeutics \/ RA Capital Management","highestDevelopmentStatusID":"10","companyTruncated":"Monopar Therapeutics \/ RA Capital Management"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Granules India Limited","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"Penicillamine","moa":"Copper","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Granules India Limited","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Granules India Limited \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Granules India Limited \/ Undisclosed"},{"orgOrder":0,"company":"Dr. Reddy's Laboratories","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"Penicillamine","moa":"Copper","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Dr. Reddy\\'s Laboratories","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Dr. Reddy\\'s Laboratories \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Dr. Reddy\\'s Laboratories \/ Undisclosed"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"Penicillamine","moa":"Copper","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Lupin Ltd \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Lupin Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Amino Acid","year":"2021","type":"Inapplicable","leadProduct":"Penicillamine","moa":"Copper","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Lupin Ltd \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Lupin Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Orphalan","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Amino Acid","year":"2018","type":"Inapplicable","leadProduct":"Penicillamine","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphalan","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orphalan \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Orphalan \/ Undisclosed"},{"orgOrder":0,"company":"Breckenridge Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2022","type":"Inapplicable","leadProduct":"Penicillamine","moa":"Copper","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Breckenridge Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Breckenridge Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Breckenridge Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Aggrega Pharma","sponsor":"Breckenridge Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2022","type":"Inapplicable","leadProduct":"Penicillamine","moa":"Copper","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Aggrega Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Aggrega Pharma \/ Breckenridge Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Aggrega Pharma \/ Breckenridge Pharmaceutical"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Tiomolibdate Choline","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Yale University","sponsor":"Bausch Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Trientine Hydrochloride","moa":"Copper","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Yale University","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Yale University \/ Bausch Health","highestDevelopmentStatusID":"1","companyTruncated":"Yale University \/ Bausch Health"},{"orgOrder":0,"company":"Univar Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Trientine Hydrochloride","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Univar Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Univar Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Univar Inc \/ Undisclosed"},{"orgOrder":0,"company":"Univar Inc","sponsor":"Aptiv Solutions","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Trientine Hydrochloride","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Univar Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Univar Inc \/ Aptiv Solutions","highestDevelopmentStatusID":"11","companyTruncated":"Univar Inc \/ Aptiv Solutions"},{"orgOrder":0,"company":"Orphalan","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Trientine Hydrochloride","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphalan","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Orphalan \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Orphalan \/ Undisclosed"},{"orgOrder":0,"company":"Orphalan","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Trientine Hydrochloride","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphalan","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orphalan \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Orphalan \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"UX701","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"UX701","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"UX701","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"UX701","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"UX701","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"UX701","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"VTX-801","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vivet Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vivet Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"VTX-801","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vivet Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"7","companyTruncated":"Vivet Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"VTX-801","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vivet Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Vivet Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Agreement","leadProduct":"VTX-801","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Vivet Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Pfizer Inc \/ Vivet Therapeutics"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"NBI-77860","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Ipsen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"BN82451B","moa":"COX-1\/COX-2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ipsen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Ipsen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ipsen \/ Undisclosed"},{"orgOrder":0,"company":"Bristol Myers Squibb","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"BMS-986470","moa":"CRBN\/ZBTB7A\/WIZ","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bristol Myers Squibb","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bristol Myers Squibb \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bristol Myers Squibb \/ Undisclosed"},{"orgOrder":0,"company":"HBM Alpha Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Licensing Agreement","leadProduct":"HAT001","moa":"CRH","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"HBM Alpha Therapeutics","amount2":0.40000000000000002,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.40000000000000002,"dosageForm":"Undisclosed","sponsorNew":"HBM Alpha Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"HBM Alpha Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"5Am Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Private Placement","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ 5Am Ventures","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ 5Am Ventures"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Kaken Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Partnership","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Kaken Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Kaken Pharmaceuticals"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Omega Funds","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Series B Financing","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Omega Funds","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Omega Funds"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tildacerfont","moa":"CRHR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Spruce Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Kamau Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Agreement","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Kamau Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Kamau Therapeutics"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Boston Medical Center","sponsor":"Celgene Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cytotoxic Drug","year":"2012","type":"Inapplicable","leadProduct":"Pomalidomide","moa":"CRL4(CRBN) E3 ubiquitin ligase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Boston Medical Center","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boston Medical Center \/ Celgene Corporation","highestDevelopmentStatusID":"7","companyTruncated":"Boston Medical Center \/ Celgene Corporation"},{"orgOrder":0,"company":"Bristol Myers Squibb","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cytotoxic Drug","year":"2012","type":"Inapplicable","leadProduct":"Pomalidomide","moa":"CRL4(CRBN) E3 ubiquitin ligase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Bristol Myers Squibb","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bristol Myers Squibb \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Bristol Myers Squibb \/ Undisclosed"},{"orgOrder":0,"company":"Seoul National University Hospital","sponsor":"CW Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Thalidomide","moa":"CRL4(CRBN) E3 ubiquitin ligase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Seoul National University Hospital","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Seoul National University Hospital \/ CW Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Seoul National University Hospital \/ CW Pharmaceutical"},{"orgOrder":0,"company":"Xenetic Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"SHP656","moa":"Cryptochrome circadian clock 1 (CRY1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Xenetic Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xenetic Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Xenetic Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Innovent Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"IBI310","moa":"CTLA-4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Innovent Biologics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Innovent Biologics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Innovent Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Gabrail Cancer Center Research","sponsor":"Emmaus Life Sciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Amino Acid","year":"2010","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase 1; Amidophosphoribosyltransferase; Glutamine synthetase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Gabrail Cancer Center Research","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Gabrail Cancer Center Research \/ Emmaus Life Sciences","highestDevelopmentStatusID":"8","companyTruncated":"Gabrail Cancer Center Research \/ Emmaus Life Sciences"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2010","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2022","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2022","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2022","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Asembia","sponsor":"Emmaus Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2021","type":"Agreement","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Asembia","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Asembia \/ Emmaus Life Sciences","highestDevelopmentStatusID":"15","companyTruncated":"Asembia \/ Emmaus Life Sciences"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2023","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2022","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2022","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2022","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2021","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Emmaus Life Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Emmaus Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Emmaus Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"ANI Pharmaceuticals Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2024","type":"Inapplicable","leadProduct":"L-Glutamine","moa":"CTP synthase-1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ANI Pharmaceuticals Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ANI Pharmaceuticals Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"ANI Pharmaceuticals Inc \/ Undisclosed"},{"orgOrder":0,"company":"Aristea Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"RIST4721","moa":"C-X-C chemokine receptor type 2 (CXCR2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aristea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Aristea Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aristea Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"St. Jude Children","sponsor":"BioLineRx","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Motixafortide","moa":"C-X-C chemokine receptor type 4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"St. Jude Children","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"St. Jude Children \/ BioLineRx","highestDevelopmentStatusID":"6","companyTruncated":"St. Jude Children \/ BioLineRx"},{"orgOrder":0,"company":"BioLineRx","sponsor":"Washington University School of Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Peptide, Unconjugated","year":"2023","type":"Collaboration","leadProduct":"Motixafortide","moa":"||C-X-C chemokine receptor type 4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"BioLineRx","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioLineRx \/ Washington University School of Medicine","highestDevelopmentStatusID":"6","companyTruncated":"BioLineRx \/ Washington University School of Medicine"},{"orgOrder":0,"company":"BioLineRx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Motixafortide","moa":"||C-X-C chemokine receptor type 4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"BioLineRx","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioLineRx \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"BioLineRx \/ Undisclosed"},{"orgOrder":0,"company":"BioLineRx","sponsor":"St. Jude Children\u2019s Research Hospital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Peptide, Unconjugated","year":"2024","type":"Agreement","leadProduct":"Motixafortide","moa":"C-X-C chemokine receptor type 4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"BioLineRx","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioLineRx \/ St. Jude Children\u2019s Research Hospital","highestDevelopmentStatusID":"6","companyTruncated":"BioLineRx \/ St. Jude Children\u2019s Research Hospital"},{"orgOrder":0,"company":"Washington University School of Medicine","sponsor":"BioLineRx | Biogen","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Motixafortide","moa":"C-X-C chemokine receptor type 4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Washington University School of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Washington University School of Medicine \/ BioLineRx | Biogen","highestDevelopmentStatusID":"6","companyTruncated":"Washington University School of Medicine \/ BioLineRx | Biogen"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Plerixafor","moa":"C-X-C chemokine receptor type 4","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Memorial Sloan Kettering Cancer Center","sponsor":"Sanofi | New York Blood Center | Weill Medical College of Cornell University | Duke University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Plerixafor","moa":"C-X-C chemokine receptor type 4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Memorial Sloan Kettering Cancer Center","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Memorial Sloan Kettering Cancer Center \/ Sanofi | New York Blood Center | Weill Medical College of Cornell University | Duke University","highestDevelopmentStatusID":"6","companyTruncated":"Memorial Sloan Kettering Cancer Center \/ Sanofi | New York Blood Center | Weill Medical College of Cornell University | Duke University"},{"orgOrder":0,"company":"Ensoma","sponsor":"Bluebird Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"MGTA-145","moa":"CXCR-2 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ensoma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ensoma \/ Bluebird Bio","highestDevelopmentStatusID":"8","companyTruncated":"Ensoma \/ Bluebird Bio"},{"orgOrder":0,"company":"Magenta Therapeutics","sponsor":"Bluebird Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"MGTA-145","moa":"||CXCR-2 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Magenta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Magenta Therapeutics \/ Bluebird Bio","highestDevelopmentStatusID":"8","companyTruncated":"Magenta Therapeutics \/ Bluebird Bio"},{"orgOrder":0,"company":"Magenta Therapeutics","sponsor":"Bluebird Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Collaboration","leadProduct":"MGTA-145","moa":"||CXCR-2 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Magenta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Magenta Therapeutics \/ Bluebird Bio","highestDevelopmentStatusID":"8","companyTruncated":"Magenta Therapeutics \/ Bluebird Bio"},{"orgOrder":0,"company":"Magenta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"MGTA-145","moa":"||CXCR-2 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Magenta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Magenta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Magenta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Magenta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"MGTA-145","moa":"||CXCR-2 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Magenta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Magenta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Magenta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Magenta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"MGTA-456","moa":"CXCR-2 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Magenta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Magenta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Magenta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ensoma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EN-374","moa":"CYBB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ensoma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ensoma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ensoma \/ Undisclosed"},{"orgOrder":0,"company":"Ensoma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EN-374","moa":"CYBB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ensoma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ensoma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ensoma \/ Undisclosed"},{"orgOrder":0,"company":"Ensoma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EN-374","moa":"CYBB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ensoma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ensoma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ensoma \/ Undisclosed"},{"orgOrder":0,"company":"University Hospital, Brest","sponsor":"ManRos Therapeutics | Cyclacel Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Seliciclib","moa":"Cyclin-dependent kinase 1 | Cyclin-dependent kinase 2 | Cyclin-dependent kinase 7 | Cyclin-dependent kinase 5 | Cyclin-dependent kinase 9","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University Hospital, Brest","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University Hospital, Brest \/ ManRos Therapeutics | Cyclacel Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"University Hospital, Brest \/ ManRos Therapeutics | Cyclacel Pharmaceuticals"},{"orgOrder":0,"company":"SRW Laboratories","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NEW ZEALAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Fisetin","moa":"Cyclin-dependent kinase 6; Fatty acid synthase; Cyclin homolog","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SRW Laboratories","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"SRW Laboratories \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SRW Laboratories \/ Undisclosed"},{"orgOrder":0,"company":"Instituto Nacional de Investigacao em Saude, Angola","sponsor":"Fundaci\u00f3n Bancaria Caixa D'estalvis I Pensions De Barcelona | Funda\u00e7ao Calouste Gulbenkian | CLINCOORD SERVICES, INC. | CLINCOORD PRESTA\u00c7\u00c3O DE SEVI\u00c7OS, LDA ANGOLA | Escola Superior de Tecnologia da Sa\u00fade de Lisboa | ClinCoord Research Center at Luanda Med","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ANGOLA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Aspirin","moa":"Cyclooxygenase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Instituto Nacional de Investigacao em Saude, Angola","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Instituto Nacional de Investigacao em Saude, Angola \/ Fundaci\u00f3n Bancaria Caixa D'estalvis I Pensions De Barcelona | Funda\u00e7ao Calouste Gulbenkian | CLINCOORD SERVICES, INC. | CLINCOORD PRESTA\u00c7\u00c3O DE SEVI\u00c7OS, LDA ANGOLA | Escola Superior de Tecnologia da Sa\u00fade de Lisboa | ClinCoord Research Center at Luanda Med","highestDevelopmentStatusID":"7","companyTruncated":"Instituto Nacional de Investigacao em Saude, Angola \/ Fundaci\u00f3n Bancaria Caixa D'estalvis I Pensions De Barcelona | Funda\u00e7ao Calouste Gulbenkian | CLINCOORD SERVICES, INC. | CLINCOORD PRESTA\u00c7\u00c3O DE SEVI\u00c7OS, LDA ANGOLA | Escola Superior de Tecnologia da Sa\u00fade de Lisboa | ClinCoord Research Center at Luanda Med"},{"orgOrder":0,"company":"Columbia University","sponsor":"Alpha-1 Foundation | Stony Wold-Herbert Fund, Inc.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Aspirin","moa":"Cyclooxygenase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Columbia University","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Columbia University \/ Alpha-1 Foundation | Stony Wold-Herbert Fund, Inc.","highestDevelopmentStatusID":"8","companyTruncated":"Columbia University \/ Alpha-1 Foundation | Stony Wold-Herbert Fund, Inc."},{"orgOrder":0,"company":"Healx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sulindac","moa":"Cyclooxygenase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Healx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Healx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Healx \/ Undisclosed"},{"orgOrder":0,"company":"Millendo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"Nevanimibe HCl","moa":"CYP11B1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Millendo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Millendo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Millendo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Highbridge Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Financing","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Tablet","sponsorNew":"Santhera Pharmaceuticals \/ Highbridge Capital Management","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Highbridge Capital Management"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"BV-101","moa":"CYP46A1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AskBio \/ Bayer AG","highestDevelopmentStatusID":"4","companyTruncated":"AskBio \/ Bayer AG"},{"orgOrder":0,"company":"BrainVectis","sponsor":"AskBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Acquisition","leadProduct":"BV-CYP01","moa":"CYP46A1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"BrainVectis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BrainVectis \/ AskBio","highestDevelopmentStatusID":"4","companyTruncated":"BrainVectis \/ AskBio"},{"orgOrder":0,"company":"Vanderbilt University Medical Center","sponsor":"Medical College of Wisconsin | Versiti","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Montelukast Sodium","moa":"Cysteinyl leukotriene receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vanderbilt University Medical Center","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vanderbilt University Medical Center \/ Medical College of Wisconsin | Versiti","highestDevelopmentStatusID":"8","companyTruncated":"Vanderbilt University Medical Center \/ Medical College of Wisconsin | Versiti"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Australian Pharmaceutical Benefits Scheme","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Agreement","leadProduct":"Elexacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Australian Pharmaceutical Benefits Scheme","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Australian Pharmaceutical Benefits Scheme"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Galapagos","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Galicaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Galapagos","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Galapagos \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Galapagos \/ Undisclosed"},{"orgOrder":0,"company":"Galapagos","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Galicaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Galapagos","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Galapagos \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Galapagos \/ Undisclosed"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Galicaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"AbbVie Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AbbVie Inc \/ Undisclosed"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Sionna Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Licensing Agreement","leadProduct":"Galicaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"AbbVie Inc \/ Sionna Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"AbbVie Inc \/ Sionna Therapeutics"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Lupin Ltd \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Lupin Ltd \/ Undisclosed"},{"orgOrder":0,"company":"NHS England","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Agreement","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"NHS England","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"NHS England \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"11","companyTruncated":"NHS England \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Medical University of South Carolina","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medical University of South Carolina","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Medical University of South Carolina \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Medical University of South Carolina \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Aifa","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Agreement","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Aifa","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Aifa"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"French Health Authorities For The Cystic Fibrosis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Agreement","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ French Health Authorities For The Cystic Fibrosis","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ French Health Authorities For The Cystic Fibrosis"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Spanish Government","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Agreement","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Spanish Government","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Spanish Government"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Cystic Fibrosis Foundation","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Acquisition","leadProduct":"Ivacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Cystic Fibrosis Foundation","amount2":0.65000000000000002,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.65000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Cystic Fibrosis Foundation \/ Royalty Pharma","highestDevelopmentStatusID":"15","companyTruncated":"Cystic Fibrosis Foundation \/ Royalty Pharma"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Fsio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Agreement","leadProduct":"Lumacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Fsio","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Fsio"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Galapagos","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Navocaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Galapagos","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Galapagos \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Galapagos \/ Undisclosed"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Navocaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AbbVie Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AbbVie Inc \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"QBW251","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Nottingham University Hospitals NHS Trust","sponsor":"Vertex Pharmaceuticals | Cystic Fibrosis Foundation | Cystic Fibrosis Trust | University of Nottingham | Nottingham University Hospitals Charity","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nottingham University Hospitals NHS Trust","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nottingham University Hospitals NHS Trust \/ Vertex Pharmaceuticals | Cystic Fibrosis Foundation | Cystic Fibrosis Trust | University of Nottingham | Nottingham University Hospitals Charity","highestDevelopmentStatusID":"8","companyTruncated":"Nottingham University Hospitals NHS Trust \/ Vertex Pharmaceuticals | Cystic Fibrosis Foundation | Cystic Fibrosis Trust | University of Nottingham | Nottingham University Hospitals Charity"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Tezacaftor","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"VX-440","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"VX-659","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"VX-659","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"VX-659","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"VX-659","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"VX-659","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"VX-659","moa":"Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Proteostasis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Dirocaftor","moa":"cystic fibrosis transmembrane regulator (CFTR) protein modulators","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Proteostasis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proteostasis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Proteostasis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Proteostasis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Dirocaftor","moa":"cystic fibrosis transmembrane regulator (CFTR) protein modulators","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Proteostasis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proteostasis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Proteostasis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Proteostasis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Dirocaftor","moa":"||cystic fibrosis transmembrane regulator (CFTR) protein modulators","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Proteostasis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proteostasis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Proteostasis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Proteostasis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Dirocaftor","moa":"||cystic fibrosis transmembrane regulator (CFTR) protein modulators","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Proteostasis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proteostasis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Proteostasis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Proteostasis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Nesolicaftor","moa":"cystic fibrosis transmembrane regulator (CFTR) protein modulators","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Proteostasis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proteostasis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Proteostasis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Proteostasis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Nesolicaftor","moa":"cystic fibrosis transmembrane regulator (CFTR) protein modulators","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Proteostasis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proteostasis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Proteostasis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Proteostasis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Nesolicaftor","moa":"cystic fibrosis transmembrane regulator (CFTR) protein modulators","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Proteostasis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proteostasis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Proteostasis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Proteostasis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Posenacaftor","moa":"cystic fibrosis transmembrane regulator (CFTR) protein modulators","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Proteostasis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proteostasis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Proteostasis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"NovaBiotics Ltd","sponsor":"Agility Clinical | PSR Group B.V.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Amino Acid","year":"2016","type":"Inapplicable","leadProduct":"Cysteamine Bitartrate","moa":"Cystine","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NovaBiotics Ltd","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"NovaBiotics Ltd \/ Agility Clinical | PSR Group B.V.","highestDevelopmentStatusID":"8","companyTruncated":"NovaBiotics Ltd \/ Agility Clinical | PSR Group B.V."},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"Cysteamine Bitartrate","moa":"Cystine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Delayed Release","sponsorNew":"Horizon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Horizon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"Cysteamine Bitartrate","moa":"Cystine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Delayed Release","sponsorNew":"Horizon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Horizon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"University of California, San Diego","sponsor":"Amgen Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2011","type":"Inapplicable","leadProduct":"Cysteamine Bitartrate","moa":"Cystine","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of California, San Diego","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of California, San Diego \/ Amgen Inc","highestDevelopmentStatusID":"1","companyTruncated":"University of California, San Diego \/ Amgen Inc"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2010","type":"Inapplicable","leadProduct":"Cysteamine Bitartrate","moa":"Cystine","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Recordati Rare Diseases","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Cysteamine Bitartrate","moa":"Cystine","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Recordati Rare Diseases","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Recordati Rare Diseases \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Recordati Rare Diseases \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"RP103","moa":"Cystine reducer","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"RP103","moa":"Cystine reducer","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-04","moa":"Cystinosin (CTNS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-04","moa":"Cystinosin (CTNS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AVR-RD-04","moa":"Cystinosin (CTNS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AVR-RD-04","moa":"Cystinosin (CTNS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-04","moa":"Cystinosin (CTNS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"University of California","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AVR-RD-04","moa":"Cystinosin (CTNS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ University of California","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ University of California"},{"orgOrder":0,"company":"Johnson & Johnson","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Abiraterone Acetate","moa":"Cytochrome P450 17A1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Johnson & Johnson","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Johnson & Johnson \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Johnson & Johnson \/ Undisclosed"},{"orgOrder":0,"company":"Bambino Ges\u00f9 Hospital and Research Institute","sponsor":"University of Exeter | Radboud University Medical Center | Consorzio per Valutazioni Biologiche e Farmacologiche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Posaconazole","moa":"Cytochrome P450 51","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Bambino Ges\u00f9 Hospital and Research Institute","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bambino Ges\u00f9 Hospital and Research Institute \/ University of Exeter | Radboud University Medical Center | Consorzio per Valutazioni Biologiche e Farmacologiche","highestDevelopmentStatusID":"9","companyTruncated":"Bambino Ges\u00f9 Hospital and Research Institute \/ University of Exeter | Radboud University Medical Center | Consorzio per Valutazioni Biologiche e Farmacologiche"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Luvadaxistat","moa":"DAAO||enzyme inhibitors","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Neurocrine Biosciences \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Neurocrine Biosciences \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Eladocagene Exuparvovec","moa":"DDC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Eladocagene Exuparvovec","moa":"DDC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"ClearPoint Neuro","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Eladocagene Exuparvovec","moa":"DDC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PTC Therapeutics \/ ClearPoint Neuro","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ ClearPoint Neuro"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Divestment","leadProduct":"Eladocagene Exuparvovec","moa":"DDC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0.14999999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14999999999999999,"dosageForm":"Infusion","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Eladocagene Exuparvovec","moa":"DDC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Masonic Cancer Center, University of Minnesota","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2014","type":"Inapplicable","leadProduct":"Galsulfase","moa":"Dermatan sulfate","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Masonic Cancer Center, University of Minnesota","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Masonic Cancer Center, University of Minnesota \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Masonic Cancer Center, University of Minnesota \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"JR-171","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Lepunafusp Alpha","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Lepunafusp Alpha","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Lepunafusp Alpha","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Lepunafusp Alpha","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"SATT Conectus","sponsor":"Lysogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"FXS01","moa":"DGKk","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SATT Conectus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SATT Conectus \/ Lysogene","highestDevelopmentStatusID":"4","companyTruncated":"SATT Conectus \/ Lysogene"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FXS01","moa":"DGKk","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lobe Sciences","sponsor":"Pentec Health","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Dietary Supplement","year":"2023","type":"Agreement","leadProduct":"DHA","moa":"DHA level","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Lobe Sciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Lobe Sciences \/ Pentec Health","highestDevelopmentStatusID":"15","companyTruncated":"Lobe Sciences \/ Pentec Health"},{"orgOrder":0,"company":"Kiora Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"KIO-301","moa":"DHODH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Kiora Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Kiora Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Kiora Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Kiora Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"KIO-301","moa":"DHODH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Kiora Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Kiora Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Kiora Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Accelagen","sponsor":"Kiora Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"KIO-301","moa":"DHODH","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Accelagen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Accelagen \/ Kiora Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Accelagen \/ Kiora Pharmaceuticals"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Pradigastat","moa":"Diacylglycerol acyltransferase 1 (DGAT1)||Diacylglycerol O-acyltransferase 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Pradigastat","moa":"Diacylglycerol acyltransferase 1 (DGAT1)||Diacylglycerol O-acyltransferase 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"First Wave BioPharma","sponsor":"H.C. Wainwright & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Public Offering","leadProduct":"Yeast Recombinant Lipase","moa":"Dietary triglyceride ester bond hydrolysis","graph1":"Genetic Disease","graph2":"Phase II","graph3":"First Wave BioPharma","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Capsule","sponsorNew":"First Wave BioPharma \/ H.C. Wainwright & Co","highestDevelopmentStatusID":"8","companyTruncated":"First Wave BioPharma \/ H.C. Wainwright & Co"},{"orgOrder":0,"company":"First Wave BioPharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Porcine PERT","moa":"||Dietary triglyceride ester bond hydrolysis","graph1":"Genetic Disease","graph2":"Phase II","graph3":"First Wave BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"First Wave BioPharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"First Wave BioPharma \/ Undisclosed"},{"orgOrder":0,"company":"First Wave BioPharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Yeast Recombinant Lipase","moa":"Dietary triglyceride ester bond hydrolysis","graph1":"Genetic Disease","graph2":"Phase II","graph3":"First Wave BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"First Wave BioPharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"First Wave BioPharma \/ Undisclosed"},{"orgOrder":0,"company":"First Wave BioPharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Yeast Recombinant Lipase","moa":"Dietary triglyceride ester bond hydrolysis","graph1":"Genetic Disease","graph2":"Phase II","graph3":"First Wave BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"First Wave BioPharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"First Wave BioPharma \/ Undisclosed"},{"orgOrder":0,"company":"First Wave BioPharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Yeast Recombinant Lipase","moa":"Dietary triglyceride ester bond hydrolysis","graph1":"Genetic Disease","graph2":"Phase II","graph3":"First Wave BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"First Wave BioPharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"First Wave BioPharma \/ Undisclosed"},{"orgOrder":0,"company":"First Wave BioPharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Yeast Recombinant Lipase","moa":"Dietary triglyceride ester bond hydrolysis","graph1":"Genetic Disease","graph2":"Phase II","graph3":"First Wave BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"First Wave BioPharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"First Wave BioPharma \/ Undisclosed"},{"orgOrder":0,"company":"First Wave BioPharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Yeast Recombinant Lipase","moa":"Dietary triglyceride ester bond hydrolysis","graph1":"Genetic Disease","graph2":"Phase II","graph3":"First Wave BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"First Wave BioPharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"First Wave BioPharma \/ Undisclosed"},{"orgOrder":0,"company":"Aldeyra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cytotoxic Drug","year":"2022","type":"Inapplicable","leadProduct":"Methotrexate","moa":"Dihydrofolate reductase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aldeyra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aldeyra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aldeyra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aldeyra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cytotoxic Drug","year":"2022","type":"Inapplicable","leadProduct":"Methotrexate","moa":"Dihydrofolate reductase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aldeyra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aldeyra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aldeyra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aldeyra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cytotoxic Drug","year":"2021","type":"Inapplicable","leadProduct":"Methotrexate","moa":"Dihydrofolate reductase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aldeyra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aldeyra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aldeyra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aldeyra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cytotoxic Drug","year":"2023","type":"Inapplicable","leadProduct":"Methotrexate","moa":"Dihydrofolate reductase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aldeyra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aldeyra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aldeyra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aldeyra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cytotoxic Drug","year":"2023","type":"Inapplicable","leadProduct":"Methotrexate","moa":"Dihydrofolate reductase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aldeyra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aldeyra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aldeyra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cytotoxic Drug","year":"2023","type":"Inapplicable","leadProduct":"Methotrexate","moa":"Dihydrofolate reductase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Insmed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Brensocatib","moa":"Dipeptidyl peptidase I","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Insmed","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Insmed \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Insmed \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"AOC-1044","moa":"DMD","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"AOC-1044","moa":"DMD","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Brogidirsen","moa":"DMD","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Brogidirsen","moa":"DMD","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Brogidirsen","moa":"DMD","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Brogidirsen","moa":"DMD","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"PBGENE-DMD","moa":"DMD","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Precision BioSciences","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Precision BioSciences \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Precision BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2016","type":"Inapplicable","leadProduct":"BMN 044","moa":"DMD exon 44","graph1":"Genetic Disease","graph2":"Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Suzhou GenAssist Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"GEN6050","moa":"DMD exon 50","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Suzhou GenAssist Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Suzhou GenAssist Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Suzhou GenAssist Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"BMN 351","moa":"DMD exon 51","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2013","type":"Inapplicable","leadProduct":"BMN053","moa":"DMD gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"F. Hoffmann-La Roche \/ Sarepta Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Hansa Biopharma","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"||DMD*","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Hansa Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Hansa Biopharma \/ Sarepta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Hansa Biopharma \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Hansa Biopharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Hansa Biopharma","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Hansa Biopharma"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Divestment","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Catalent Pharma Solutions","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Agreement","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalent Pharma Solutions","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Catalent Pharma Solutions \/ Sarepta Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Catalent Pharma Solutions \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"AOC-1001","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"AOC-1001","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARO-DM1","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ARO-DM1","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARO-DM1","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Licensing Agreement","leadProduct":"ARO-DM1","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":11.380000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":11.380000000000001,"dosageForm":"Infusion","sponsorNew":"Arrowhead Pharmaceuticals \/ Sarepta Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0.33000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0.37,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0.23000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2021","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"NeuBase Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"NT0200","moa":"DMPK","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NeuBase Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"NeuBase Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"NeuBase Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vera Therapeutics","sponsor":"NeuBase Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Acquisition","leadProduct":"NT0200","moa":"DMPK","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vera Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Vera Therapeutics \/ NeuBase Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Vera Therapeutics \/ NeuBase Therapeutics"},{"orgOrder":0,"company":"NeuBase Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"NT0200","moa":"DMPK","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NeuBase Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"NeuBase Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"NeuBase Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Protalix","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"Alidornase alfa","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Protalix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Protalix \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Protalix \/ Undisclosed"},{"orgOrder":0,"company":"Protalix","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Alidornase alfa","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Protalix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Protalix \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Protalix \/ Undisclosed"},{"orgOrder":0,"company":"Columbia University","sponsor":"CEPHALON INC","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Bendamustine Hydrochloride","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Columbia University","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Columbia University \/ CEPHALON INC","highestDevelopmentStatusID":"8","companyTruncated":"Columbia University \/ CEPHALON INC"},{"orgOrder":0,"company":"Tufts Medical Center","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Bendamustine Hydrochloride","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tufts Medical Center","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tufts Medical Center \/ Sanofi","highestDevelopmentStatusID":"8","companyTruncated":"Tufts Medical Center \/ Sanofi"},{"orgOrder":0,"company":"Essen Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Busulfan","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Essen Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Essen Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Essen Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Generium","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Dornase Alfa","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nebulizer","sponsorNew":"Generium \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Generium \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2012","type":"Inapplicable","leadProduct":"Dornase Alfa","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nebulizer","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"Oncopeptides","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Melphalan Flufenamide","moa":"||DNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Oncopeptides","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Oncopeptides \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Oncopeptides \/ Undisclosed"},{"orgOrder":0,"company":"Oncopeptides","sponsor":"PRA Health Sciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Peptide, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Melphalan Flufenamide","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Oncopeptides","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oncopeptides \/ PRA Health Sciences","highestDevelopmentStatusID":"7","companyTruncated":"Oncopeptides \/ PRA Health Sciences"},{"orgOrder":0,"company":"Boston Medical Center","sponsor":"Spectrum Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cytotoxic Drug","year":"2016","type":"Inapplicable","leadProduct":"Melphalan Hydrochloride","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Boston Medical Center","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boston Medical Center \/ Spectrum Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Boston Medical Center \/ Spectrum Pharmaceuticals"},{"orgOrder":0,"company":"medac","sponsor":"Celerion | Venn Life Sciences | Syneos Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cytotoxic Drug","year":"2015","type":"Inapplicable","leadProduct":"Treosulfan","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"medac","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"medac \/ Celerion | Venn Life Sciences | Syneos Health","highestDevelopmentStatusID":"8","companyTruncated":"medac \/ Celerion | Venn Life Sciences | Syneos Health"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Cytotoxic Drug","year":"2022","type":"Inapplicable","leadProduct":"Decitabine","moa":"DNA (cytosine-5)-methyltransferase 1 | DNA | DNA (cytosine-5)-methyltransferase 3A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"National Guard Health Affairs","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SAUDI ARABIA","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Fludarabine Phosphate","moa":"DNA polymerase (alpha\/delta\/epsilon) | Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"National Guard Health Affairs","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Guard Health Affairs \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"National Guard Health Affairs \/ Undisclosed"},{"orgOrder":0,"company":"Amplo Biotechnology","sponsor":"Andelyn Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Partnership","leadProduct":"AMP-101","moa":"DOK7","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Amplo Biotechnology","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amplo Biotechnology \/ Andelyn Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"Amplo Biotechnology \/ Andelyn Biosciences"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Carbidopa","moa":"DOPA decarboxylase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NYU Langone Health \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"NYU Langone Health \/ Undisclosed"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Carbidopa","moa":"DOPA decarboxylase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NYU Langone Health \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"NYU Langone Health \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia","sponsor":"European Huntington's Disease Network | Huntington Study Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia \/ European Huntington's Disease Network | Huntington Study Group","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia \/ European Huntington's Disease Network | Huntington Study Group"},{"orgOrder":0,"company":"Prilenia","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Morningside Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2020","type":"Series A Financing","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Morningside Ventures","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Morningside Ventures"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Sands Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2021","type":"Series B Financing","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Sands Capital","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Sands Capital"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Ferrer Internacional","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2025","type":"Licensing Agreement","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0.56999999999999995,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.56999999999999995,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Grupo Ferrer Internacional","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Grupo Ferrer Internacional"},{"orgOrder":0,"company":"Huntington Study Group","sponsor":"Prilenia therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Partnership","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Huntington Study Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Huntington Study Group \/ Prilenia therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Huntington Study Group \/ Prilenia therapeutics"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"BI1291583","moa":"DPP-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"University of Alabama at Birmingham","sponsor":"Pfizer Inc | Pacific Pediatric Neuro-Oncology Consortium","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Binimetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 1 | Dual specificity mitogen-activated protein kinase kinase 2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of Alabama at Birmingham","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Alabama at Birmingham \/ Pfizer Inc | Pacific Pediatric Neuro-Oncology Consortium","highestDevelopmentStatusID":"8","companyTruncated":"University of Alabama at Birmingham \/ Pfizer Inc | Pacific Pediatric Neuro-Oncology Consortium"},{"orgOrder":0,"company":"Johns Hopkins University","sponsor":"Neurofibromatosis Therapeutic Acceleration Program | SpringWorks Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Mirdametinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Johns Hopkins University","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johns Hopkins University \/ Neurofibromatosis Therapeutic Acceleration Program | SpringWorks Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Johns Hopkins University \/ Neurofibromatosis Therapeutic Acceleration Program | SpringWorks Therapeutics"},{"orgOrder":0,"company":"SpringWorks Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Mirdametinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SpringWorks Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SpringWorks Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SpringWorks Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Great Ormond Street Hospital for Children NHS Foundation Trust","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Selumetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Great Ormond Street Hospital for Children NHS Foundation Trust","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Great Ormond Street Hospital for Children NHS Foundation Trust \/ AstraZeneca","highestDevelopmentStatusID":"7","companyTruncated":"Great Ormond Street Hospital for Children NHS Foundation Trust \/ AstraZeneca"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Merck & Co","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Selumetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"AstraZeneca \/ Merck & Co","highestDevelopmentStatusID":"15","companyTruncated":"AstraZeneca \/ Merck & Co"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Merck & Co","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Selumetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"AstraZeneca \/ Merck & Co","highestDevelopmentStatusID":"15","companyTruncated":"AstraZeneca \/ Merck & Co"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Merck & Co","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Selumetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"AstraZeneca \/ Merck & Co","highestDevelopmentStatusID":"15","companyTruncated":"AstraZeneca \/ Merck & Co"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Selumetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Selumetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Selumetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Selumetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Merck & Co","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Selumetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AstraZeneca \/ Merck & Co","highestDevelopmentStatusID":"15","companyTruncated":"AstraZeneca \/ Merck & Co"},{"orgOrder":0,"company":"Children's Hospital Medical Center, Cincinnati","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Selumetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Children's Hospital Medical Center, Cincinnati","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Children's Hospital Medical Center, Cincinnati \/ AstraZeneca","highestDevelopmentStatusID":"8","companyTruncated":"Children's Hospital Medical Center, Cincinnati \/ AstraZeneca"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Merck & Co","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Selumetinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alexion Pharmaceuticals \/ Merck & Co","highestDevelopmentStatusID":"15","companyTruncated":"Alexion Pharmaceuticals \/ Merck & Co"},{"orgOrder":0,"company":"Armatus Bio","sponsor":"SOLVE FSHD","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Financing","leadProduct":"ARM-201","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Armatus Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Armatus Bio \/ SOLVE FSHD","highestDevelopmentStatusID":"4","companyTruncated":"Armatus Bio \/ SOLVE FSHD"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Licensing Agreement","leadProduct":"ARO-DUX4","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":11.380000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":11.380000000000001,"dosageForm":"Infusion","sponsorNew":"Arrowhead Pharmaceuticals \/ Sarepta Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARO-DUX4","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Licensing Agreement","leadProduct":"ARO-DUX4","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":11.380000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":11.380000000000001,"dosageForm":"Undisclosed","sponsorNew":"Arrowhead Pharmaceuticals \/ Sarepta Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARO-DUX4","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Epicrispr Biotechnologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Epicrispr Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Epicrispr Biotechnologies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Epicrispr Biotechnologies \/ Undisclosed"},{"orgOrder":0,"company":"Epicrispr Biotechnologies","sponsor":"Springbok Analytics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Partnership","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Epicrispr Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Epicrispr Biotechnologies \/ Springbok Analytics","highestDevelopmentStatusID":"7","companyTruncated":"Epicrispr Biotechnologies \/ Springbok Analytics"},{"orgOrder":0,"company":"Epicrispr Biotechnologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Epicrispr Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Epicrispr Biotechnologies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Epicrispr Biotechnologies \/ Undisclosed"},{"orgOrder":0,"company":"Epic Bio","sponsor":"SOLVE FSHD","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Financing","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Epic Bio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Epic Bio \/ SOLVE FSHD","highestDevelopmentStatusID":"5","companyTruncated":"Epic Bio \/ SOLVE FSHD"},{"orgOrder":0,"company":"Epicrispr Biotechnologies","sponsor":"Ally Bridge Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Series B Financing","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Epicrispr Biotechnologies","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Injection","sponsorNew":"Epicrispr Biotechnologies \/ Ally Bridge Group","highestDevelopmentStatusID":"7","companyTruncated":"Epicrispr Biotechnologies \/ Ally Bridge Group"},{"orgOrder":0,"company":"Epic Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Epic Bio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Epic Bio \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Epic Bio \/ Undisclosed"},{"orgOrder":0,"company":"Epic Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Epic Bio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Epic Bio \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Epic Bio \/ Undisclosed"},{"orgOrder":0,"company":"miRecule","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Licensing Agreement","leadProduct":"MC-DX4","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"miRecule","amount2":0.42999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.42999999999999999,"dosageForm":"Undisclosed","sponsorNew":"miRecule \/ Sanofi","highestDevelopmentStatusID":"4","companyTruncated":"miRecule \/ Sanofi"},{"orgOrder":0,"company":"Modalis Therapeutics","sponsor":"SOLVE FSHD","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Collaboration","leadProduct":"MDL-103","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Modalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Modalis Therapeutics \/ SOLVE FSHD","highestDevelopmentStatusID":"2","companyTruncated":"Modalis Therapeutics \/ SOLVE FSHD"},{"orgOrder":0,"company":"Neuronascent","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"NNI-351","moa":"DYRK","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Neuronascent","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neuronascent \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Neuronascent \/ Undisclosed"},{"orgOrder":0,"company":"Perha Pharmaceuticals","sponsor":"France 2030 | European Innovation Council | Fondation J\u00e9r\u00f4me Lejeune","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Leucettinib-21","moa":"DYRK1A","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Perha Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Perha Pharmaceuticals \/ France 2030 | European Innovation Council | Fondation J\u00e9r\u00f4me Lejeune","highestDevelopmentStatusID":"6","companyTruncated":"Perha Pharmaceuticals \/ France 2030 | European Innovation Council | Fondation J\u00e9r\u00f4me Lejeune"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SRP-6004","moa":"Dysferlin (DYSF)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2021","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2021","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Public Offering","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0.17999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17999999999999999,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Goldman Sachs & Co","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Janus Henderson Investors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Public Offering","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Janus Henderson Investors","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Janus Henderson Investors"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ENTR-601-45","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-45","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-45","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-45","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Insmed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"INS1201","moa":"dystrophin","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Insmed","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Insmed \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Insmed \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PF-06939926","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"PF-06939926","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"PF-06939926","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Fordadistrogene Movaparvovec","moa":"Dystrophin (DMD)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Fordadistrogene Movaparvovec","moa":"Dystrophin (DMD)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Fordadistrogene Movaparvovec","moa":"Dystrophin (DMD)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Fordadistrogene Movaparvovec","moa":"Dystrophin (DMD)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Fordadistrogene Movaparvovec","moa":"Dystrophin (DMD)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Fordadistrogene Movaparvovec","moa":"Dystrophin (DMD)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Fordadistrogene Movaparvovec","moa":"Dystrophin (DMD)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Inapplicable","leadProduct":"Delpacibart Zotadirsen","moa":"Dystrophin exon-44","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"Delpacibart Zotadirsen","moa":"Dystrophin exon-44","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"Delpacibart Zotadirsen","moa":"Dystrophin exon-44","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Inapplicable","leadProduct":"Delpacibart Zotadirsen","moa":"Dystrophin exon-44","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"Delpacibart Zotadirsen","moa":"Dystrophin exon-44","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"Delpacibart Zotadirsen","moa":"Dystrophin exon-44","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Public Offering","leadProduct":"Delpacibart Zotadirsen","moa":"Dystrophin exon-44","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0.5,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.68999999999999995,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Leerink Partners"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Public Offering","leadProduct":"Delpacibart Zotadirsen","moa":"Dystrophin exon-44","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0.59999999999999998,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.68999999999999995,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Leerink Partners"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"SRP-5051","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"SRP-5051","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vesleteplirsen","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Vesleteplirsen","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Vesleteplirsen","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Vesleteplirsen","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Vesleteplirsen","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Vesleteplirsen","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Vesleteplirsen","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"Casimersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Casimersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Casimersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Casimersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2013","type":"Inapplicable","leadProduct":"Drisapersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"Drisapersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2013","type":"Inapplicable","leadProduct":"Drisapersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2013","type":"Inapplicable","leadProduct":"Drisapersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"DS-5141b","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Daiichi Sankyo \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Daiichi Sankyo \/ Undisclosed"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Daiichi Sankyo","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"DS-5141b","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Daiichi Sankyo \/ Daiichi Sankyo","highestDevelopmentStatusID":"8","companyTruncated":"Daiichi Sankyo \/ Daiichi Sankyo"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"Eteplirsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Eteplirsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2011","type":"Inapplicable","leadProduct":"Eteplirsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2012","type":"Inapplicable","leadProduct":"Eteplirsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"Eteplirsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"Eteplirsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2017","type":"Inapplicable","leadProduct":"Eteplirsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Eteplirsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Rare Disease Research, LLC","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Golodirsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Rare Disease Research, LLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Rare Disease Research, LLC \/ Sarepta Therapeutics","highestDevelopmentStatusID":"11","companyTruncated":"Rare Disease Research, LLC \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2010","type":"Inapplicable","leadProduct":"GSK2402968","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2010","type":"Inapplicable","leadProduct":"GSK2402968","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2010","type":"Inapplicable","leadProduct":"GSK2402968","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2011","type":"Inapplicable","leadProduct":"GSK2402968","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2011","type":"Inapplicable","leadProduct":"GSK2402968","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"SRP-4045","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"SRP-4045","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Institut de Myologie | Consultants for Research in Imaging and Spectroscopy | Great Ormond Street Hospital for Children NHS Foundation Trust | Catholic University of the Sacred Heart | Royal Holloway University | SYSNAV Healthcare | University College, Lo","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"SRP-4053","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Institut de Myologie | Consultants for Research in Imaging and Spectroscopy | Great Ormond Street Hospital for Children NHS Foundation Trust | Catholic University of the Sacred Heart | Royal Holloway University | SYSNAV Healthcare | University College, Lo","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Institut de Myologie | Consultants for Research in Imaging and Spectroscopy | Great Ormond Street Hospital for Children NHS Foundation Trust | Catholic University of the Sacred Heart | Royal Holloway University | SYSNAV Healthcare | University College, Lo"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Suvodirsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"National Center of Neurology and Psychiatry, Japan","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase I","graph3":"National Center of Neurology and Psychiatry, Japan","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Center of Neurology and Psychiatry, Japan \/ Nippon Shinyaku","highestDevelopmentStatusID":"6","companyTruncated":"National Center of Neurology and Psychiatry, Japan \/ Nippon Shinyaku"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Nippon Shinyaku | Cooperative International Neuromuscular Research Group | Therapeutic Research in Neuromuscular Disorders Solutions","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2016","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NS Pharma \/ Nippon Shinyaku | Cooperative International Neuromuscular Research Group | Therapeutic Research in Neuromuscular Disorders Solutions","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Nippon Shinyaku | Cooperative International Neuromuscular Research Group | Therapeutic Research in Neuromuscular Disorders Solutions"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Nippon Shinyaku | Cooperative International Neuromuscular Research Group | Therapeutic Research in Neuromuscular Disorders Solutions","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2017","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NS Pharma \/ Nippon Shinyaku | Cooperative International Neuromuscular Research Group | Therapeutic Research in Neuromuscular Disorders Solutions","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Nippon Shinyaku | Cooperative International Neuromuscular Research Group | Therapeutic Research in Neuromuscular Disorders Solutions"},{"orgOrder":0,"company":"Nippon Shinyaku","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Nippon Shinyaku","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nippon Shinyaku \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Nippon Shinyaku \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Nippon Shinyaku","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Nippon Shinyaku"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Nippon Shinyaku","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Nippon Shinyaku"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Nippon Shinyaku","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Nippon Shinyaku"},{"orgOrder":0,"company":"Option Care Health","sponsor":"NS Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Agreement","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Option Care Health","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Option Care Health \/ NS Pharma","highestDevelopmentStatusID":"15","companyTruncated":"Option Care Health \/ NS Pharma"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"WVE-210201","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"PRO044","moa":"Dystrophin pre-mRNA exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"PRO045","moa":"Dystrophin pre-mRNA exon-53","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Cure Rare","sponsor":"Charles River Laboratories, Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"CRD-TMH-001","moa":"Dystrophin protein","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Cure Rare","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cure Rare \/ Charles River Laboratories, Inc","highestDevelopmentStatusID":"5","companyTruncated":"Cure Rare \/ Charles River Laboratories, Inc"},{"orgOrder":0,"company":"Cure Rare","sponsor":"University of Massachusetts, Worcester","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"CRD-TMH-001","moa":"Dystrophin protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Cure Rare","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Cure Rare \/ University of Massachusetts, Worcester","highestDevelopmentStatusID":"6","companyTruncated":"Cure Rare \/ University of Massachusetts, Worcester"},{"orgOrder":0,"company":"Suzhou GenAssist Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"GEN6050X","moa":"E50","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Suzhou GenAssist Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Suzhou GenAssist Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Suzhou GenAssist Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"National Ataxia Foundation","sponsor":"Biohaven Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Partnership","leadProduct":"Troriluzole","moa":"EAAT2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"National Ataxia Foundation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"National Ataxia Foundation \/ Biohaven Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"National Ataxia Foundation \/ Biohaven Pharmaceuticals"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Troriluzole","moa":"EAAT2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Troriluzole","moa":"EAAT2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Troriluzole","moa":"EAAT2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Troriluzole","moa":"EAAT2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Troriluzole","moa":"EAAT2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Troriluzole","moa":"EAAT2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Troriluzole","moa":"EAAT2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Troriluzole","moa":"EAAT2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"IQVIA","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ IQVIA","highestDevelopmentStatusID":"8","companyTruncated":"Inozyme Pharma \/ IQVIA"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Thomas Jefferson University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Thomas Jefferson University","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ Thomas Jefferson University"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"IQVIA","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ IQVIA","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ IQVIA"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Thomas Jefferson University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Thomas Jefferson University","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ Thomas Jefferson University"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"FTX-6058","moa":"EED","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"FTX-6058","moa":"EED","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"FTX-6058","moa":"EED","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"FTX-6058","moa":"EED","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"FTX-6058","moa":"EED","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"FTX-6058","moa":"EED","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"FTX-6058","moa":"EED","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"FTX-6058","moa":"EED","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"FTX-6058","moa":"EED","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"FTX-6058","moa":"EED","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Calico Life Sciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Fosigotifator","moa":"EIF2B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AbbVie Inc \/ Calico Life Sciences","highestDevelopmentStatusID":"7","companyTruncated":"AbbVie Inc \/ Calico Life Sciences"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Fosigotifator","moa":"EIF2B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AbbVie Inc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AbbVie Inc \/ Undisclosed"},{"orgOrder":0,"company":"Calico Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Fosigotifator","moa":"EIF2B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Calico Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Calico Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Calico Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Calico Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Fosigotifator","moa":"EIF2B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Calico Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Calico Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Calico Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"The University of Pittsburgh School of Medicine","sponsor":"Parion Sciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Hypertonic Saline","moa":"Electrolyte\/osmotic gradient","graph1":"Genetic Disease","graph2":"Phase I","graph3":"The University of Pittsburgh School of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"The University of Pittsburgh School of Medicine \/ Parion Sciences","highestDevelopmentStatusID":"6","companyTruncated":"The University of Pittsburgh School of Medicine \/ Parion Sciences"},{"orgOrder":0,"company":"Aceragen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2022","type":"Inapplicable","leadProduct":"Sodium Fusidate","moa":"Elongation factor G","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aceragen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Aceragen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aceragen \/ Undisclosed"},{"orgOrder":0,"company":"Arrevus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2020","type":"Inapplicable","leadProduct":"Sodium Fusidate","moa":"Elongation factor G","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arrevus","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Arrevus \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arrevus \/ Undisclosed"},{"orgOrder":0,"company":"Arrevus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2020","type":"Inapplicable","leadProduct":"Sodium Fusidate","moa":"Elongation factor G","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arrevus","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Arrevus \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arrevus \/ Undisclosed"},{"orgOrder":0,"company":"TikunTX","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"BPN-36964","moa":"ELP1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"TikunTX","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"TikunTX \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"TikunTX \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Aro-Enac","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"ARO-ENaC","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ARO-ENaC","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ARO-ENaC","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ARO-ENaC","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"AZD5634","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"BI1265162","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"BI443651","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"ETD001","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Enterprise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Enterprise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"ETD001","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Enterprise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Enterprise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"ETD001","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Enterprise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Enterprise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"ETD001","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Enterprise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Enterprise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"ETD001","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Enterprise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Enterprise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Panakes Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2024","type":"Series B Financing","leadProduct":"ETD001","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enterprise Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Inhalation","sponsorNew":"Enterprise Therapeutics \/ Panakes Partners","highestDevelopmentStatusID":"8","companyTruncated":"Enterprise Therapeutics \/ Panakes Partners"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"IONIS-ENAC-2.5Rx","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Augusta University","sponsor":"Gilead Sciences | National Heart, Lung, and Blood Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Ambrisentan","moa":"Endothelin receptor, ET-A\/ET-B","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Augusta University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pill","sponsorNew":"Augusta University \/ Gilead Sciences | National Heart, Lung, and Blood Institute","highestDevelopmentStatusID":"6","companyTruncated":"Augusta University \/ Gilead Sciences | National Heart, Lung, and Blood Institute"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Rallybio","sponsor":"Recursion Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"REV102","moa":"ENPP1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rallybio","amount2":0.029999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Rallybio \/ Recursion Pharma","highestDevelopmentStatusID":"4","companyTruncated":"Rallybio \/ Recursion Pharma"},{"orgOrder":0,"company":"Rallybio","sponsor":"Recursion Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"REV102","moa":"ENPP1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rallybio","amount2":0.029999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Rallybio \/ Recursion Pharma","highestDevelopmentStatusID":"4","companyTruncated":"Rallybio \/ Recursion Pharma"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Galactosemia Foundation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Galactosemia Foundation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Galactosemia Foundation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Galactosemia Foundation \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Advanz Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Partnership","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0.14000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14000000000000001,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Advanz Pharma","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Advanz Pharma"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Perceptive Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Private Placement","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Perceptive Advisors","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Perceptive Advisors"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"SVB Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Public Offering","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ SVB Securities","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ SVB Securities"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Venrock Healthcare Capital Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Private Placement","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Venrock Healthcare Capital Partners","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Venrock Healthcare Capital Partners"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Zydus Lifesciences","sponsor":"Indian Council of Medical Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Desidustat","moa":"enzyme inhibitors: hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitors","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zydus Lifesciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zydus Lifesciences \/ Indian Council of Medical Research","highestDevelopmentStatusID":"8","companyTruncated":"Zydus Lifesciences \/ Indian Council of Medical Research"},{"orgOrder":0,"company":"Italfarmaco","sponsor":"Syneos Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"enzyme inhibitors: inhibitors of histone deacetylase; vinca alkaloids","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Italfarmaco","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco \/ Syneos Health","highestDevelopmentStatusID":"10","companyTruncated":"Italfarmaco \/ Syneos Health"},{"orgOrder":0,"company":"Italfarmaco","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"enzyme inhibitors: inhibitors of histone deacetylase; vinca alkaloids","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Italfarmaco","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Italfarmaco \/ Undisclosed"},{"orgOrder":0,"company":"Italfarmaco","sponsor":"Cromsource","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"enzyme inhibitors: inhibitors of histone deacetylase; vinca alkaloids","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Italfarmaco","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco \/ Cromsource","highestDevelopmentStatusID":"10","companyTruncated":"Italfarmaco \/ Cromsource"},{"orgOrder":0,"company":"Italfarmaco","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"enzyme inhibitors: inhibitors of histone deacetylase; vinca alkaloids","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Italfarmaco","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Italfarmaco \/ Undisclosed"},{"orgOrder":0,"company":"Italfarmaco","sponsor":"Fortrea","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"enzyme inhibitors: inhibitors of histone deacetylase; vinca alkaloids","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Italfarmaco","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Italfarmaco \/ Fortrea","highestDevelopmentStatusID":"10","companyTruncated":"Italfarmaco \/ Fortrea"},{"orgOrder":0,"company":"Immusoft Corporation","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Funding","leadProduct":"Iduronate Sulfatase","moa":"Enzyme Replacement","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Immusoft Corporation","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Immusoft Corporation \/ California Institute for Regenerative Medicine","highestDevelopmentStatusID":"5","companyTruncated":"Immusoft Corporation \/ California Institute for Regenerative Medicine"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2015","type":"Inapplicable","leadProduct":"HGT-1410","moa":"enzymes","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2014","type":"Inapplicable","leadProduct":"SBC-103","moa":"enzymes","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2015","type":"Inapplicable","leadProduct":"SBC-103","moa":"enzymes","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2019","type":"Inapplicable","leadProduct":"TAK-755","moa":"enzymes","graph1":"Genetic Disease","graph2":"Phase I","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"SHIRE PLC \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"6","companyTruncated":"SHIRE PLC \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Cystetic Medicines","sponsor":"DevPro Biopharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2023","type":"Inapplicable","leadProduct":"Amphotericin B Cystetic","moa":"Ergosterol","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Cystetic Medicines","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Cystetic Medicines \/ DevPro Biopharma","highestDevelopmentStatusID":"6","companyTruncated":"Cystetic Medicines \/ DevPro Biopharma"},{"orgOrder":0,"company":"NFlection Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"NFX-179","moa":"ERK activator kinase (MEK)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NFlection Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"NFlection Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NFlection Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"NFlection Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"NFX-179","moa":"ERK activator kinase (MEK)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NFlection Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"NFlection Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NFlection Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ISU ABXIS","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"ISU302","moa":"Erythropoietin receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ISU ABXIS","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ISU ABXIS \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"ISU ABXIS \/ Undisclosed"},{"orgOrder":0,"company":"ISU ABXIS","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"ISU302","moa":"Erythropoietin receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ISU ABXIS","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ISU ABXIS \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"ISU ABXIS \/ Undisclosed"},{"orgOrder":0,"company":"ISU ABXIS","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"ISU302","moa":"Erythropoietin receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ISU ABXIS","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ISU ABXIS \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"ISU ABXIS \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"PF-07209326","moa":"E-selectin (SELE)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2012","type":"Inapplicable","leadProduct":"Estradiol","moa":"Estrogen receptor alpha","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"London Health Sciences Centre Research Institute (Lawson)","sponsor":"Greenstone Biosciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Genistein","moa":"Estrogen receptor beta (ESR2)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"London Health Sciences Centre Research Institute (Lawson)","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"London Health Sciences Centre Research Institute (Lawson) \/ Greenstone Biosciences","highestDevelopmentStatusID":"7","companyTruncated":"London Health Sciences Centre Research Institute (Lawson) \/ Greenstone Biosciences"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Public Offering","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"PepGen \/ RA Capital Management","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ RA Capital Management"},{"orgOrder":0,"company":"PepGen","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Financing","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0.11,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Infusion","sponsorNew":"PepGen \/ RA Capital Management","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ RA Capital Management"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"BofA Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Public Offering","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ BofA Securities","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ BofA Securities"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"PGN-EDO51","moa":"Exon-51","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"PGN-EDO53","moa":"Exon-53","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"PepGen \/ Undisclosed"},{"orgOrder":0,"company":"Capricor","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"CAP-1002","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Capricor","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Capricor \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Capricor \/ Undisclosed"},{"orgOrder":0,"company":"Nippon Shinyaku","sponsor":"Capricor Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell & Gene Therapy","year":"2022","type":"Partnership","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Nippon Shinyaku","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nippon Shinyaku \/ Capricor Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Nippon Shinyaku \/ Capricor Therapeutics"},{"orgOrder":0,"company":"Lonza Group","sponsor":"Capricor Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2021","type":"Collaboration","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Lonza Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lonza Group \/ Capricor Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Lonza Group \/ Capricor Therapeutics"},{"orgOrder":0,"company":"Capricor","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Capricor \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor \/ Undisclosed"},{"orgOrder":0,"company":"Capricor","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Capricor","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Capricor \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Cantor Fitzgerald","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Public Offering","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Cantor Fitzgerald","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Cantor Fitzgerald"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Nippon Shinyaku","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Nippon Shinyaku"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Partnership","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.73999999999999999,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Nippon Shinyaku","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Nippon Shinyaku"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Partnership","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0.73999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.73999999999999999,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Nippon Shinyaku","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Nippon Shinyaku"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Partnership","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0.73999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.73999999999999999,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Nippon Shinyaku","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Nippon Shinyaku"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Piper Sandler","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Piper Sandler","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Piper Sandler"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Piper Sandler","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Piper Sandler","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Piper Sandler"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Piper Sandler","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Piper Sandler","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Piper Sandler"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Capricor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Capricor Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Chia Tai Tianqing Pharmaceutical Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Antihemophilic Factor (Recombinant)","moa":"F10","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Chia Tai Tianqing Pharmaceutical Group","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Garadacimab","moa":"F12a","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Garadacimab","moa":"F12a","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Garadacimab","moa":"F12a","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Garadacimab","moa":"F12a","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Garadacimab","moa":"F12a","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Garadacimab","moa":"F12a","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Garadacimab","moa":"F12a","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Garadacimab","moa":"F12a","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"LFB Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Coagulation Factor VIIa","moa":"F7a","graph1":"Genetic Disease","graph2":"Phase I","graph3":"LFB Group","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"LFB Group \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"LFB Group \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Giroctocogene Fitelparovec","moa":"F8 gene (Factor VIII)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Coagulation Factor VIII","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Coagulation Factor VIII","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2013","type":"Inapplicable","leadProduct":"Coagulation Factor VIII","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Coagulation Factor VIII","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Coagulation Factor VIII","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"Coagulation Factor VIII","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Coagulation Factor VIII","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Coagulation Factor VIII","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Coagulation Factor VIII","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"City of Hope","sponsor":"Charta Foundation | Grifols International | CSL Behring | Biotest","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Coagulation Factor VIII","moa":"F8*","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"City of Hope","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"City of Hope \/ Charta Foundation | Grifols International | CSL Behring | Biotest","highestDevelopmentStatusID":"1","companyTruncated":"City of Hope \/ Charta Foundation | Grifols International | CSL Behring | Biotest"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"PSI CRO","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Efanesoctocog Alfa","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ PSI CRO","highestDevelopmentStatusID":"11","companyTruncated":"Swedish Orphan Biovitrum AB \/ PSI CRO"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Efanesoctocog Alfa","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Efanesoctocog Alfa","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Efanesoctocog Alfa","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Efanesoctocog Alfa","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Efanesoctocog Alfa","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Efanesoctocog Alfa","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Syneos Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Efanesoctocog Alfa","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Swedish Orphan Biovitrum AB \/ Syneos Health","highestDevelopmentStatusID":"11","companyTruncated":"Swedish Orphan Biovitrum AB \/ Syneos Health"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"PSI CRO","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Efanesoctocog Alfa","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Swedish Orphan Biovitrum AB \/ PSI CRO","highestDevelopmentStatusID":"11","companyTruncated":"Swedish Orphan Biovitrum AB \/ PSI CRO"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"PSI CRO","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Efanesoctocog Alfa","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Swedish Orphan Biovitrum AB \/ PSI CRO","highestDevelopmentStatusID":"11","companyTruncated":"Swedish Orphan Biovitrum AB \/ PSI CRO"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"PSI CRO","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Efanesoctocog Alfa","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Swedish Orphan Biovitrum AB \/ PSI CRO","highestDevelopmentStatusID":"11","companyTruncated":"Swedish Orphan Biovitrum AB \/ PSI CRO"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"BCX10013","moa":"Factor D","graph1":"Genetic Disease","graph2":"Phase I","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AAV5-hFIX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2015","type":"Inapplicable","leadProduct":"AAV5-hFIX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Children's Hospital of Philadelphia | The University of Pittsburgh School of Medicine | Royal Prince Alfred Hospital, Sydney | St. James's Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"AAV8-hFIX19","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Spark Therapeutics, Inc \/ Children's Hospital of Philadelphia | The University of Pittsburgh School of Medicine | Royal Prince Alfred Hospital, Sydney | St. James's Hospital","highestDevelopmentStatusID":"6","companyTruncated":"Spark Therapeutics, Inc \/ Children's Hospital of Philadelphia | The University of Pittsburgh School of Medicine | Royal Prince Alfred Hospital, Sydney | St. James's Hospital"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"BAX 326","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"BAX 326","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"BAX326","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"CB 2679d-GT","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Coagulation Factor IX (Recombinant)","moa":"factor IX","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Baxalta Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2018","type":"Inapplicable","leadProduct":"Coagulation Factor IX (Recombinant)","moa":"factor IX","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Coagulation Factor IX (Recombinant)","moa":"factor IX","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Coagulation Factor IX (Recombinant)","moa":"factor IX","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Dalcinonacog Alpha","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Dalcinonacog Alpha","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Dalcinonacog Alpha","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"DTX101","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Factor IX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Factor IX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Factor IX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Factor IX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Factor IX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Factor IX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein-drug Conjugate","year":"2011","type":"Inapplicable","leadProduct":"Factor IX Fc Fusion Protein","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein-drug Conjugate","year":"2011","type":"Inapplicable","leadProduct":"Factor IX Fc Fusion Protein","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein-drug Conjugate","year":"2014","type":"Inapplicable","leadProduct":"Factor IX Fc Fusion Protein","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"FLT180a","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aptevo Therapeutics","sponsor":"Medexus Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Acquisition","leadProduct":"Recombinant Coagulation Factor Ix","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Aptevo Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Infusion","sponsorNew":"Aptevo Therapeutics \/ Medexus Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Aptevo Therapeutics \/ Medexus Pharmaceuticals"},{"orgOrder":0,"company":"Aptevo Therapeutics","sponsor":"Medexus Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Acquisition","leadProduct":"Recombinant Coagulation Factor Ix","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Aptevo Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Infusion","sponsorNew":"Aptevo Therapeutics \/ Medexus Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Aptevo Therapeutics \/ Medexus Pharmaceuticals"},{"orgOrder":0,"company":"Aptevo Therapeutics","sponsor":"XOMA","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Agreement","leadProduct":"Recombinant Coagulation Factor Ix","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Aptevo Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Infusion","sponsorNew":"Aptevo Therapeutics \/ XOMA","highestDevelopmentStatusID":"15","companyTruncated":"Aptevo Therapeutics \/ XOMA"},{"orgOrder":0,"company":"Medexus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Recombinant Coagulation Factor Ix","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Medexus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Medexus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Medexus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Recombinant Coagulation Factor IX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Recombinant Coagulation Factor IX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Portfolio Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Public Offering","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Syncona Portfolio Limited","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Syncona Portfolio Limited"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Organovo Holdings","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Series C Financing","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0.12,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Organovo Holdings","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Organovo Holdings"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Public Offering","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Portfolio Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Public Offering","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Syncona Portfolio Limited","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Syncona Portfolio Limited"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"University College London","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ University College London","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ University College London"},{"orgOrder":0,"company":"Thermo Fisher Scientific","sponsor":"Spur Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Agreement","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Thermo Fisher Scientific","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Thermo Fisher Scientific \/ Spur Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Thermo Fisher Scientific \/ Spur Therapeutics"},{"orgOrder":0,"company":"GeneVentiv Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"GENV HEM","moa":"Factor Va","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"GeneVentiv Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"GeneVentiv Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"GeneVentiv Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"GeneVentiv Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Licensing Agreement","leadProduct":"GENV HEM","moa":"Factor Va","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"University of North Carolina","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"University of North Carolina \/ GeneVentiv Therapeutics","highestDevelopmentStatusID":"5","companyTruncated":"University of North Carolina \/ GeneVentiv Therapeutics"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"BAY86-6150","moa":"Factor VIIa","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2013","type":"Inapplicable","leadProduct":"BAY86-6150","moa":"Factor VIIa","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"LFB Group","sponsor":"LFB Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2013","type":"Inapplicable","leadProduct":"Coagulation Factor VIIa, Recombinant","moa":"Factor VIIa","graph1":"Genetic Disease","graph2":"Phase III","graph3":"LFB Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"LFB Group \/ LFB Group","highestDevelopmentStatusID":"10","companyTruncated":"LFB Group \/ LFB Group"},{"orgOrder":0,"company":"LFB Group","sponsor":"LFB USA","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Coagulation Factor VIIa, Recombinant","moa":"Factor VIIa","graph1":"Genetic Disease","graph2":"Phase III","graph3":"LFB Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"LFB Group \/ LFB USA","highestDevelopmentStatusID":"10","companyTruncated":"LFB Group \/ LFB USA"},{"orgOrder":0,"company":"LFB Group","sponsor":"LFB USA","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Coagulation Factor VIIa, Recombinant","moa":"Factor VIIa","graph1":"Genetic Disease","graph2":"Phase III","graph3":"LFB Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"LFB Group \/ LFB USA","highestDevelopmentStatusID":"10","companyTruncated":"LFB Group \/ LFB USA"},{"orgOrder":0,"company":"LFB Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Coagulation Factor VIIa, Recombinant","moa":"Factor VIIa","graph1":"Genetic Disease","graph2":"Phase III","graph3":"LFB Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"LFB Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"LFB Group \/ Undisclosed"},{"orgOrder":0,"company":"American Thrombosis and Hemostasis Network","sponsor":"LFB USA","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Coagulation Factor VIIa, Recombinant","moa":"Factor VIIa","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"American Thrombosis and Hemostasis Network","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"American Thrombosis and Hemostasis Network \/ LFB USA","highestDevelopmentStatusID":"11","companyTruncated":"American Thrombosis and Hemostasis Network \/ LFB USA"},{"orgOrder":0,"company":"Grifols International","sponsor":"Grifols International","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Alphanate","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grifols International \/ Grifols International","highestDevelopmentStatusID":"8","companyTruncated":"Grifols International \/ Grifols International"},{"orgOrder":0,"company":"Gabrail Cancer Center Research","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Protein-drug Conjugate","year":"2017","type":"Inapplicable","leadProduct":"Antihemophilic Factor (Recombinant), Fc Fusion Protein","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Gabrail Cancer Center Research","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Gabrail Cancer Center Research \/ Sanofi","highestDevelopmentStatusID":"6","companyTruncated":"Gabrail Cancer Center Research \/ Sanofi"},{"orgOrder":0,"company":"Jiangsu Gensciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Antihemophilic Factor (Recombinant), Plasma","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Jiangsu Gensciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jiangsu Gensciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Jiangsu Gensciences \/ Undisclosed"},{"orgOrder":0,"company":"Jiangsu Gensciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Antihemophilic Factor (Recombinant), Plasma","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Jiangsu Gensciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jiangsu Gensciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Jiangsu Gensciences \/ Undisclosed"},{"orgOrder":0,"company":"Oregon Health and Science University","sponsor":"Baxter Healthcare Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Antihemophilic Factor (Recombinant), Plasma","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Oregon Health and Science University","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oregon Health and Science University \/ Baxter Healthcare Corporation","highestDevelopmentStatusID":"10","companyTruncated":"Oregon Health and Science University \/ Baxter Healthcare Corporation"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Antihemophilic Factor (Recombinant), Plasma","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Bayer AG","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Antihemophilic Factor (Recombinant), Plasma","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Bayer AG","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Bayer AG"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Antihemophilic Factor (recombinant), PeGylated","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Antihemophilic Factor Recombinant PEGylated","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"ASC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ASC618","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ASC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ASC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ASC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"ASC Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Expanded Collaboration","leadProduct":"ASC618","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Charles River Laboratories, Inc \/ ASC Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Charles River Laboratories, Inc \/ ASC Therapeutics"},{"orgOrder":0,"company":"ASC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ASC618","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ASC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ASC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ASC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"BAX 826","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Baxalta Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"BAX855","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"BAY94-9027","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"BAY94-9027","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"BAY94-9027","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Atlantic Research Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Beroctocog Alfa","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Green Cross Lab Cell \/ Atlantic Research Group","highestDevelopmentStatusID":"10","companyTruncated":"Green Cross Lab Cell \/ Atlantic Research Group"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Atlantic Research Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Beroctocog Alfa","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Green Cross Lab Cell \/ Atlantic Research Group","highestDevelopmentStatusID":"10","companyTruncated":"Green Cross Lab Cell \/ Atlantic Research Group"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Parexel","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Biostate","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Parexel","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Parexel"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Parexel","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Biostate","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Parexel","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Parexel"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Biostate","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Biostate","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Agnafit Bidco","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2021","type":"Acquisition","leadProduct":"Efmoroctocog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":8,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":8,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Agnafit Bidco","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Agnafit Bidco"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"National Health Service (Nhs)","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2020","type":"Agreement","leadProduct":"Efmoroctocog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ National Health Service (Nhs)","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ National Health Service (Nhs)"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein-drug Conjugate","year":"2014","type":"Inapplicable","leadProduct":"Factor VIII Fc Fusion Protein","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein-drug Conjugate","year":"2017","type":"Inapplicable","leadProduct":"Factor VIII Fc Fusion Protein","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder","sponsorNew":"Sanofi \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein-drug Conjugate","year":"2011","type":"Inapplicable","leadProduct":"Factor VIII Fc Fusion Protein","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein-drug Conjugate","year":"2015","type":"Inapplicable","leadProduct":"Factor VIII Fc Fusion Protein","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein-drug Conjugate","year":"2014","type":"Inapplicable","leadProduct":"Factor VIII Fc Fusion Protein","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Children's Hospital Los Angeles","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"Feiba","moa":"Factor VIII inhibitor bypassing activity","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Children's Hospital Los Angeles","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Children's Hospital Los Angeles \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"Children's Hospital Los Angeles \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Baxalta Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"Feiba","moa":"Factor VIII inhibitor bypassing activity","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2018","type":"Inapplicable","leadProduct":"Coagulation Factor VIIa","moa":"F7a||Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Marzeptacog Alfa","moa":"Factor VIIa||Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Marzeptacog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Marzeptacog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Marzeptacog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Marzeptacog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Marzeptacog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Marzeptacog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Marzeptacog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"GC Biopharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Acquisition","leadProduct":"Marzeptacog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ GC Biopharma","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ GC Biopharma"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Marzeptacog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Marzeptacog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Marzeptacog Alfa","moa":"Factor VIIa||Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"NNC0365-3769","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"NNC0365-3769","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"NNC0365-3769","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"NNC0365-3769","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"OCTA101","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"OCTA12","moa":"||Factor VIII","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Chia Tai Tianqing Pharmaceutical Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Recombinant Activated Coagulation Factor VIII","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chia Tai Tianqing Pharmaceutical Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Recombinant Von Willebrand Factor","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Recombinant Von Willebrand Factor","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Recombinant Von Willebrand Factor","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Aryogen Pharmed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRAN","productType":"Protein-drug Conjugate","year":"2023","type":"Inapplicable","leadProduct":"Rfviii-Fc","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Aryogen Pharmed","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aryogen Pharmed \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Aryogen Pharmed \/ Undisclosed"},{"orgOrder":0,"company":"H\u00e4mophilie-Zentrum Rhein Main","sponsor":"Octapharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Simoctocog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"H\u00e4mophilie-Zentrum Rhein Main","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"H\u00e4mophilie-Zentrum Rhein Main \/ Octapharma","highestDevelopmentStatusID":"15","companyTruncated":"H\u00e4mophilie-Zentrum Rhein Main \/ Octapharma"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Simoctocog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Simoctocog Alpha","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Antihemophilic Factor (Recombinant)","moa":"F10||Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Antihemophilic Factor (Recombinant)","moa":"F10||Factor VIII","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Gabrail Cancer Center Research","sponsor":"Genentech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Antihemophilic Factor (Recombinant)","moa":"F10||Factor VIII","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Gabrail Cancer Center Research","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Gabrail Cancer Center Research \/ Genentech","highestDevelopmentStatusID":"10","companyTruncated":"Gabrail Cancer Center Research \/ Genentech"},{"orgOrder":0,"company":"TeraImmune","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"TI-168","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"TeraImmune","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"TeraImmune \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"TeraImmune \/ Undisclosed"},{"orgOrder":0,"company":"TeraImmune","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Agreement","leadProduct":"TI-168","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"TeraImmune","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"TeraImmune \/ National Institutes of Health","highestDevelopmentStatusID":"5","companyTruncated":"TeraImmune \/ National Institutes of Health"},{"orgOrder":0,"company":"TeraImmune","sponsor":"Baudax Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Acquisition","leadProduct":"TI-168","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"TeraImmune","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"TeraImmune \/ Baudax Bio","highestDevelopmentStatusID":"5","companyTruncated":"TeraImmune \/ Baudax Bio"},{"orgOrder":0,"company":"Baudax Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"TI-168","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Baudax Bio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baudax Bio \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Baudax Bio \/ Undisclosed"},{"orgOrder":0,"company":"Fondazione IRCCS Ca' Granda","sponsor":"Sintesi Research Srl","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Plasma-derived FVIII\/VWF Concentrate","moa":"Factor VIII\/VWF","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Fondazione IRCCS Ca' Granda","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Fondazione IRCCS Ca' Granda \/ Sintesi Research Srl","highestDevelopmentStatusID":"11","companyTruncated":"Fondazione IRCCS Ca' Granda \/ Sintesi Research Srl"},{"orgOrder":0,"company":"Grifols International","sponsor":"Grifols International","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Plasma-derived FVIII\/VWF Concentrate","moa":"Factor VIII\/VWF","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Grifols International","highestDevelopmentStatusID":"11","companyTruncated":"Grifols International \/ Grifols International"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Mim8 B","moa":"Factor VIIIa mimetic","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"NXT007","moa":"Factor VIIIa mimetic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Albutrepenonacog Alpha","moa":"Factor X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"LFB Group","sponsor":"HEMA Biologics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Eptacog Alpha","moa":"Factor X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"LFB Group","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"LFB Group \/ HEMA Biologics","highestDevelopmentStatusID":"15","companyTruncated":"LFB Group \/ HEMA Biologics"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"ARC19499","moa":"Factor XI","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Castle Creek Biosciences","sponsor":"Paragon Biosciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"LV-FAH","moa":"FAH","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Castle Creek Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Castle Creek Biosciences \/ Paragon Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"Castle Creek Biosciences \/ Paragon Biosciences"},{"orgOrder":0,"company":"Philogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"177-Lu OncoFAP-23","moa":"FAP","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Philogen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Philogen \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Philogen \/ Undisclosed"},{"orgOrder":0,"company":"Hospital for Special Surgery, New York","sponsor":"The New York Community Trust | East River Medical Imaging | Northwell Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Risedronate Sodium","moa":"Farnesyl diphosphate synthase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Hospital for Special Surgery, New York","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Hospital for Special Surgery, New York \/ The New York Community Trust | East River Medical Imaging | Northwell Health","highestDevelopmentStatusID":"11","companyTruncated":"Hospital for Special Surgery, New York \/ The New York Community Trust | East River Medical Imaging | Northwell Health"},{"orgOrder":0,"company":"Papworth Hospital NHS Foundation Trust","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Zoledronic Acid","moa":"Farnesyl diphosphate synthase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Papworth Hospital NHS Foundation Trust","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Papworth Hospital NHS Foundation Trust \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"11","companyTruncated":"Papworth Hospital NHS Foundation Trust \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Cytokinetics","sponsor":"Astellas Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Reldesemtiv","moa":"Fast skeletal troponin complex","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cytokinetics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Cytokinetics \/ Astellas Pharma","highestDevelopmentStatusID":"8","companyTruncated":"Cytokinetics \/ Astellas Pharma"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2019","type":"Inapplicable","leadProduct":"Pancrelipase","moa":"Fatty acids | Starch | Polypeptides","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Delayed Release","sponsorNew":"AbbVie Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"AbbVie Inc \/ Undisclosed"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Pancrelipase","moa":"Fatty acids | Starch | Polypeptides","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Delayed Release","sponsorNew":"AbbVie Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"AbbVie Inc \/ Undisclosed"},{"orgOrder":0,"company":"Acceleron Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"ACE-083","moa":"Fc fusion protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Acceleron Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Acceleron Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Acceleron Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Acceleron Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"ACE-083","moa":"Fc fusion protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Acceleron Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Acceleron Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Acceleron Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Acceleron Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"ACE-083","moa":"Fc fusion protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Acceleron Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Acceleron Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Acceleron Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Acceleron Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"ACE-083","moa":"Fc fusion protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Acceleron Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Acceleron Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Acceleron Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Gammaplex","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2013","type":"Inapplicable","leadProduct":"Gammaplex","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Atlantic Research Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"GC5107","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Green Cross Lab Cell \/ Atlantic Research Group","highestDevelopmentStatusID":"10","companyTruncated":"Green Cross Lab Cell \/ Atlantic Research Group"},{"orgOrder":0,"company":"GC Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"GC5107","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GC Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"GC Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GC Pharma \/ Undisclosed"},{"orgOrder":0,"company":"GC Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"GC5107","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GC Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"GC Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GC Pharma \/ Undisclosed"},{"orgOrder":0,"company":"GC Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"GC5107","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GC Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GC Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GC Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Parexel | Atlantic Research Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Antibody, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"GC5107","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Green Cross Lab Cell \/ Parexel | Atlantic Research Group","highestDevelopmentStatusID":"10","companyTruncated":"Green Cross Lab Cell \/ Parexel | Atlantic Research Group"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Human Immunoglobulin","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"GC Biopharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GC Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Public Offering","leadProduct":"Human Immunoglobulin","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ADMA Biologics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Infusion","sponsorNew":"ADMA Biologics \/ Morgan Stanley","highestDevelopmentStatusID":"15","companyTruncated":"ADMA Biologics \/ Morgan Stanley"},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Raymond James & Associates, Inc.","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Public Offering","leadProduct":"Human Immunoglobulin","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ADMA Biologics","amount2":0.070000000000000007,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Infusion","sponsorNew":"ADMA Biologics \/ Raymond James & Associates, Inc.","highestDevelopmentStatusID":"15","companyTruncated":"ADMA Biologics \/ Raymond James & Associates, Inc."},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Raymond James & Associates, Inc.","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Public Offering","leadProduct":"Human Immunoglobulin","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ADMA Biologics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Infusion","sponsorNew":"ADMA Biologics \/ Raymond James & Associates, Inc.","highestDevelopmentStatusID":"15","companyTruncated":"ADMA Biologics \/ Raymond James & Associates, Inc."},{"orgOrder":0,"company":"Evolve Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Human Immunoglobulin","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Evolve Biologics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Evolve Biologics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Evolve Biologics \/ Undisclosed"},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Human Immunoglobulin","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ADMA Biologics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ADMA Biologics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"ADMA Biologics \/ Undisclosed"},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Human Immunoglobulin","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ADMA Biologics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ADMA Biologics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"ADMA Biologics \/ Undisclosed"},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Public Offering","leadProduct":"Human Immunoglobulin","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ADMA Biologics","amount2":0.089999999999999997,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Infusion","sponsorNew":"ADMA Biologics \/ Morgan Stanley","highestDevelopmentStatusID":"15","companyTruncated":"ADMA Biologics \/ Morgan Stanley"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Human Immunoglobulin","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Raymond James & Associates, Inc.","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Public Offering","leadProduct":"Human Immunoglobulin","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ADMA Biologics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"ADMA Biologics \/ Raymond James & Associates, Inc.","highestDevelopmentStatusID":"15","companyTruncated":"ADMA Biologics \/ Raymond James & Associates, Inc."},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Raymond James & Associates, Inc.","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Public Offering","leadProduct":"Human Immunoglobulin","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ADMA Biologics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"ADMA Biologics \/ Raymond James & Associates, Inc.","highestDevelopmentStatusID":"15","companyTruncated":"ADMA Biologics \/ Raymond James & Associates, Inc."},{"orgOrder":0,"company":"Liverpool School of Tropical Medicine","sponsor":"University of Bergen | University of Malawi | Makerere University | Indiana University | Global Health Uganda","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Dihydroartemisinin","moa":"Ferriprotoporphyrin IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liverpool School of Tropical Medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Liverpool School of Tropical Medicine \/ University of Bergen | University of Malawi | Makerere University | Indiana University | Global Health Uganda","highestDevelopmentStatusID":"10","companyTruncated":"Liverpool School of Tropical Medicine \/ University of Bergen | University of Malawi | Makerere University | Indiana University | Global Health Uganda"},{"orgOrder":0,"company":"YolTech Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"YOLT-204","moa":"Fetal hemoglobin expression","graph1":"Genetic Disease","graph2":"Phase I","graph3":"YolTech Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"YolTech Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"YolTech Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"PF-05280602","moa":"FGF21","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Catalyst Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Catalyst Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Paradigm Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Pentosan Polysulfate Sodium","moa":"FGFR-2\/FGFR-3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Paradigm Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Paradigm Biopharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Paradigm Biopharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ribomic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Umedaptanib Pegol","moa":"FGFR-2\/FGFR-3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ribomic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ribomic \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ribomic \/ Undisclosed"},{"orgOrder":0,"company":"Ribomic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Umedaptanib Pegol","moa":"FGFR-2\/FGFR-3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ribomic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ribomic \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ribomic \/ Undisclosed"},{"orgOrder":0,"company":"Ribomic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Umedaptanib Pegol","moa":"FGFR-2\/FGFR-3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ribomic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ribomic \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ribomic \/ Undisclosed"},{"orgOrder":0,"company":"Ribomic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Umedaptanib Pegol","moa":"FGFR-2\/FGFR-3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ribomic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ribomic \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ribomic \/ Undisclosed"},{"orgOrder":0,"company":"Tyra Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"TYRA-300","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tyra Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Tyra Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Tyra Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Tyra Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"TYRA-300","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tyra Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tyra Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Tyra Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Tyra Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"TYRA-300","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tyra Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tyra Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Tyra Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Tyra Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"TYRA-300","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tyra Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tyra Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Tyra Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Tyra Biosciences","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Private Placement","leadProduct":"TYRA-300","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tyra Biosciences","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Tyra Biosciences \/ RA Capital Management","highestDevelopmentStatusID":"8","companyTruncated":"Tyra Biosciences \/ RA Capital Management"},{"orgOrder":0,"company":"Tyra Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"TYRA-300","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tyra Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tyra Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Tyra Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"University of Virginia","sponsor":"Octapharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Fibrinogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"University of Virginia","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"University of Virginia \/ Octapharma","highestDevelopmentStatusID":"11","companyTruncated":"University of Virginia \/ Octapharma"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Liminal BioSciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Liminal BioSciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Liminal BioSciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Liminal BioSciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Liminal BioSciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Kedrion","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2021","type":"Divestment","leadProduct":"Plasminogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Infusion","sponsorNew":"Liminal BioSciences \/ Kedrion","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Kedrion"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Burosumab","moa":"Fibroblast growth factor 23","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Kyowa Kirin \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Kyowa Kirin \/ Undisclosed"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Burosumab","moa":"Fibroblast growth factor 23","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Kyowa Kirin \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Kyowa Kirin \/ Undisclosed"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Burosumab","moa":"Fibroblast growth factor 23","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Kyowa Kirin \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Kyowa Kirin \/ Undisclosed"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Burosumab","moa":"Fibroblast growth factor 23","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Kyowa Kirin \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Kyowa Kirin \/ Undisclosed"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Burosumab","moa":"Fibroblast growth factor 23","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Kyowa Kirin \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Kyowa Kirin \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"QED Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"QED Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"QED Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"QED Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"QED Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"QED Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"QED Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"QED Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"QED Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"QED Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"QED Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"QED Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Licensing Agreement","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Kyowa Kirin","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Kyowa Kirin"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"QED Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"QED Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"QED Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"QED Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ISU ABXIS","sponsor":"Catalyst Biosciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"ISU304","moa":"Filgrastim receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ISU ABXIS","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ISU ABXIS \/ Catalyst Biosciences","highestDevelopmentStatusID":"6","companyTruncated":"ISU ABXIS \/ Catalyst Biosciences"},{"orgOrder":0,"company":"Belief BioMed","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Dalnacogene Ponparvovec","moa":"FIX gene","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Belief BioMed","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Belief BioMed \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Belief BioMed \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2024","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"CPRIT","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2024","type":"Funding","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ CPRIT","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ CPRIT"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2025","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2025","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2025","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2024","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Ladenburg Thalmann & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2024","type":"Private Placement","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Ladenburg Thalmann & Co","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Ladenburg Thalmann & Co"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Ladenburg Thalmann & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2024","type":"Public Offering","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Ladenburg Thalmann & Co","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Ladenburg Thalmann & Co"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2025","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Emtora Biosciences","sponsor":"Biodexa Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2025","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Emtora Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Emtora Biosciences \/ Biodexa Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Emtora Biosciences \/ Biodexa Pharmaceuticals"},{"orgOrder":0,"company":"Emtora Biosciences","sponsor":"Cancer Insight | Biodexa Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2020","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Emtora Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Emtora Biosciences \/ Cancer Insight | Biodexa Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Emtora Biosciences \/ Cancer Insight | Biodexa Pharmaceuticals"},{"orgOrder":0,"company":"Emtora Biosciences","sponsor":"CPRIT","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2025","type":"Funding","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Emtora Biosciences","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Capsule","sponsorNew":"Emtora Biosciences \/ CPRIT","highestDevelopmentStatusID":"10","companyTruncated":"Emtora Biosciences \/ CPRIT"},{"orgOrder":0,"company":"Emtora Biosciences","sponsor":"Biodexa Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2024","type":"Licensing Agreement","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Emtora Biosciences","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Capsule","sponsorNew":"Emtora Biosciences \/ Biodexa Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Emtora Biosciences \/ Biodexa Pharmaceuticals"},{"orgOrder":0,"company":"Emtora Biosciences","sponsor":"Cancer Prevention and Research Institute of Texas","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2022","type":"Funding","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Emtora Biosciences","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Capsule","sponsorNew":"Emtora Biosciences \/ Cancer Prevention and Research Institute of Texas","highestDevelopmentStatusID":"10","companyTruncated":"Emtora Biosciences \/ Cancer Prevention and Research Institute of Texas"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Petrichor Healthcare","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2023","type":"Series D Financing","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palvella Therapeutics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Petrichor Healthcare","highestDevelopmentStatusID":"10","companyTruncated":"Palvella Therapeutics \/ Petrichor Healthcare"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2023","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palvella Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Palvella Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2023","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palvella Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Palvella Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2022","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palvella Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Palvella Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Everolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NYU Langone Health \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NYU Langone Health \/ Undisclosed"},{"orgOrder":0,"company":"Assistance Publique \u2013 H\u00f4pitaux de Paris","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Everolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Assistance Publique \u2013 H\u00f4pitaux de Paris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"10","companyTruncated":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Assistance Publique \u2013 H\u00f4pitaux de Paris","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Everolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Assistance Publique \u2013 H\u00f4pitaux de Paris","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"8","companyTruncated":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Radboud University Medical Center","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Everolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Radboud University Medical Center","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Radboud University Medical Center \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"8","companyTruncated":"Radboud University Medical Center \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Jonsson Comprehensive Cancer Center","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Everolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Jonsson Comprehensive Cancer Center","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jonsson Comprehensive Cancer Center \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"8","companyTruncated":"Jonsson Comprehensive Cancer Center \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Novartis Pharmaceuticals Corporation | The Children's Tumor Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Everolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"NYU Langone Health \/ Novartis Pharmaceuticals Corporation | The Children's Tumor Foundation","highestDevelopmentStatusID":"8","companyTruncated":"NYU Langone Health \/ Novartis Pharmaceuticals Corporation | The Children's Tumor Foundation"},{"orgOrder":0,"company":"TransDerm, Inc.","sponsor":"Pachyonychia Congenita Project","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2014","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase I","graph3":"TransDerm, Inc.","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Cream","sponsorNew":"TransDerm, Inc. \/ Pachyonychia Congenita Project","highestDevelopmentStatusID":"6","companyTruncated":"TransDerm, Inc. \/ Pachyonychia Congenita Project"},{"orgOrder":0,"company":"Unity Health Toronto","sponsor":"National Institutes of Health | University of California, San Francisco","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Antibiotic","year":"2022","type":"Inapplicable","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Unity Health Toronto","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Unity Health Toronto \/ National Institutes of Health | University of California, San Francisco","highestDevelopmentStatusID":"8","companyTruncated":"Unity Health Toronto \/ National Institutes of Health | University of California, San Francisco"},{"orgOrder":0,"company":"Ligand Pharmaceuticals","sponsor":"Palvella Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2020","type":"Agreement","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ligand Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ligand Pharmaceuticals \/ Palvella Therapeutics","highestDevelopmentStatusID":"9","companyTruncated":"Ligand Pharmaceuticals \/ Palvella Therapeutics"},{"orgOrder":0,"company":"Unity Health Toronto","sponsor":"US Department of Defense","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Antibiotic","year":"2020","type":"Inapplicable","leadProduct":"Tacrolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Unity Health Toronto","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Unity Health Toronto \/ US Department of Defense","highestDevelopmentStatusID":"8","companyTruncated":"Unity Health Toronto \/ US Department of Defense"},{"orgOrder":0,"company":"AskBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AB-1003","moa":"FKRP","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Undisclosed"},{"orgOrder":0,"company":"AskBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AB-1003","moa":"FKRP","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Undisclosed"},{"orgOrder":0,"company":"AskBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AB-1003","moa":"FKRP","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Undisclosed"},{"orgOrder":0,"company":"Cure Rare","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"CRD-003","moa":"FKRP","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cure Rare","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cure Rare \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Cure Rare \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Veristat","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Veristat","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Veristat"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Public Offering","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17000000000000001,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Leerink Partners"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Public Offering","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0.17000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17000000000000001,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Leerink Partners"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Public Offering","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17000000000000001,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Leerink Partners"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Public Offering","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Guggenheim Securities","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Guggenheim Securities"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Public Offering","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Guggenheim Securities","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Guggenheim Securities"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Veristat","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Veristat","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Veristat"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Veristat","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Veristat","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Veristat"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Zafgen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Merger","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Zafgen","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Zafgen"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Nomlabofusp","moa":"Frataxin protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nanoform","sponsor":"Nacuity Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FINLAND","productType":"Miscellaneous","year":"2021","type":"Agreement","leadProduct":"N-Acetylcysteine Amide","moa":"Free radical","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nanoform","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Nanoform \/ Nacuity Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Nanoform \/ Nacuity Pharmaceuticals"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"N-Acetylcysteine Amide","moa":"Free radical","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nacuity Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Nacuity Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nacuity Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Foundation Fighting Blindness","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Series B Financing","leadProduct":"N-Acetylcysteine Amide","moa":"Free radical","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nacuity Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Tablet","sponsorNew":"Nacuity Pharmaceuticals \/ Foundation Fighting Blindness","highestDevelopmentStatusID":"7","companyTruncated":"Nacuity Pharmaceuticals \/ Foundation Fighting Blindness"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"N-Acetylcysteine Amide","moa":"Free radical","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nacuity Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Nacuity Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nacuity Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"ArmaGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"AGT-181","moa":"fusion proteins","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ArmaGen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ArmaGen \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ArmaGen \/ Undisclosed"},{"orgOrder":0,"company":"ArmaGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"AGT-181","moa":"fusion proteins","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ArmaGen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ArmaGen \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ArmaGen \/ Undisclosed"},{"orgOrder":0,"company":"ArmaGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2015","type":"Inapplicable","leadProduct":"AGT-181","moa":"fusion proteins","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ArmaGen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ArmaGen \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ArmaGen \/ Undisclosed"},{"orgOrder":0,"company":"ArmaGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Valanafusp Alfa","moa":"fusion proteins","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ArmaGen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ArmaGen \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ArmaGen \/ Undisclosed"},{"orgOrder":0,"company":"Cornell University","sponsor":"Lexeo Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Licensing Agreement","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Cornell University","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cornell University \/ Lexeo Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Cornell University \/ Lexeo Therapeutics"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Private Placement","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ J.P. Morgan"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Public Offering","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ J.P. Morgan"},{"orgOrder":0,"company":"Adverum Biotechnologies","sponsor":"Lexeo Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Adverum Biotechnologies","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Adverum Biotechnologies \/ Lexeo Therapeutics","highestDevelopmentStatusID":"5","companyTruncated":"Adverum Biotechnologies \/ Lexeo Therapeutics"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Private Placement","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ J.P. Morgan"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"D1 Capital Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Series B Financing","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ D1 Capital Partners","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ D1 Capital Partners"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SGT-212","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SGT-212","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"DT-216","moa":"FXN transcription","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Design Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Design Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"DT-216","moa":"FXN transcription","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Design Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Design Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"DT-216","moa":"FXN transcription","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Design Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Design Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"DT-216","moa":"FXN transcription","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Design Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Design Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"DT-216","moa":"FXN transcription","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Design Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Design Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"BEAM-301","moa":"GAA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Basmisanil","moa":"GABA(A) receptor alpha-5 (GABRA5)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"Ovid Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Gaboxadol","moa":"GABA-A receptor; agonist GABA site","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ovid Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Ovid Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ovid Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ovid Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Gaboxadol","moa":"GABA-A receptor; agonist GABA site","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ovid Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Ovid Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ovid Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Cowen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Public Offering","leadProduct":"Ganaxolone","moa":"GABA-A receptor; anion channel","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Marinus Pharmaceuticals","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Injection","sponsorNew":"Marinus Pharmaceuticals \/ Cowen","highestDevelopmentStatusID":"10","companyTruncated":"Marinus Pharmaceuticals \/ Cowen"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ganaxolone","moa":"GABA-A receptor; anion channel","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Marinus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Marinus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"University of California, Davis | U.S. Army Medical Research and Development Command","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Ganaxolone","moa":"GABA-A receptor; anion channel","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ University of California, Davis | U.S. Army Medical Research and Development Command","highestDevelopmentStatusID":"8","companyTruncated":"Marinus Pharmaceuticals \/ University of California, Davis | U.S. Army Medical Research and Development Command"},{"orgOrder":0,"company":"Children\u2019s Hospital Los Angeles","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2023","type":"Funding","leadProduct":"Lorazepam","moa":"||GABA-A receptor; anion channel","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Children\u2019s Hospital Los Angeles","amount2":0.01,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"Children\u2019s Hospital Los Angeles \/ National Institutes of Health","highestDevelopmentStatusID":"15","companyTruncated":"Children\u2019s Hospital Los Angeles \/ National Institutes of Health"},{"orgOrder":0,"company":"Aelis Farma","sponsor":"European Commission","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"AEF0217","moa":"GABAA-alpha-5 receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aelis Farma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Aelis Farma \/ European Commission","highestDevelopmentStatusID":"7","companyTruncated":"Aelis Farma \/ European Commission"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"RG1662","moa":"GABAA-alpha-5 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"RG1662","moa":"GABAA-alpha-5 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"RG1662","moa":"GABAA-alpha-5 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"Seaside Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Baclofen","moa":"GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Seaside Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Seaside Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Seaside Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seaside Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Baclofen","moa":"GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Seaside Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Seaside Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Seaside Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seaside Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Baclofen","moa":"GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Seaside Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Seaside Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Seaside Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seaside Therapeutics","sponsor":"Allos Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Acquisition","leadProduct":"Baclofen","moa":"GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Seaside Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Seaside Therapeutics \/ Allos Pharma","highestDevelopmentStatusID":"10","companyTruncated":"Seaside Therapeutics \/ Allos Pharma"},{"orgOrder":0,"company":"Pharnext","sponsor":"Alpha Blue Ocean","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2021","type":"Financing","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Solution","sponsorNew":"Pharnext \/ Alpha Blue Ocean","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Alpha Blue Ocean"},{"orgOrder":0,"company":"Pharnext","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Pharnext \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Undisclosed"},{"orgOrder":0,"company":"Pharnext","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Pharnext \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Undisclosed"},{"orgOrder":0,"company":"Pharnext","sponsor":"Neovacs","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Financing","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Solution","sponsorNew":"Pharnext \/ Neovacs","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Neovacs"},{"orgOrder":0,"company":"Pharnext","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Pharnext \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Undisclosed"},{"orgOrder":0,"company":"Pharnext","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Pharnext \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Undisclosed"},{"orgOrder":0,"company":"Pharnext","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Pharnext \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Undisclosed"},{"orgOrder":0,"company":"Pharnext","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Pharnext \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Undisclosed"},{"orgOrder":0,"company":"Pharnext","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Pharnext \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Undisclosed"},{"orgOrder":0,"company":"Charcot Marie Tooth Association","sponsor":"Pharnext","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Collaboration","leadProduct":"Baclofen","moa":"||GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Charcot Marie Tooth Association","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Charcot Marie Tooth Association \/ Pharnext","highestDevelopmentStatusID":"10","companyTruncated":"Charcot Marie Tooth Association \/ Pharnext"},{"orgOrder":0,"company":"Allos Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Baclofen","moa":"GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Allos Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Allos Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Allos Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Allos Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Baclofen","moa":"GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Allos Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Allos Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Allos Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Seaside Therapeutics","sponsor":"Allos Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Licensing Agreement","leadProduct":"Baclofen","moa":"GABA-B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Seaside Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Seaside Therapeutics \/ Allos Pharma","highestDevelopmentStatusID":"10","companyTruncated":"Seaside Therapeutics \/ Allos Pharma"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Perceptive Xontogeny Venture Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Series A Financing","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Perceptive Xontogeny Venture Fund","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Perceptive Xontogeny Venture Fund"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"PBKR03","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"PBKR03","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"PBKR03","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"University of Pennsylvania","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"PBKR03","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ University of Pennsylvania","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ University of Pennsylvania"},{"orgOrder":0,"company":"Azafaros","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Azafaros \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Undisclosed"},{"orgOrder":0,"company":"Azafaros","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Azafaros \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Azafaros \/ Undisclosed"},{"orgOrder":0,"company":"Azafaros","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Azafaros \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Azafaros \/ Undisclosed"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"GT-02329","moa":"GCase level","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gain Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AmideBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"AB-G023","moa":"GCGR","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"AmideBio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"AmideBio \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"AmideBio \/ Undisclosed"},{"orgOrder":0,"company":"AmideBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"AB-G023","moa":"GCGR","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"AmideBio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"AmideBio \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"AmideBio \/ Undisclosed"},{"orgOrder":0,"company":"AceLink Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"AL01211","moa":"GCS","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AceLink Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"AceLink Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AceLink Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AceLink Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"AL01211","moa":"GCS","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AceLink Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AceLink Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AceLink Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AceLink Therapeutics","sponsor":"Novotech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"AL01211","moa":"GCS","graph1":"Genetic Disease","graph2":"Phase I","graph3":"AceLink Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AceLink Therapeutics \/ Novotech","highestDevelopmentStatusID":"6","companyTruncated":"AceLink Therapeutics \/ Novotech"},{"orgOrder":0,"company":"AceLink Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"AL01211","moa":"GCS","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AceLink Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AceLink Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AceLink Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AceLink Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"AL01211","moa":"GCS","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AceLink Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AceLink Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AceLink Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AceLink Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"AL01211","moa":"GCS","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AceLink Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AceLink Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AceLink Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AceLink Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"AL1211","moa":"GCS","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AceLink Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AceLink Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AceLink Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"CSPC Baike","sponsor":"Beijing Friendship Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Pegfilgrastim","moa":"Granulocyte colony stimulating factor receptor","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"CSPC Baike","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSPC Baike \/ Beijing Friendship Hospital","highestDevelopmentStatusID":"1","companyTruncated":"CSPC Baike \/ Beijing Friendship Hospital"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-102","moa":"Gene","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Gamut Therapeutics","sponsor":"SparingVision","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Acquisition","leadProduct":"SPVN20","moa":"GIRK","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gamut Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Gamut Therapeutics \/ SparingVision","highestDevelopmentStatusID":"4","companyTruncated":"Gamut Therapeutics \/ SparingVision"},{"orgOrder":0,"company":"SparingVision","sponsor":"Tenpoint Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Divestment","leadProduct":"SPVN20","moa":"GIRK","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Tenpoint Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"SparingVision \/ Tenpoint Therapeutics"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Hanmi Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"alpha-galactosidase A","moa":"Globotriaosylceramide","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GC Biopharma \/ Hanmi Pharmaceutical","highestDevelopmentStatusID":"5","companyTruncated":"GC Biopharma \/ Hanmi Pharmaceutical"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Agalsidase Alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Agalsidase Alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2011","type":"Inapplicable","leadProduct":"Agalsidase Alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Agalsidase Alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Agalsidase Alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2011","type":"Inapplicable","leadProduct":"Agalsidase Alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2011","type":"Inapplicable","leadProduct":"Agalsidase Alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"ISU ABXIS","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ISU ABXIS","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ISU ABXIS \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"ISU ABXIS \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Sumitomo Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2022","type":"Agreement","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Sumitomo Pharma","highestDevelopmentStatusID":"15","companyTruncated":"JCR Pharmaceuticals \/ Sumitomo Pharma"},{"orgOrder":0,"company":"ISU ABXIS","sponsor":"Petrovax","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2022","type":"Licensing Agreement","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ISU ABXIS","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ISU ABXIS \/ Petrovax","highestDevelopmentStatusID":"15","companyTruncated":"ISU ABXIS \/ Petrovax"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Enzyme","year":"2012","type":"Inapplicable","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Enzyme","year":"2019","type":"Inapplicable","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Petrovax","sponsor":"ISU ABXIS","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Petrovax","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Petrovax \/ ISU ABXIS","highestDevelopmentStatusID":"15","companyTruncated":"Petrovax \/ ISU ABXIS"},{"orgOrder":0,"company":"Mabxience","sponsor":"Biosidus","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Enzyme","year":"2024","type":"Agreement","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Mabxience","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mabxience \/ Biosidus","highestDevelopmentStatusID":"10","companyTruncated":"Mabxience \/ Biosidus"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Bio Sidus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ARGENTINA","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Sidus","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bio Sidus \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Sidus \/ Undisclosed"},{"orgOrder":0,"company":"Bio Sidus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ARGENTINA","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Sidus","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bio Sidus \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Sidus \/ Undisclosed"},{"orgOrder":0,"company":"Biosidus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ARGENTINA","productType":"Enzyme","year":"2025","type":"Inapplicable","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biosidus","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Biosidus \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biosidus \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2018","type":"Inapplicable","leadProduct":"Pegunigalsidase alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chiesi Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2018","type":"Inapplicable","leadProduct":"Pegunigalsidase alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chiesi Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Icon Plc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Pegunigalsidase alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chiesi Group \/ Icon Plc","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Icon Plc"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Icon Plc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Pegunigalsidase alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chiesi Group \/ Icon Plc","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Icon Plc"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegunigalsidase alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chiesi Group \/ Chiesi Group","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Chiesi Group"},{"orgOrder":0,"company":"Protalix","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2017","type":"Inapplicable","leadProduct":"Pegunigalsidase alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Protalix","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix \/ Chiesi Group","highestDevelopmentStatusID":"10","companyTruncated":"Protalix \/ Chiesi Group"},{"orgOrder":0,"company":"Protalix","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2016","type":"Inapplicable","leadProduct":"Pegunigalsidase alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Protalix","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Protalix \/ Chiesi Group","highestDevelopmentStatusID":"10","companyTruncated":"Protalix \/ Chiesi Group"},{"orgOrder":0,"company":"Protalix","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2016","type":"Inapplicable","leadProduct":"Pegunigalsidase alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Protalix","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Protalix \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Protalix \/ Undisclosed"},{"orgOrder":0,"company":"Protalix","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2017","type":"Inapplicable","leadProduct":"Pegunigalsidase alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Protalix","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Protalix \/ Chiesi Group","highestDevelopmentStatusID":"10","companyTruncated":"Protalix \/ Chiesi Group"},{"orgOrder":0,"company":"Protalix","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2012","type":"Inapplicable","leadProduct":"Pegunigalsidase alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Protalix","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Protalix \/ Chiesi Group","highestDevelopmentStatusID":"10","companyTruncated":"Protalix \/ Chiesi Group"},{"orgOrder":0,"company":"Protalix","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2013","type":"Inapplicable","leadProduct":"Pegunigalsidase alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Protalix","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Protalix \/ Chiesi Group","highestDevelopmentStatusID":"10","companyTruncated":"Protalix \/ Chiesi Group"},{"orgOrder":0,"company":"Immunoforge","sponsor":"PhaseBio Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Froniglutide","moa":"GLP-1 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Immunoforge","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Immunoforge \/ PhaseBio Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Immunoforge \/ PhaseBio Pharmaceuticals"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Hanmi Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"HM15421","moa":"GLP-1R","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GC Biopharma \/ Hanmi Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"GC Biopharma \/ Hanmi Pharmaceutical"},{"orgOrder":0,"company":"Hanmi Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"HM15136","moa":"GLP-1R\/GIPR\/GCGR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Hanmi Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Hanmi Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Hanmi Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2018","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zealand Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2019","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zealand Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2019","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zealand Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2024","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zealand Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Danske Bank","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2023","type":"Private Placement","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0.22,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.22,"dosageForm":"Injection","sponsorNew":"Zealand Pharma \/ Danske Bank","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Danske Bank"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zealand Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zealand Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2023","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zealand Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2023","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zealand Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zealand Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Oberland Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2021","type":"Financing","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20000000000000001,"dosageForm":"Injection","sponsorNew":"Zealand Pharma \/ Oberland Capital Management","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Oberland Capital Management"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2023","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zealand Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Undisclosed"},{"orgOrder":0,"company":"DEKA Research & Development","sponsor":"Zealand Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2021","type":"Collaboration","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"DEKA Research & Development","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"DEKA Research & Development \/ Zealand Pharma","highestDevelopmentStatusID":"10","companyTruncated":"DEKA Research & Development \/ Zealand Pharma"},{"orgOrder":0,"company":"Xeris Pharmaceuticals","sponsor":"SGS Life Sciences | Integrated Medical Development","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Glucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xeris Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Xeris Pharmaceuticals \/ SGS Life Sciences | Integrated Medical Development","highestDevelopmentStatusID":"10","companyTruncated":"Xeris Pharmaceuticals \/ SGS Life Sciences | Integrated Medical Development"},{"orgOrder":0,"company":"Xeris Pharmaceuticals","sponsor":"Empiristat","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Glucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xeris Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Xeris Pharmaceuticals \/ Empiristat","highestDevelopmentStatusID":"10","companyTruncated":"Xeris Pharmaceuticals \/ Empiristat"},{"orgOrder":0,"company":"AmideBio","sponsor":"NIDDK","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Funding","leadProduct":"Glucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AmideBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AmideBio \/ NIDDK","highestDevelopmentStatusID":"4","companyTruncated":"AmideBio \/ NIDDK"},{"orgOrder":0,"company":"Xeris Pharmaceuticals","sponsor":"National Institute of Diabetes and Digestive and Kidney Diseases","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"Glucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xeris Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Xeris Pharmaceuticals \/ National Institute of Diabetes and Digestive and Kidney Diseases","highestDevelopmentStatusID":"10","companyTruncated":"Xeris Pharmaceuticals \/ National Institute of Diabetes and Digestive and Kidney Diseases"},{"orgOrder":0,"company":"Xeris Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Glucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xeris Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Xeris Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xeris Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Children's Hospital of Philadelphia","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Avexitide","moa":"Glucagon-like peptide 1 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eiger BioPharmaceuticals \/ Children's Hospital of Philadelphia","highestDevelopmentStatusID":"8","companyTruncated":"Eiger BioPharmaceuticals \/ Children's Hospital of Philadelphia"},{"orgOrder":0,"company":"Tracey McLaughlin","sponsor":"Eiger BioPharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Avexitide","moa":"Glucagon-like peptide 1 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tracey McLaughlin","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tracey McLaughlin \/ Eiger BioPharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Tracey McLaughlin \/ Eiger BioPharmaceuticals"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"CAN103","moa":"Glucocerebrosidase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"WuXi Biologics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"glucocerebrosidase","moa":"Glucocerebrosidase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ WuXi Biologics","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ WuXi Biologics"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"glucocerebrosidase","moa":"Glucocerebrosidase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"glucocerebrosidase","moa":"Glucocerebrosidase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Peking Union Medical College Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"glucocerebrosidase","moa":"Glucocerebrosidase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Peking Union Medical College Hospital","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ Peking Union Medical College Hospital"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"glucocerebrosidase","moa":"Glucocerebrosidase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"ISU ABXIS","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Abcertin","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ISU ABXIS","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ISU ABXIS \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ISU ABXIS \/ Undisclosed"},{"orgOrder":0,"company":"ISU ABXIS","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Imiglucerase","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ISU ABXIS","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ISU ABXIS \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"ISU ABXIS \/ Undisclosed"},{"orgOrder":0,"company":"ISU ABXIS","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Imiglucerase","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ISU ABXIS","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ISU ABXIS \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"ISU ABXIS \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Imiglucerase","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Protalix","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2014","type":"Inapplicable","leadProduct":"PRX-112","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Protalix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Protalix \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Protalix \/ Undisclosed"},{"orgOrder":0,"company":"Protalix","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2012","type":"Inapplicable","leadProduct":"PRX-112","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Protalix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Protalix \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Protalix \/ Undisclosed"},{"orgOrder":0,"company":"Shaare Zedek Medical Center","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Taliglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Shaare Zedek Medical Center","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Shaare Zedek Medical Center \/ Pfizer Inc","highestDevelopmentStatusID":"1","companyTruncated":"Shaare Zedek Medical Center \/ Pfizer Inc"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Taliglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2017","type":"Inapplicable","leadProduct":"Taliglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2011","type":"Inapplicable","leadProduct":"Taliglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2011","type":"Inapplicable","leadProduct":"Taliglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Velaglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2015","type":"Inapplicable","leadProduct":"Velaglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2012","type":"Inapplicable","leadProduct":"Velaglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Quintiles Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2013","type":"Inapplicable","leadProduct":"Velaglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"SHIRE PLC \/ Quintiles Inc","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Quintiles Inc"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2012","type":"Inapplicable","leadProduct":"Velaglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"Shaare Zedek Medical Center","sponsor":"SHIRE PLC","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2019","type":"Inapplicable","leadProduct":"Velaglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Shaare Zedek Medical Center","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Shaare Zedek Medical Center \/ SHIRE PLC","highestDevelopmentStatusID":"11","companyTruncated":"Shaare Zedek Medical Center \/ SHIRE PLC"},{"orgOrder":0,"company":"Grace Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Steroid","year":"2022","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Grace Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"Grace Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Grace Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Grace Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Steroid","year":"2022","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Grace Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"Grace Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Grace Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aurobindo Pharma Limited","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Deflazacort","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Aurobindo Pharma Limited","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Aurobindo Pharma Limited \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Aurobindo Pharma Limited \/ Undisclosed"},{"orgOrder":0,"company":"Zydus Lifesciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Steroid","year":"2025","type":"Inapplicable","leadProduct":"Deflazacort","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Zydus Lifesciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Zydus Lifesciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Zydus Lifesciences \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2018","type":"Inapplicable","leadProduct":"Deflazacort","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2018","type":"Inapplicable","leadProduct":"Deflazacort","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2014","type":"Inapplicable","leadProduct":"Deflazacort","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Deflazacort","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2014","type":"Inapplicable","leadProduct":"Deflazacort","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Parexel | Dohmen Life Science Services","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2015","type":"Inapplicable","leadProduct":"Deflazacort","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Parexel | Dohmen Life Science Services","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Parexel | Dohmen Life Science Services"},{"orgOrder":0,"company":"Cranbury Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Deflazacort","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Cranbury Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cranbury Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Cranbury Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Barbara Ann Karmanos Cancer Institute","sponsor":"National Cancer Institute | Bristol Myers Squibb | Multiple Myeloma Research Consortium","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2017","type":"Inapplicable","leadProduct":"Dexamethasone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Barbara Ann Karmanos Cancer Institute","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Barbara Ann Karmanos Cancer Institute \/ National Cancer Institute | Bristol Myers Squibb | Multiple Myeloma Research Consortium","highestDevelopmentStatusID":"8","companyTruncated":"Barbara Ann Karmanos Cancer Institute \/ National Cancer Institute | Bristol Myers Squibb | Multiple Myeloma Research Consortium"},{"orgOrder":0,"company":"EryDel","sponsor":"Quince Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Steroid","year":"2023","type":"Acquisition","leadProduct":"Dexamethasone Sodium Phosphate","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"EryDel","amount2":0.48999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.48999999999999999,"dosageForm":"Infusion","sponsorNew":"EryDel \/ Quince Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"EryDel \/ Quince Therapeutics"},{"orgOrder":0,"company":"EryDel","sponsor":"Quince Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Steroid","year":"2023","type":"Acquisition","leadProduct":"Dexamethasone Sodium Phosphate","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"EryDel","amount2":0.48999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.48999999999999999,"dosageForm":"Infusion","sponsorNew":"EryDel \/ Quince Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"EryDel \/ Quince Therapeutics"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Biotrial","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Dexamethasone Sodium Phosphate","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Quince Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Quince Therapeutics \/ Biotrial","highestDevelopmentStatusID":"10","companyTruncated":"Quince Therapeutics \/ Biotrial"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Biotrial","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Dexamethasone Sodium Phosphate","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Quince Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Quince Therapeutics \/ Biotrial","highestDevelopmentStatusID":"10","companyTruncated":"Quince Therapeutics \/ Biotrial"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2018","type":"Inapplicable","leadProduct":"Dexamethasone Sodium Phosphate","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Quince Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Quince Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Quince Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Dexamethasone Sodium Phosphate","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Quince Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Quince Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Quince Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Dexamethasone Sodium Phosphate","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Quince Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Quince Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Quince Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2023","type":"Inapplicable","leadProduct":"Dexamethasone Sodium Phosphate","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Quince Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Quince Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Quince Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Nantahala Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2025","type":"Private Placement","leadProduct":"Dexamethasone Sodium Phosphate","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Quince Therapeutics","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Undisclosed","sponsorNew":"Quince Therapeutics \/ Nantahala Capital","highestDevelopmentStatusID":"10","companyTruncated":"Quince Therapeutics \/ Nantahala Capital"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Citrine Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Steroid","year":"2021","type":"Partnership","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Diurnal Group","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Modified Release","sponsorNew":"Diurnal Group \/ Citrine Medicine","highestDevelopmentStatusID":"15","companyTruncated":"Diurnal Group \/ Citrine Medicine"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Diurnal Group","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Modified Release","sponsorNew":"Diurnal Group \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Diurnal Group \/ Undisclosed"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Citrine Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Steroid","year":"2021","type":"Licensing Agreement","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Diurnal Group","amount2":0.01,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Capsule, Modified Release","sponsorNew":"Diurnal Group \/ Citrine Medicine","highestDevelopmentStatusID":"15","companyTruncated":"Diurnal Group \/ Citrine Medicine"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Diurnal Group","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Modified Release","sponsorNew":"Diurnal Group \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Diurnal Group \/ Undisclosed"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Diurnal Group","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Modified Release","sponsorNew":"Diurnal Group \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Diurnal Group \/ Undisclosed"},{"orgOrder":0,"company":"EffRx Pharmaceuticals","sponsor":"Diurnal Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Steroid","year":"2022","type":"Licensing Agreement","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"EffRx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Modified Release","sponsorNew":"EffRx Pharmaceuticals \/ Diurnal Group","highestDevelopmentStatusID":"15","companyTruncated":"EffRx Pharmaceuticals \/ Diurnal Group"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Diurnal Group","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Modified Release","sponsorNew":"Diurnal Group \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Diurnal Group \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2022","type":"Inapplicable","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"National Institutes of Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2018","type":"Inapplicable","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Modified Release","sponsorNew":"Neurocrine Biosciences \/ National Institutes of Health","highestDevelopmentStatusID":"10","companyTruncated":"Neurocrine Biosciences \/ National Institutes of Health"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2016","type":"Inapplicable","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2017","type":"Inapplicable","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Simbec Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2017","type":"Inapplicable","leadProduct":"Hydrocortisone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurocrine Biosciences \/ Simbec Research","highestDevelopmentStatusID":"10","companyTruncated":"Neurocrine Biosciences \/ Simbec Research"},{"orgOrder":0,"company":"University Medical Center Groningen","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"6,6-2H2 Glucose","moa":"Glucose transporter","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"University Medical Center Groningen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pill","sponsorNew":"University Medical Center Groningen \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"University Medical Center Groningen \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"DTX401","moa":"Glucose-6-phosphatase (G6PC)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"DTX401","moa":"Glucose-6-phosphatase (G6PC)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"DTX401","moa":"||Glucose-6-phosphatase (G6PC)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"mRNA-3745","moa":"Glucose-6-phosphatase catalytic subunit 1 messenger RNA (G6PC1 mRNA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Moderna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"mRNA-3745","moa":"Glucose-6-phosphatase catalytic subunit 1 messenger RNA (G6PC1 mRNA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Moderna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Inserm","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"GLUT1 DS","moa":"GLUT1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inserm","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inserm \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Inserm \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Cameroon Baptist Convention Health","sponsor":"Carolinas Medical Center | Muhimbili National Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CAMEROON","productType":"Controlled Substance","year":"2015","type":"Inapplicable","leadProduct":"Ketamine Hydrochloride","moa":"Glutamate [NMDA] receptor","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Cameroon Baptist Convention Health","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cameroon Baptist Convention Health \/ Carolinas Medical Center | Muhimbili National Hospital","highestDevelopmentStatusID":"1","companyTruncated":"Cameroon Baptist Convention Health \/ Carolinas Medical Center | Muhimbili National Hospital"},{"orgOrder":0,"company":"University of Colorado, Denver","sponsor":"Forest Laboratories","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Memantine Hydrochloride","moa":"Glutamate [NMDA] receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"University of Colorado, Denver","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Colorado, Denver \/ Forest Laboratories","highestDevelopmentStatusID":"11","companyTruncated":"University of Colorado, Denver \/ Forest Laboratories"},{"orgOrder":0,"company":"University of British Columbia","sponsor":"Huntington Society of Canada | Huntington Study Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Memantine Hydrochloride","moa":"Glutamate [NMDA] receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of British Columbia","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"University of British Columbia \/ Huntington Society of Canada | Huntington Study Group","highestDevelopmentStatusID":"8","companyTruncated":"University of British Columbia \/ Huntington Society of Canada | Huntington Study Group"},{"orgOrder":0,"company":"GRIN Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Radiprodil","moa":"Glutamate NMDA receptor; GRIN1\/GRIN2B","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GRIN Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"GRIN Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"GRIN Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GRIN Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Radiprodil","moa":"Glutamate NMDA receptor; GRIN1\/GRIN2B","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GRIN Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"GRIN Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"GRIN Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"National Jewish Health","sponsor":"University of Alabama at Birmingham | Children's Hospital of Philadelphia | Johns Hopkins University | Amgen Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"National Jewish Health","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"National Jewish Health \/ University of Alabama at Birmingham | Children's Hospital of Philadelphia | Johns Hopkins University | Amgen Inc","highestDevelopmentStatusID":"7","companyTruncated":"National Jewish Health \/ University of Alabama at Birmingham | Children's Hospital of Philadelphia | Johns Hopkins University | Amgen Inc"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"The University of Pittsburgh School of Medicine","sponsor":"Amgen Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase I","graph3":"The University of Pittsburgh School of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"The University of Pittsburgh School of Medicine \/ Amgen Inc","highestDevelopmentStatusID":"6","companyTruncated":"The University of Pittsburgh School of Medicine \/ Amgen Inc"},{"orgOrder":0,"company":"SYSNAV Healthcare","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"SYSNAV Healthcare","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"SYSNAV Healthcare \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"SYSNAV Healthcare \/ Undisclosed"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Acer Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Licensing Agreement","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Relief Therapeutics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Suspension","sponsorNew":"Relief Therapeutics \/ Acer Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Relief Therapeutics \/ Acer Therapeutics"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Acer Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Relief Therapeutics \/ Acer Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Relief Therapeutics \/ Acer Therapeutics"},{"orgOrder":0,"company":"Duchesnay","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Duchesnay","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Duchesnay \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Duchesnay \/ Undisclosed"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Perceptive Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Financing","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Zevra Therapeutics \/ Perceptive Advisors","highestDevelopmentStatusID":"15","companyTruncated":"Zevra Therapeutics \/ Perceptive Advisors"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Relief Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Relief Therapeutics"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Marathon Asset Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Financing","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Marathon Asset Management","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Marathon Asset Management"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Relief Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Relief Therapeutics"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Relief Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Relief Therapeutics"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Christopher Schelling","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Financing","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Christopher Schelling","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Christopher Schelling"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Relief Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Relief Therapeutics"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Relief Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Relief Therapeutics"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Zevra Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Acquisition","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Zevra Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Zevra Therapeutics"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Zevra Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Acquisition","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Zevra Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Zevra Therapeutics"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Collaboration","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Relief Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Relief Therapeutics"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Collaboration","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Relief Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Relief Therapeutics"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Acer Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Acer Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Acer Therapeutics \/ Relief Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Acer Therapeutics \/ Relief Therapeutics"},{"orgOrder":0,"company":"Academisch Medisch Centrum - Universiteit van Amsterdam","sponsor":"Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation | Haga Hospital | University Medical Center Groningen | ZonMw | Fonds NutsOhra | Stichting Janivo | CHU Brugmann, Brussels | GALZU INSTITUTE OF RESEARC","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Acetylcysteine","moa":"Glutathione synthetase; Cystine\/glutamate transporter; NAPQI (N-acetyl-p-benzoquinone imine); Aminoacylase-1; Inhibitor of nuclear factor kappa-B kinase subunit beta; Inhibitor of nuclear factor kappa-B kinase subunit alpha; Glutamate receptor ionotropic, NMDA 2B; Glutamate receptor ionotropic, NMDA 1; Glutamate receptor ionotropic, NMDA 2A; Glutamate receptor ionotropic, NMDA 2D; Glutamate receptor ionotropic, NMDA 3A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Academisch Medisch Centrum - Universiteit van Amsterdam","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Academisch Medisch Centrum - Universiteit van Amsterdam \/ Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation | Haga Hospital | University Medical Center Groningen | ZonMw | Fonds NutsOhra | Stichting Janivo | CHU Brugmann, Brussels | GALZU INSTITUTE OF RESEARC","highestDevelopmentStatusID":"10","companyTruncated":"Academisch Medisch Centrum - Universiteit van Amsterdam \/ Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation | Haga Hospital | University Medical Center Groningen | ZonMw | Fonds NutsOhra | Stichting Janivo | CHU Brugmann, Brussels | GALZU INSTITUTE OF RESEARC"},{"orgOrder":0,"company":"Johns Hopkins University","sponsor":"Zambon Switzerland","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Acetylcysteine","moa":"Glutathione synthetase; Cystine\/glutamate transporter; NAPQI (N-acetyl-p-benzoquinone imine); Aminoacylase-1; Inhibitor of nuclear factor kappa-B kinase subunit beta; Inhibitor of nuclear factor kappa-B kinase subunit alpha; Glutamate receptor ionotropic, NMDA 2B; Glutamate receptor ionotropic, NMDA 1; Glutamate receptor ionotropic, NMDA 2A; Glutamate receptor ionotropic, NMDA 2D; Glutamate receptor ionotropic, NMDA 3A","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Johns Hopkins University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Effervescent","sponsorNew":"Johns Hopkins University \/ Zambon Switzerland","highestDevelopmentStatusID":"6","companyTruncated":"Johns Hopkins University \/ Zambon Switzerland"},{"orgOrder":0,"company":"Western Sydney Local Health District","sponsor":"Deakin University | Monash University | Royal Perth Hospital | University of Queensland | University of Melbourne","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Acetylcysteine","moa":"Glutathione synthetase; Cystine\/glutamate transporter; NAPQI (N-acetyl-p-benzoquinone imine); Aminoacylase-1; Inhibitor of nuclear factor kappa-B kinase subunit beta; Inhibitor of nuclear factor kappa-B kinase subunit alpha; Glutamate receptor ionotropic, NMDA 2B; Glutamate receptor ionotropic, NMDA 1; Glutamate receptor ionotropic, NMDA 2A; Glutamate receptor ionotropic, NMDA 2D; Glutamate receptor ionotropic, NMDA 3A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Western Sydney Local Health District","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Western Sydney Local Health District \/ Deakin University | Monash University | Royal Perth Hospital | University of Queensland | University of Melbourne","highestDevelopmentStatusID":"8","companyTruncated":"Western Sydney Local Health District \/ Deakin University | Monash University | Royal Perth Hospital | University of Queensland | University of Melbourne"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Disc medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Licensing Agreement","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"F. Hoffmann-La Roche","amount2":0.20000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20000000000000001,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Disc medicine","highestDevelopmentStatusID":"4","companyTruncated":"F. Hoffmann-La Roche \/ Disc medicine"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Morgan Stanley","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Morgan Stanley"},{"orgOrder":0,"company":"Disc medicine","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Hercules Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Financing","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Hercules Capital","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ Hercules Capital"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ Undisclosed"},{"orgOrder":0,"company":"Disc medicine","sponsor":"OrbiMed Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Series B Financing","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ OrbiMed Advisors","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ OrbiMed Advisors"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.14000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Morgan Stanley","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Morgan Stanley"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.16,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Morgan Stanley","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Morgan Stanley"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ Undisclosed"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0.23000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ Jefferies"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0.23000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ Jefferies"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Bain Capital Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Private Placement","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Bain Capital Life Sciences","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Bain Capital Life Sciences"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Gemini Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Merger","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.17999999999999999,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17999999999999999,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Gemini Therapeutics","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Gemini Therapeutics"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Undisclosed"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Undisclosed"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Disc medicine \/ Undisclosed"},{"orgOrder":0,"company":"Disc medicine","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Collaboration","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ National Institutes of Health","highestDevelopmentStatusID":"7","companyTruncated":"Disc medicine \/ National Institutes of Health"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"DISC-1459","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Disc medicine \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ Undisclosed"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"DISC-1459","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Disc medicine \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ Undisclosed"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"DISC-1459","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Disc medicine \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Odiparcil","moa":"Glycosaminoglycans","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inventiva Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Inventiva Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Inventiva Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Odiparcil","moa":"Glycosaminoglycans","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inventiva Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Inventiva Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Inventiva Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Odiparcil","moa":"Glycosaminoglycans","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inventiva Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Inventiva Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Inventiva Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Odiparcil","moa":"Glycosaminoglycans","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inventiva Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Inventiva Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Inventiva Pharma \/ Undisclosed"},{"orgOrder":0,"company":"ML Bio Solutions","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"BBP-418","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ML Bio Solutions","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Sachet","sponsorNew":"ML Bio Solutions \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"ML Bio Solutions \/ Undisclosed"},{"orgOrder":0,"company":"Bridge Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bridge Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Sachet","sponsorNew":"Bridge Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bridge Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ML Bio Solutions","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ML Bio Solutions","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"ML Bio Solutions \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"ML Bio Solutions \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Sachet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Sachet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Sachet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase I","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Fezagepras","moa":"G-protein coupled receptor 84 | Free fatty acid receptor 1||GPR84","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Liminal BioSciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Liminal BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"Asklepion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GREECE","productType":"Amino Acid","year":"2021","type":"Inapplicable","leadProduct":"Citrulline","moa":"G-protein coupled receptor GPCR33 (GPRC6A)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Asklepion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Asklepion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Asklepion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Asklepion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GREECE","productType":"Amino Acid","year":"2025","type":"Inapplicable","leadProduct":"Citrulline","moa":"G-protein coupled receptor GPCR33 (GPRC6A)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Asklepion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Asklepion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Asklepion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Theranexus","sponsor":"Exelixis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2024","type":"Licensing Agreement","leadProduct":"TX01","moa":"Granulocyte colony stimulating factor receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Theranexus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Theranexus \/ Exelixis","highestDevelopmentStatusID":"4","companyTruncated":"Theranexus \/ Exelixis"},{"orgOrder":0,"company":"TJ Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Plonmarlimab","moa":"Granulocyte-macrophage colony-stimulating factor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"TJ Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"TJ Biopharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"TJ Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Partner Therapeutics","sponsor":"University of Colorado","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Sargramostim","moa":"Granulocyte-macrophage colony-stimulating factor receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Partner Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Partner Therapeutics \/ University of Colorado","highestDevelopmentStatusID":"4","companyTruncated":"Partner Therapeutics \/ University of Colorado"},{"orgOrder":0,"company":"LG Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Hormone","year":"2017","type":"Inapplicable","leadProduct":"Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"LG Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"LG Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"LG Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"LG Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Hormone","year":"2014","type":"Inapplicable","leadProduct":"Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"LG Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"LG Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"LG Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"G\u00f6teborg University","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Hormone","year":"2016","type":"Inapplicable","leadProduct":"Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"G\u00f6teborg University","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"G\u00f6teborg University \/ Pfizer Inc","highestDevelopmentStatusID":"9","companyTruncated":"G\u00f6teborg University \/ Pfizer Inc"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2012","type":"Inapplicable","leadProduct":"Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2012","type":"Inapplicable","leadProduct":"Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2012","type":"Inapplicable","leadProduct":"Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Merck Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Hormone","year":"2010","type":"Inapplicable","leadProduct":"Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Merck Group","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Merck Group \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Merck Group \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2013","type":"Inapplicable","leadProduct":"Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2011","type":"Inapplicable","leadProduct":"Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2012","type":"Inapplicable","leadProduct":"Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2012","type":"Inapplicable","leadProduct":"Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2021","type":"Inapplicable","leadProduct":"Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Corporacion Parc Tauli","sponsor":"Parc de Salut Mar","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Hormone","year":"2018","type":"Inapplicable","leadProduct":"Growth Hormone","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Corporacion Parc Tauli","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Corporacion Parc Tauli \/ Parc de Salut Mar","highestDevelopmentStatusID":"11","companyTruncated":"Corporacion Parc Tauli \/ Parc de Salut Mar"},{"orgOrder":0,"company":"GeneScience Pharmaceuticals Co Ltd","sponsor":"Tongji Hospital | The First Hospital of Jilin University | Affiliated Hospital of Jiangnan University | The First Affiliated Hospital with Nanjing Medical University | Shanghai Children's Hospital | The Children's Hospital of Zhejiang University School of","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Hormone","year":"2017","type":"Inapplicable","leadProduct":"Pegylated Recombinant Human Growth Hormone","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"GeneScience Pharmaceuticals Co Ltd","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneScience Pharmaceuticals Co Ltd \/ Tongji Hospital | The First Hospital of Jilin University | Affiliated Hospital of Jiangnan University | The First Affiliated Hospital with Nanjing Medical University | Shanghai Children's Hospital | The Children's Hospital of Zhejiang University School of","highestDevelopmentStatusID":"8","companyTruncated":"GeneScience Pharmaceuticals Co Ltd \/ Tongji Hospital | The First Hospital of Jilin University | Affiliated Hospital of Jiangnan University | The First Affiliated Hospital with Nanjing Medical University | Shanghai Children's Hospital | The Children's Hospital of Zhejiang University School of"},{"orgOrder":0,"company":"GeneScience Pharmaceuticals Co Ltd","sponsor":"Tongji Hospital | The First Hospital of Jilin University | Affiliated Hospital of Jiangnan University | The First Affiliated Hospital with Nanjing Medical University | Shanghai Children's Hospital | The Children's Hospital of Zhejiang University School of","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Hormone","year":"2017","type":"Inapplicable","leadProduct":"Pegylated Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"GeneScience Pharmaceuticals Co Ltd","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GeneScience Pharmaceuticals Co Ltd \/ Tongji Hospital | The First Hospital of Jilin University | Affiliated Hospital of Jiangnan University | The First Affiliated Hospital with Nanjing Medical University | Shanghai Children's Hospital | The Children's Hospital of Zhejiang University School of","highestDevelopmentStatusID":"8","companyTruncated":"GeneScience Pharmaceuticals Co Ltd \/ Tongji Hospital | The First Hospital of Jilin University | Affiliated Hospital of Jiangnan University | The First Affiliated Hospital with Nanjing Medical University | Shanghai Children's Hospital | The Children's Hospital of Zhejiang University School of"},{"orgOrder":0,"company":"GeneScience Pharmaceuticals Co Ltd","sponsor":"Children's Hospital of Fudan University | Tongji Hospital | Children's Hospital of Nanjing Medical University | Jiangxi Province Children's Hospital | Chengdu Women's and Children's Central Hospital | Shandong Provincial Hospital | Shengjing Hospital | Sh","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Recombinant Human Growth Hormone","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"GeneScience Pharmaceuticals Co Ltd","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneScience Pharmaceuticals Co Ltd \/ Children's Hospital of Fudan University | Tongji Hospital | Children's Hospital of Nanjing Medical University | Jiangxi Province Children's Hospital | Chengdu Women's and Children's Central Hospital | Shandong Provincial Hospital | Shengjing Hospital | Sh","highestDevelopmentStatusID":"11","companyTruncated":"GeneScience Pharmaceuticals Co Ltd \/ Children's Hospital of Fudan University | Tongji Hospital | Children's Hospital of Nanjing Medical University | Jiangxi Province Children's Hospital | Chengdu Women's and Children's Central Hospital | Shandong Provincial Hospital | Shengjing Hospital | Sh"},{"orgOrder":0,"company":"GeneScience Pharmaceuticals Co Ltd","sponsor":"Children's Hospital of Fudan University | Beijing Children's Hospital | Shanghai Children's Hospital | Xin Hua Hospital, Shanghai Jiaotong University School of Medicine | The Children's Hospital of Zhejiang University School of Medicine | Children's Hospi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Hormone","year":"2018","type":"Inapplicable","leadProduct":"Recombinant Human Growth Hormone","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GeneScience Pharmaceuticals Co Ltd","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneScience Pharmaceuticals Co Ltd \/ Children's Hospital of Fudan University | Beijing Children's Hospital | Shanghai Children's Hospital | Xin Hua Hospital, Shanghai Jiaotong University School of Medicine | The Children's Hospital of Zhejiang University School of Medicine | Children's Hospi","highestDevelopmentStatusID":"10","companyTruncated":"GeneScience Pharmaceuticals Co Ltd \/ Children's Hospital of Fudan University | Beijing Children's Hospital | Shanghai Children's Hospital | Xin Hua Hospital, Shanghai Jiaotong University School of Medicine | The Children's Hospital of Zhejiang University School of Medicine | Children's Hospi"},{"orgOrder":0,"company":"GeneScience Pharmaceuticals Co Ltd","sponsor":"Children's Hospital of Fudan University | Tongji Medical College, Huazhong University of Science and Technology | Beijing Children's Hospital | Peking University First Hospital | Peking Union Medical College Hospital | The Children's Hospital of Zhejiang ","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Hormone","year":"2018","type":"Inapplicable","leadProduct":"Recombinant Human Growth Hormone","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GeneScience Pharmaceuticals Co Ltd","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneScience Pharmaceuticals Co Ltd \/ Children's Hospital of Fudan University | Tongji Medical College, Huazhong University of Science and Technology | Beijing Children's Hospital | Peking University First Hospital | Peking Union Medical College Hospital | The Children's Hospital of Zhejiang ","highestDevelopmentStatusID":"10","companyTruncated":"GeneScience Pharmaceuticals Co Ltd \/ Children's Hospital of Fudan University | Tongji Medical College, Huazhong University of Science and Technology | Beijing Children's Hospital | Peking University First Hospital | Peking Union Medical College Hospital | The Children's Hospital of Zhejiang "},{"orgOrder":0,"company":"Merck Group","sponsor":"Merck Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Hormone","year":"2010","type":"Inapplicable","leadProduct":"Recombinant Human Growth Hormone","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Merck Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Merck Group \/ Merck Group","highestDevelopmentStatusID":"8","companyTruncated":"Merck Group \/ Merck Group"},{"orgOrder":0,"company":"Acceleron Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"ACE-031","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Acceleron Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Acceleron Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Acceleron Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Acceleron Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"ACE-031","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Acceleron Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Acceleron Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Acceleron Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Public Offering","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0.29999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ J.P. Morgan"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Public Offering","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ J.P. Morgan"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Public Offering","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0.23000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ J.P. Morgan","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ J.P. Morgan"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Public Offering","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0.20999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ J.P. Morgan"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Public Offering","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ J.P. Morgan"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Apitegromab","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2014","type":"Inapplicable","leadProduct":"PF-06252616","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"PF-06252616","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"SRK-015","moa":"Growth\/differentiation factor 8","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Scholar Rock \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Undisclosed"},{"orgOrder":0,"company":"4M Therapeutics","sponsor":"SynGAP Research Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Funding","leadProduct":"4MT2001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"4M Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"4M Therapeutics \/ SynGAP Research Fund","highestDevelopmentStatusID":"4","companyTruncated":"4M Therapeutics \/ SynGAP Research Fund"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"MeiraGTx \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"MeiraGTx \/ Undisclosed"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Agreement","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"MeiraGTx","amount2":0.41999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.41999999999999998,"dosageForm":"Injection","sponsorNew":"MeiraGTx \/ Johnson & Johnson Innovative Medicine","highestDevelopmentStatusID":"10","companyTruncated":"MeiraGTx \/ Johnson & Johnson Innovative Medicine"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"MeiraGTx","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ MeiraGTx","highestDevelopmentStatusID":"7","companyTruncated":"Johnson & Johnson Innovative Medicine \/ MeiraGTx"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"MeiraGTx \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"MeiraGTx \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Tisento Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Zagociguat","moa":"GUCY1A||sGC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tisento Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Tisento Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Tisento Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Tisento Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Zagociguat","moa":"GUCY1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tisento Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Tisento Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Tisento Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Tisento Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Zagociguat","moa":"GUCY1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tisento Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Tisento Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Tisento Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Zagociguat","moa":"GUCY1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cyclerion Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cyclerion Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cyclerion Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Divestment","leadProduct":"Zagociguat","moa":"GUCY1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cyclerion Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Tablet","sponsorNew":"Cyclerion Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cyclerion Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Zagociguat","moa":"GUCY1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cyclerion Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cyclerion Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cyclerion Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Tisento Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Divestment","leadProduct":"Zagociguat","moa":"GUCY1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cyclerion Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Tablet","sponsorNew":"Cyclerion Therapeutics \/ Tisento Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Cyclerion Therapeutics \/ Tisento Therapeutics"},{"orgOrder":0,"company":"Tisento Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Zagociguat","moa":"GUCY1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tisento Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Tisento Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Tisento Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Tisento Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Zagociguat","moa":"GUCY1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tisento Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Tisento Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Tisento Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Tisento Therapeutics","sponsor":"Invus","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Series A Financing","leadProduct":"Zagociguat","moa":"GUCY1A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tisento Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Tablet","sponsorNew":"Tisento Therapeutics \/ Invus","highestDevelopmentStatusID":"8","companyTruncated":"Tisento Therapeutics \/ Invus"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Pitolisant Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Harmony Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Harmony Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Pitolisant Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Harmony Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Harmony Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Pitolisant Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Harmony Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Harmony Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Pitolisant Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Harmony Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Harmony Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Pitolisant Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Harmony Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Harmony Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Pitolisant Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Harmony Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Harmony Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Pitolisant Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Harmony Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Harmony Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Pitolisant Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Harmony Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Harmony Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"OPKO Health","sponsor":"Perrigo Company plc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Inositol","moa":"Beta-amyloid||HCAR3\/NIACR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"OPKO Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"OPKO Health \/ Perrigo Company plc","highestDevelopmentStatusID":"8","companyTruncated":"OPKO Health \/ Perrigo Company plc"},{"orgOrder":0,"company":"The Italfarmaco Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Phase III","graph3":"The Italfarmaco Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"The Italfarmaco Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"The Italfarmaco Group \/ Undisclosed"},{"orgOrder":0,"company":"The Italfarmaco Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Phase III","graph3":"The Italfarmaco Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"The Italfarmaco Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"The Italfarmaco Group \/ Undisclosed"},{"orgOrder":0,"company":"The Italfarmaco Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Phase III","graph3":"The Italfarmaco Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"The Italfarmaco Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"The Italfarmaco Group \/ Undisclosed"},{"orgOrder":0,"company":"The Italfarmaco Group","sponsor":"Syneos Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Phase III","graph3":"The Italfarmaco Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"The Italfarmaco Group \/ Syneos Health","highestDevelopmentStatusID":"10","companyTruncated":"The Italfarmaco Group \/ Syneos Health"},{"orgOrder":0,"company":"Italfarmaco","sponsor":"Fortrea","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Italfarmaco","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Italfarmaco \/ Fortrea","highestDevelopmentStatusID":"8","companyTruncated":"Italfarmaco \/ Fortrea"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Italfarmaco S.p.A","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco S.p.A \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Italfarmaco S.p.A \/ Undisclosed"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Italfarmaco S.p.A","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco S.p.A \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Italfarmaco S.p.A \/ Undisclosed"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Medis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Italfarmaco S.p.A","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco S.p.A \/ Medis","highestDevelopmentStatusID":"15","companyTruncated":"Italfarmaco S.p.A \/ Medis"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Italfarmaco S.p.A","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco S.p.A \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Italfarmaco S.p.A \/ Undisclosed"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Italfarmaco S.p.A","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco S.p.A \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Italfarmaco S.p.A \/ Undisclosed"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Italfarmaco S.p.A","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco S.p.A \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Italfarmaco S.p.A \/ Undisclosed"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Italfarmaco S.p.A","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco S.p.A \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Italfarmaco S.p.A \/ Undisclosed"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"ITF Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Partnership","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PANTHERx Rare Pharmacy \/ ITF Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"PANTHERx Rare Pharmacy \/ ITF Therapeutics"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"REC-2282","moa":"HDAC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Recursion Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Recursion Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Recursion Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Augustine Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"AGT-100216","moa":"HDAC-6","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Augustine Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Augustine Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Augustine Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Augustine Therapeutics","sponsor":"Novo Holdings","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Miscellaneous","year":"2025","type":"Series A Financing","leadProduct":"AGT-100216","moa":"HDAC-6","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Augustine Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Augustine Therapeutics \/ Novo Holdings","highestDevelopmentStatusID":"6","companyTruncated":"Augustine Therapeutics \/ Novo Holdings"},{"orgOrder":0,"company":"Chong Kun Dang Pharm","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2023","type":"Licensing Agreement","leadProduct":"CKD-510","moa":"HDAC-6","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Chong Kun Dang Pharm","amount2":1.3100000000000001,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":1.3100000000000001,"dosageForm":"Tablet","sponsorNew":"Chong Kun Dang Pharm \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"6","companyTruncated":"Chong Kun Dang Pharm \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"ORY-4001","moa":"HDAC-6","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oryzon Genomics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Oryzon Genomics \/ Undisclosed"},{"orgOrder":0,"company":"ZevraDenmark","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Arimoclomol Citrate","moa":"heat-shock protein inducers","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ZevraDenmark","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ZevraDenmark \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"ZevraDenmark \/ Undisclosed"},{"orgOrder":0,"company":"Talaris Therapeutics","sponsor":"Duke University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2011","type":"Inapplicable","leadProduct":"Hematopoetic Stem Cell","moa":"Hematopoietic stem cell","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Talaris Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Talaris Therapeutics \/ Duke University","highestDevelopmentStatusID":"7","companyTruncated":"Talaris Therapeutics \/ Duke University"},{"orgOrder":0,"company":"Allife Medical Science and Technology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"Induced Hematopoietic Stem Cell","moa":"Hematopoietic system","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Allife Medical Science and Technology","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Allife Medical Science and Technology \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Allife Medical Science and Technology \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Osivelotor","moa":"Hemoglobin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Osivelotor","moa":"Hemoglobin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"GBT021601","moa":"Hemoglobin (HB)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"GBT021601","moa":"Hemoglobin (HB)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"GBT021601","moa":"Hemoglobin (HB)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"HemaQuest Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"HQK-1001","moa":"Hemoglobin (HB)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"HemaQuest Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"HemaQuest Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"HemaQuest Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"HemaQuest Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"HQK-1001","moa":"Hemoglobin (HB)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"HemaQuest Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"HemaQuest Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"HemaQuest Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Betibeglogene Autotemcel","moa":"Hemoglobin beta chain","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Assistance Publique \u2013 H\u00f4pitaux de Paris","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Assistance Publique \u2013 H\u00f4pitaux de Paris","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Pfizer Inc","highestDevelopmentStatusID":"8","companyTruncated":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Pfizer Inc"},{"orgOrder":0,"company":"Biopharma","sponsor":"Global Blood Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UKRAINE","productType":"Miscellaneous","year":"2020","type":"Agreement","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biopharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Biopharma \/ Global Blood Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Biopharma \/ Global Blood Therapeutics"},{"orgOrder":0,"company":"University of Illinois, Chicago","sponsor":"Global Blood Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"University of Illinois, Chicago","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Illinois, Chicago \/ Global Blood Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"University of Illinois, Chicago \/ Global Blood Therapeutics"},{"orgOrder":0,"company":"Inova Health Care Services","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inova Health Care Services","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inova Health Care Services \/ Pfizer Inc","highestDevelopmentStatusID":"8","companyTruncated":"Inova Health Care Services \/ Pfizer Inc"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Acquisition","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":5.4000000000000004,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":5.4000000000000004,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Acquisition","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":5.4000000000000004,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":5.4000000000000004,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Public Offering","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0.29999999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.29999999999999999,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Aes-103","moa":"Hemoglobin polymerization","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Frederick National Laboratory | Therapeutics for Rare and Neglected Diseases | QS Pharma | National Chung Cheng University | NIBIB Infrared Imaging and Thermometry Unit | ClinPharm Consulting | Olon Ricerca Bioscience | National Heart, Lung, and Blood Ins","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Aes-103","moa":"Hemoglobin polymerization","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Baxalta Pharmaceutical \/ Frederick National Laboratory | Therapeutics for Rare and Neglected Diseases | QS Pharma | National Chung Cheng University | NIBIB Infrared Imaging and Thermometry Unit | ClinPharm Consulting | Olon Ricerca Bioscience | National Heart, Lung, and Blood Ins","highestDevelopmentStatusID":"8","companyTruncated":"Baxalta Pharmaceutical \/ Frederick National Laboratory | Therapeutics for Rare and Neglected Diseases | QS Pharma | National Chung Cheng University | NIBIB Infrared Imaging and Thermometry Unit | ClinPharm Consulting | Olon Ricerca Bioscience | National Heart, Lung, and Blood Ins"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"GBT601","moa":"Hemoglobin polymerization","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"GBT601","moa":"Hemoglobin polymerization","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"GBT601","moa":"Hemoglobin polymerization","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"GBT601","moa":"Hemoglobin polymerization","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Hitachi Chemicals","sponsor":"Bluebird Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell & Gene Therapy","year":"2020","type":"Agreement","leadProduct":"Lentiglobin","moa":"Hemoglobin subunit beta (HBB)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Hitachi Chemicals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hitachi Chemicals \/ Bluebird Bio","highestDevelopmentStatusID":"7","companyTruncated":"Hitachi Chemicals \/ Bluebird Bio"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Lentiglobin","moa":"||Hemoglobin subunit beta (HBB)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Lentiglobin","moa":"Hemoglobin subunit beta (HBB)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Lentiglobin","moa":"Hemoglobin subunit beta (HBB)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Lentiglobin","moa":"Hemoglobin subunit beta (HBB)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Sangart","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"MP4CO","moa":"Hemoglobins","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sangart","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Sangart \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sangart \/ Undisclosed"},{"orgOrder":0,"company":"Sangart","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"MP4CO","moa":"Hemoglobins","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sangart","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Sangart \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sangart \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"CSL889","moa":"Hemojuvelin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"CSL889","moa":"Hemojuvelin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2019","type":"Inapplicable","leadProduct":"GC1111","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Green Cross Lab Cell \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Green Cross Lab Cell \/ Undisclosed"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2011","type":"Inapplicable","leadProduct":"GC1111","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Green Cross Lab Cell \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Green Cross Lab Cell \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Idursulfase","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Idursulfase","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"Takeda Pharmaceutical \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Idursulfase","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Idursulfase","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2015","type":"Inapplicable","leadProduct":"Idursulfase","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"SHIRE PLC \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2015","type":"Inapplicable","leadProduct":"Idursulfase","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2012","type":"Inapplicable","leadProduct":"Idursulfase","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2014","type":"Inapplicable","leadProduct":"Idursulfase","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Lepunafusp Alfa","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Spruce Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Acquisition","leadProduct":"Tralesinidase Alfa","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Spruce Biosciences","highestDevelopmentStatusID":"8","companyTruncated":"BioMarin Pharmaceutical \/ Spruce Biosciences"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2012","type":"Inapplicable","leadProduct":"Idursulfase Beta","moa":"Heparan sulfate | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Green Cross Lab Cell \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Green Cross Lab Cell \/ Undisclosed"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2016","type":"Inapplicable","leadProduct":"Idursulfase Beta","moa":"Heparan sulfate | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Green Cross Lab Cell \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Green Cross Lab Cell \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"3W Fund Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2020","type":"Series E Financing","leadProduct":"Idursulfase Beta","moa":"Heparan sulfate | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CANbridge Pharmaceuticals","amount2":0.040000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ 3W Fund Management","highestDevelopmentStatusID":"15","companyTruncated":"CANbridge Pharmaceuticals \/ 3W Fund Management"},{"orgOrder":0,"company":"GC Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Idursulfase Beta","moa":"Heparan sulfate | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"GC Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"GC Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"GC Pharma \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"General Atlantic","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2020","type":"Series D Financing","leadProduct":"Idursulfase Beta","moa":"Heparan sulfate | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CANbridge Pharmaceuticals","amount2":0.10000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ General Atlantic","highestDevelopmentStatusID":"15","companyTruncated":"CANbridge Pharmaceuticals \/ General Atlantic"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"alpha L Idosiduronase","moa":"Heparan sulfate | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"CinnaGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRAN","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"alpha L Idosiduronase","moa":"Heparan sulfate | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CinnaGen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CinnaGen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CinnaGen \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Modus Therapeutics","sponsor":"Ergomed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Sevuparin","moa":"heparin derivatives and low molecular weight (or depolymerized) heparins","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Modus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Modus Therapeutics \/ Ergomed","highestDevelopmentStatusID":"8","companyTruncated":"Modus Therapeutics \/ Ergomed"},{"orgOrder":0,"company":"Helixmith","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Donaperminogene Seltoplasmid","moa":"Hepatocyte growth factor (HGF)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Helixmith","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Helixmith \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Helixmith \/ Undisclosed"},{"orgOrder":0,"company":"GEMMA Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"GB221","moa":"HER2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GEMMA Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GEMMA Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GEMMA Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Phoenix Nest","sponsor":"Cure Sanfilippo Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Funding","leadProduct":"AAV-TT","moa":"HGSNAT","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Phoenix Nest","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Phoenix Nest \/ Cure Sanfilippo Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Phoenix Nest \/ Cure Sanfilippo Foundation"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Human Normal Immunoglobulin","moa":"High affinity immunoglobulin gamma Fc receptor I; Putative high affinity immunoglobulin gamma Fc receptor IB; Low affinity immunoglobulin gamma Fc region receptor II-a; Low affinity immunoglobulin gamma Fc region receptor II-b; Low affinity immunoglobulin gamma Fc region receptor II-c; Low affinity immunoglobulin gamma Fc region receptor III-A; Low affinity immunoglobulin gamma Fc region receptor III-B; Complement C3; Complement C4-A; Complement C4-B; Complement C5","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Antibody, Unconjugated","year":"2011","type":"Inapplicable","leadProduct":"Human Normal Immunoglobulin","moa":"High affinity immunoglobulin gamma Fc receptor I; Putative high affinity immunoglobulin gamma Fc receptor IB; Low affinity immunoglobulin gamma Fc region receptor II-a; Low affinity immunoglobulin gamma Fc region receptor II-b; Low affinity immunoglobulin gamma Fc region receptor II-c; Low affinity immunoglobulin gamma Fc region receptor III-A; Low affinity immunoglobulin gamma Fc region receptor III-B; Complement C3; Complement C4-A; Complement C4-B; Complement C5","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibody, Unconjugated","year":"2014","type":"Inapplicable","leadProduct":"Human Normal Immunoglobulin","moa":"High affinity immunoglobulin gamma Fc receptor I; Putative high affinity immunoglobulin gamma Fc receptor IB; Low affinity immunoglobulin gamma Fc region receptor II-a; Low affinity immunoglobulin gamma Fc region receptor II-b; Low affinity immunoglobulin gamma Fc region receptor II-c; Low affinity immunoglobulin gamma Fc region receptor III-A; Low affinity immunoglobulin gamma Fc region receptor III-B; Complement C3; Complement C4-A; Complement C4-B; Complement C5","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2013","type":"Inapplicable","leadProduct":"Human Normal Immunoglobulin","moa":"High affinity immunoglobulin gamma Fc receptor I; Putative high affinity immunoglobulin gamma Fc receptor IB; Low affinity immunoglobulin gamma Fc region receptor II-a; Low affinity immunoglobulin gamma Fc region receptor II-b; Low affinity immunoglobulin gamma Fc region receptor II-c; Low affinity immunoglobulin gamma Fc region receptor III-A; Low affinity immunoglobulin gamma Fc region receptor III-B; Complement C3; Complement C4-A; Complement C4-B; Complement C5","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Human Normal Immunoglobulin","moa":"High affinity immunoglobulin gamma Fc receptor I; Putative high affinity immunoglobulin gamma Fc receptor IB; Low affinity immunoglobulin gamma Fc region receptor II-a; Low affinity immunoglobulin gamma Fc region receptor II-b; Low affinity immunoglobulin gamma Fc region receptor II-c; Low affinity immunoglobulin gamma Fc region receptor III-A; Low affinity immunoglobulin gamma Fc region receptor III-B; Complement C3; Complement C4-A; Complement C4-B; Complement C5","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Immune Globulin (Human)","moa":"High affinity immunoglobulin gamma Fc receptor I; Putative high affinity immunoglobulin gamma Fc receptor IB; Low affinity immunoglobulin gamma Fc region receptor II-a; Low affinity immunoglobulin gamma Fc region receptor II-b; Low affinity immunoglobulin gamma Fc region receptor II-c; Low affinity immunoglobulin gamma Fc region receptor III-A; Low affinity immunoglobulin gamma Fc region receptor III-B; Complement C3; Complement C4-A; Complement C4-B; Complement C5","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Soleo Health","sponsor":"Grifols International","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Agreement","leadProduct":"Immune Globulin (Human)","moa":"High affinity immunoglobulin gamma Fc receptor I; Putative high affinity immunoglobulin gamma Fc receptor IB; Low affinity immunoglobulin gamma Fc region receptor II-a; Low affinity immunoglobulin gamma Fc region receptor II-b; Low affinity immunoglobulin gamma Fc region receptor II-c; Low affinity immunoglobulin gamma Fc region receptor III-A; Low affinity immunoglobulin gamma Fc region receptor III-B; Complement C3; Complement C4-A; Complement C4-B; Complement C5","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Soleo Health","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Soleo Health \/ Grifols International","highestDevelopmentStatusID":"15","companyTruncated":"Soleo Health \/ Grifols International"},{"orgOrder":0,"company":"Kedrion","sponsor":"Atlantic Research Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Immune Globulin (Human)","moa":"High affinity immunoglobulin gamma Fc receptor I; Putative high affinity immunoglobulin gamma Fc receptor IB; Low affinity immunoglobulin gamma Fc region receptor II-a; Low affinity immunoglobulin gamma Fc region receptor II-b; Low affinity immunoglobulin gamma Fc region receptor II-c; Low affinity immunoglobulin gamma Fc region receptor III-A; Low affinity immunoglobulin gamma Fc region receptor III-B; Complement C3; Complement C4-A; Complement C4-B; Complement C5","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kedrion","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Kedrion \/ Atlantic Research Group","highestDevelopmentStatusID":"10","companyTruncated":"Kedrion \/ Atlantic Research Group"},{"orgOrder":0,"company":"Zymenex","sponsor":"European Commission","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Enzyme","year":"2012","type":"Inapplicable","leadProduct":"Velmanase Alfa","moa":"High mannose oligosaccharides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zymenex","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Zymenex \/ European Commission","highestDevelopmentStatusID":"10","companyTruncated":"Zymenex \/ European Commission"},{"orgOrder":0,"company":"Zymenex","sponsor":"European Commission","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Enzyme","year":"2012","type":"Inapplicable","leadProduct":"Velmanase Alfa","moa":"High mannose oligosaccharides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zymenex","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Zymenex \/ European Commission","highestDevelopmentStatusID":"10","companyTruncated":"Zymenex \/ European Commission"},{"orgOrder":0,"company":"Zymenex","sponsor":"European Commission","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Enzyme","year":"2011","type":"Inapplicable","leadProduct":"Velmanase Alfa","moa":"High mannose oligosaccharides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zymenex","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Zymenex \/ European Commission","highestDevelopmentStatusID":"10","companyTruncated":"Zymenex \/ European Commission"},{"orgOrder":0,"company":"Zymenex","sponsor":"European Commission","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Velmanase Alfa","moa":"High mannose oligosaccharides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zymenex","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Zymenex \/ European Commission","highestDevelopmentStatusID":"10","companyTruncated":"Zymenex \/ European Commission"},{"orgOrder":0,"company":"Zymenex","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Enzyme","year":"2015","type":"Inapplicable","leadProduct":"Velmanase Alfa","moa":"High mannose oligosaccharides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zymenex","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zymenex \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zymenex \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2013","type":"Inapplicable","leadProduct":"Velmanase Alfa","moa":"High mannose oligosaccharides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Chiesi Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2013","type":"Inapplicable","leadProduct":"Velmanase Alfa","moa":"High mannose oligosaccharides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Chiesi Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2019","type":"Inapplicable","leadProduct":"Velmanase Alfa","moa":"High mannose oligosaccharides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Chiesi Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Cromsource","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2016","type":"Inapplicable","leadProduct":"Velmanase Alfa","moa":"High mannose oligosaccharides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Chiesi Group \/ Cromsource","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Cromsource"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Velmanase Alfa","moa":"High mannose oligosaccharides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chiesi Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Undisclosed"},{"orgOrder":0,"company":"Assistance Publique \u2013 H\u00f4pitaux de Paris","sponsor":"INSERM U1163","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Famotidine","moa":"Histamine H2 receptor","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Assistance Publique \u2013 H\u00f4pitaux de Paris","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ INSERM U1163","highestDevelopmentStatusID":"9","companyTruncated":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ INSERM U1163"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Pitolisant Hydrochloride","moa":"Histamine H3 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Harmony Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Harmony Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Pitolisant Hydrochloride","moa":"Histamine H3 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Harmony Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Harmony Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Pitolisant Hydrochloride","moa":"Histamine H3 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Harmony Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Harmony Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Italfarmaco","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"497833-27-9","moa":"Histone deacetylase (HDAC)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Italfarmaco","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Italfarmaco \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Rosuvastatin Calcium","moa":"HMG-CoA reductase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Rosuvastatin Calcium","moa":"HMG-CoA reductase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"Hillhurst Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Carbon Monoxide","moa":"HO metabolic pathway","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Hillhurst Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Hillhurst Biopharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Hillhurst Biopharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Hillhurst Biopharmaceuticals","sponsor":"National Heart, Lung, And Blood Institute","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Funding","leadProduct":"Carbon Monoxide","moa":"HO metabolic pathway","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Hillhurst Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Hillhurst Biopharmaceuticals \/ National Heart, Lung, And Blood Institute","highestDevelopmentStatusID":"6","companyTruncated":"Hillhurst Biopharmaceuticals \/ National Heart, Lung, And Blood Institute"},{"orgOrder":0,"company":"Hillhurst Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Carbon Monoxide","moa":"HO metabolic pathway","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Hillhurst Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Hillhurst Biopharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Hillhurst Biopharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Hillhurst Biopharmaceuticals","sponsor":"National Heart, Lung, and Blood Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"HBI-002","moa":"HO metabolic pathway","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Hillhurst Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Hillhurst Biopharmaceuticals \/ National Heart, Lung, and Blood Institute","highestDevelopmentStatusID":"7","companyTruncated":"Hillhurst Biopharmaceuticals \/ National Heart, Lung, and Blood Institute"},{"orgOrder":0,"company":"Hillhurst Biopharmaceuticals","sponsor":"National Heart, Lung, and Blood Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"HBI-002","moa":"HO metabolic pathway","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Hillhurst Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Hillhurst Biopharmaceuticals \/ National Heart, Lung, and Blood Institute","highestDevelopmentStatusID":"7","companyTruncated":"Hillhurst Biopharmaceuticals \/ National Heart, Lung, and Blood Institute"},{"orgOrder":0,"company":"Prolong Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2013","type":"Inapplicable","leadProduct":"Pegylated Carboxyhemoglobin Bovine","moa":"Homeostasis","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Prolong Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prolong Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Prolong Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Prolong Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Pegylated Carboxyhemoglobin Bovine","moa":"Homeostasis","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Prolong Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prolong Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Prolong Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Prolong Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"Pegylated Carboxyhemoglobin Bovine","moa":"Homeostasis","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Prolong Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prolong Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Prolong Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Phrixus Pharmaceuticals","sponsor":"Charley's Fund","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Poloxamer 188","moa":"Homeostasis","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Phrixus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Phrixus Pharmaceuticals \/ Charley's Fund","highestDevelopmentStatusID":"8","companyTruncated":"Phrixus Pharmaceuticals \/ Charley's Fund"},{"orgOrder":0,"company":"Arbor Biotechnologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ABO-101","moa":"HS","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Arbor Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Arbor Biotechnologies \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Arbor Biotechnologies \/ Undisclosed"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ABO-101","moa":"HS","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Abeona Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Abeona Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Arimoclomol Citrate","moa":"HSF-1 pathway","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Orphazyme \/ Undisclosed"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Arimoclomol Citrate","moa":"HSF-1 pathway","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Orphazyme \/ Undisclosed"},{"orgOrder":0,"company":"Nobias Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"NB-001","moa":"HSV DNA polymerase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nobias Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Nobias Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nobias Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Licensing Agreement","leadProduct":"Votoplam","moa":"HTT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":2.8999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":2.8999999999999999,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Votoplam","moa":"HTT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Votoplam","moa":"HTT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Votoplam","moa":"HTT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Votoplam","moa":"HTT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Votoplam","moa":"HTT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Votoplam","moa":"HTT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Votoplam","moa":"HTT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Votoplam","moa":"HTT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"VY-HTT01","moa":"HTT expression","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Voyager Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Voyager Therapeutics, Inc \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Voyager Therapeutics, Inc \/ Undisclosed"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"VY-HTT01","moa":"HTT expression","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Voyager Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Voyager Therapeutics, Inc \/ Sanofi","highestDevelopmentStatusID":"6","companyTruncated":"Voyager Therapeutics, Inc \/ Sanofi"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"VY-HTT01","moa":"HTT expression","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Voyager Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Voyager Therapeutics, Inc \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Voyager Therapeutics, Inc \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"ALN-HTT02","moa":"HTT mRNA","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Lonodelestat","moa":"Human neutrophil elastase||Neutrophil elastase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eyevensys","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"EYS611","moa":"Human transferrin","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Eyevensys","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eyevensys \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Eyevensys \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Leerink Partners","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Leerink Partners"},{"orgOrder":0,"company":"Uniqure","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Leerink Partners","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Leerink Partners"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"ISIS 443139","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"PTC518","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"PTC518","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"RO7234292","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"RO7234292","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2017","type":"Inapplicable","leadProduct":"RO7234292","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"RO7234292","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Tominersen","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chugai Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chugai Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Tominersen","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Tominersen","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Ionis Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Tominersen","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Ionis Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Tominersen","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Partnership","leadProduct":"Tominersen","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2014","type":"Inapplicable","leadProduct":"Kinetin","moa":"Hyaluronic acid","graph1":"Genetic Disease","graph2":"Phase I","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NYU Langone Health \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"NYU Langone Health \/ Undisclosed"},{"orgOrder":0,"company":"DeNova Research","sponsor":"Merz Aesthetics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"HYDROXYPROPYLMETHYLCELLULOSE","moa":"Hydroxylapatite","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"DeNova Research","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"DeNova Research \/ Merz Aesthetics","highestDevelopmentStatusID":"11","companyTruncated":"DeNova Research \/ Merz Aesthetics"},{"orgOrder":0,"company":"The University of Manchester","sponsor":"Avrobio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Licensing Agreement","leadProduct":"AVR-RD-05","moa":"IDS","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"The University of Manchester","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"The University of Manchester \/ Avrobio","highestDevelopmentStatusID":"4","companyTruncated":"The University of Manchester \/ Avrobio"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AVR-RD-05","moa":"IDS","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AVR-RD-05","moa":"IDS","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AVR-RD-05","moa":"IDS","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Capsida","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"CAP-001","moa":"IDS","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Capsida","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Capsida \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Capsida \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Public Offering","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0.28000000000000003,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.32000000000000001,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Goldman Sachs & Co","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Public Offering","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0.32000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.32000000000000001,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Goldman Sachs & Co","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Idursulfase","moa":"||IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Private Placement","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0.5,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.5,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Tividenofusp Alfa","moa":"IDS*","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Tividenofusp Alfa","moa":"IDS*","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Tividenofusp Alfa","moa":"IDS*","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Partnership","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0.81000000000000005,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.81000000000000005,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Nippon Shinyaku","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Nippon Shinyaku"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"SB-913","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Azidus","sponsor":"Boya Bio Pharmaceutical Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BRAZIL","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Boya Immunoglobulin","moa":"IgG","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Azidus","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Azidus \/ Boya Bio Pharmaceutical Group","highestDevelopmentStatusID":"10","companyTruncated":"Azidus \/ Boya Bio Pharmaceutical Group"},{"orgOrder":0,"company":"Paradox Immunotherapeutics","sponsor":"Symbiosis Pharmaceutical Services","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Antibody, Unconjugated","year":"2025","type":"Financing","leadProduct":"LX-96","moa":"IgG","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Paradox Immunotherapeutics","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"Paradox Immunotherapeutics \/ Symbiosis Pharmaceutical Services","highestDevelopmentStatusID":"4","companyTruncated":"Paradox Immunotherapeutics \/ Symbiosis Pharmaceutical Services"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Octanorm","moa":"IgG","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"RI-002","moa":"IgG","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ADMA Biologics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ADMA Biologics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"ADMA Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"VCA-894A","moa":"IGHMBP2","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Vanda Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vanda Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Vanda Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"VCA-894A","moa":"IGHMBP2","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Vanda Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vanda Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Vanda Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"VCA-894A","moa":"IGHMBP2","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vanda Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vanda Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vanda Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Telomir Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Telomir-1","moa":"IL-17 receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Telomir Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Telomir Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Telomir Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Telomir Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Telomir-1","moa":"IL-17 receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Telomir Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Telomir Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Telomir Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"AB2 Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Tadekinig Alpha","moa":"IL-18 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AB2 Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AB2 Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AB2 Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Minaris Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Agreement","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Minaris Regenerative Medicine","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Minaris Regenerative Medicine"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"MB-107","moa":"IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Biocad","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"ANB-002","moa":"IL-36 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biocad","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Biocad \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biocad \/ Undisclosed"},{"orgOrder":0,"company":"Biocad","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"ANB-002","moa":"IL-36 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biocad","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Biocad \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biocad \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Satralizumab","moa":"IL6Ralpha\/GP130","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Mirum Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2021","type":"Licensing Agreement","leadProduct":"Maralixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Takeda Pharmaceutical \/ Mirum Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Takeda Pharmaceutical \/ Mirum Pharmaceuticals"},{"orgOrder":0,"company":"Eversana","sponsor":"Mirum Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Partnership","leadProduct":"Maralixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eversana","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Eversana \/ Mirum Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Eversana \/ Mirum Pharmaceuticals"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Maralixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Lumena Pharmaceuticals | Childhood Liver Disease Network","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Maralixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mirum Pharmaceuticals \/ Lumena Pharmaceuticals | Childhood Liver Disease Network","highestDevelopmentStatusID":"8","companyTruncated":"Mirum Pharmaceuticals \/ Lumena Pharmaceuticals | Childhood Liver Disease Network"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Maralixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Clinigen Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Maralixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mirum Pharmaceuticals \/ Clinigen Group","highestDevelopmentStatusID":"8","companyTruncated":"Mirum Pharmaceuticals \/ Clinigen Group"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Mirum Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"CANbridge Pharmaceuticals \/ Mirum Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"CANbridge Pharmaceuticals \/ Mirum Pharmaceuticals"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Mirum Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"CANbridge Pharmaceuticals \/ Mirum Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"CANbridge Pharmaceuticals \/ Mirum Pharmaceuticals"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Neopharm","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Neopharm","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Neopharm"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Agreement","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0.11,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Satiogen","sponsor":"Mirum Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Acquisition","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Satiogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Satiogen \/ Mirum Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Satiogen \/ Mirum Pharmaceuticals"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Licensing Agreement","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0.12,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ CANbridge Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"GC Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Licensing Agreement","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ GC Pharma","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ GC Pharma"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ipsen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ipsen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Ipsen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ipsen \/ Undisclosed"},{"orgOrder":0,"company":"Ipsen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ipsen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Ipsen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ipsen \/ Undisclosed"},{"orgOrder":0,"company":"Ipsen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ipsen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Ipsen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ipsen \/ Undisclosed"},{"orgOrder":0,"company":"Ipsen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ipsen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Ipsen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ipsen \/ Undisclosed"},{"orgOrder":0,"company":"Albireo","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Albireo","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Albireo \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Albireo \/ Undisclosed"},{"orgOrder":0,"company":"Albireo","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Albireo","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Albireo \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Albireo \/ Undisclosed"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Jadeite Medicines","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Licensing Agreement","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Albireo Pharma","amount2":0.14000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14000000000000001,"dosageForm":"Capsule","sponsorNew":"Albireo Pharma \/ Jadeite Medicines","highestDevelopmentStatusID":"15","companyTruncated":"Albireo Pharma \/ Jadeite Medicines"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Divestment","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Albireo Pharma","amount2":0.11,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Capsule","sponsorNew":"Albireo Pharma \/ Jefferies","highestDevelopmentStatusID":"15","companyTruncated":"Albireo Pharma \/ Jefferies"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Albireo Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Albireo Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Albireo Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Albireo Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Albireo Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Albireo Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Albireo Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Albireo Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Albireo Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Albireo Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Albireo Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Albireo Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Albireo Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Albireo Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Albireo Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Albireo Pharma \/ Undisclosed"},{"orgOrder":0,"company":"University of Alabama at Birmingham","sponsor":"Children's Healthcare of Atlanta | Children's Hospital Los Angeles","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Polyinosinic Acid","moa":"Immune","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of Alabama at Birmingham","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Alabama at Birmingham \/ Children's Healthcare of Atlanta | Children's Hospital Los Angeles","highestDevelopmentStatusID":"8","companyTruncated":"University of Alabama at Birmingham \/ Children's Healthcare of Atlanta | Children's Hospital Los Angeles"},{"orgOrder":0,"company":"Rambam Health Care Campus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Probiotic","year":"2013","type":"Inapplicable","leadProduct":"Probiotic","moa":"Immune","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Rambam Health Care Campus","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Rambam Health Care Campus \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Rambam Health Care Campus \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Immune Globulin Infusion (Human)","moa":"Immunoglobulin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Kedrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-protein Conjugate","year":"2012","type":"Inapplicable","leadProduct":"Intravenous Immune Globulin","moa":"Immunoglobulins","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kedrion","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kedrion \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kedrion \/ Undisclosed"},{"orgOrder":0,"company":"Kedrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-protein Conjugate","year":"2019","type":"Inapplicable","leadProduct":"Intravenous Immune Globulin","moa":"Immunoglobulins","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kedrion","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kedrion \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kedrion \/ Undisclosed"},{"orgOrder":0,"company":"Kedrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-protein Conjugate","year":"2021","type":"Inapplicable","leadProduct":"Intravenous Immune Globulin","moa":"Immunoglobulins","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kedrion","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kedrion \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kedrion \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Antibody-protein Conjugate","year":"2020","type":"Inapplicable","leadProduct":"IVIG-PEG","moa":"Immunoglobulins","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Teva Pharmaceutical Industries","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Laquinimod","moa":"immunomodulators","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Teva Pharmaceutical Industries","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Teva Pharmaceutical Industries \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Teva Pharmaceutical Industries \/ Undisclosed"},{"orgOrder":0,"company":"Northwell Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Sotatercept","moa":"Inhibin beta A chain","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Northwell Health","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Northwell Health \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Northwell Health \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Cam Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Private Placement","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Cam Capital","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Cam Capital"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Public Offering","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0.12,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Jefferies"},{"orgOrder":0,"company":"Rezolute","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Public Offering","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0.13,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Jefferies"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Public Offering","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Jefferies"},{"orgOrder":0,"company":"Rezolute","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Public Offering","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Jefferies"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Kaufmann Funds","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Public Offering","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Kaufmann Funds","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Kaufmann Funds"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Kaufmann Funds","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Public Offering","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Kaufmann Funds","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Kaufmann Funds"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"SLR Capital Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Financing","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ SLR Capital Partners","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ SLR Capital Partners"},{"orgOrder":0,"company":"XOMA","sponsor":"Rezolute","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Agreement","leadProduct":"Ersodetug","moa":"INSR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"XOMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"XOMA \/ Rezolute","highestDevelopmentStatusID":"8","companyTruncated":"XOMA \/ Rezolute"},{"orgOrder":0,"company":"ArmaGen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2014","type":"Inapplicable","leadProduct":"AGT-182","moa":"Insulin receptor (INSR)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ArmaGen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ArmaGen \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ArmaGen \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"RZ358","moa":"Insulin receptor (INSR)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Rezolute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"RZ358","moa":"Insulin receptor (INSR)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Undisclosed"},{"orgOrder":0,"company":"Children's Hospital Medical Center, Cincinnati","sponsor":"Ipsen | Charley's Fund","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Mecasermin","moa":"Insulin-like growth factor I receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Children's Hospital Medical Center, Cincinnati","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Children's Hospital Medical Center, Cincinnati \/ Ipsen | Charley's Fund","highestDevelopmentStatusID":"7","companyTruncated":"Children's Hospital Medical Center, Cincinnati \/ Ipsen | Charley's Fund"},{"orgOrder":0,"company":"St. Louis University","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2013","type":"Inapplicable","leadProduct":"Abciximab","moa":"Integrin alpha-IIb\/beta-3 | Integrin alpha-V\/beta-3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"St. Louis University","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"St. Louis University \/ Johnson & Johnson Innovative Medicine","highestDevelopmentStatusID":"8","companyTruncated":"St. Louis University \/ Johnson & Johnson Innovative Medicine"},{"orgOrder":0,"company":"Rocket Pharma","sponsor":"California Institute for Regenerative Medicine (CIRM)","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"RP-L201","moa":"Integrin beta-2 (ITGB2)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharma \/ California Institute for Regenerative Medicine (CIRM)","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharma \/ California Institute for Regenerative Medicine (CIRM)"},{"orgOrder":0,"company":"Rocket Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"RP-L201","moa":"Integrin beta-2 (ITGB2)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Friedreich's Ataxia Research Alliance","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Interferon Beta-1b","moa":"Interferon alpha\/beta receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amgen Inc \/ Friedreich's Ataxia Research Alliance","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Friedreich's Ataxia Research Alliance"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Friedreich's Ataxia Research Alliance","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Interferon Beta-1b","moa":"Interferon alpha\/beta receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amgen Inc \/ Friedreich's Ataxia Research Alliance","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Friedreich's Ataxia Research Alliance"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Friedreich's Ataxia Research Alliance","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"Interferon Beta-1b","moa":"Interferon alpha\/beta receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amgen Inc \/ Friedreich's Ataxia Research Alliance","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Friedreich's Ataxia Research Alliance"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Emapalumab","moa":"Interferon gamma","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Light Chain Bioscience","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Emapalumab","moa":"Interferon gamma","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Swedish Orphan Biovitrum AB \/ Light Chain Bioscience","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Light Chain Bioscience"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Emapalumab","moa":"Interferon gamma","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Emapalumab","moa":"Interferon gamma","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Emapalumab","moa":"Interferon gamma","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Emapalumab","moa":"Interferon gamma","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Emapalumab","moa":"Interferon gamma","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Seventh Framework Programme (FP7)","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Antibody, Unconjugated","year":"2014","type":"Inapplicable","leadProduct":"Emapalumab","moa":"Interferon gamma","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Swedish Orphan Biovitrum AB \/ Seventh Framework Programme (FP7)","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Seventh Framework Programme (FP7)"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Seventh Framework Programme (FP7)","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Antibody, Unconjugated","year":"2013","type":"Inapplicable","leadProduct":"NI-0501","moa":"Interferon gamma","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Swedish Orphan Biovitrum AB \/ Seventh Framework Programme (FP7)","highestDevelopmentStatusID":"9","companyTruncated":"Swedish Orphan Biovitrum AB \/ Seventh Framework Programme (FP7)"},{"orgOrder":0,"company":"Lundquist Institute for Biomedical Innovation","sponsor":"University of Minnesota | Amgen Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"Interferon Gamma-1b","moa":"Interferon gamma receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Lundquist Institute for Biomedical Innovation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Lundquist Institute for Biomedical Innovation \/ University of Minnesota | Amgen Inc","highestDevelopmentStatusID":"8","companyTruncated":"Lundquist Institute for Biomedical Innovation \/ University of Minnesota | Amgen Inc"},{"orgOrder":0,"company":"Indiana University","sponsor":"Amgen Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Interferon Gamma-1b","moa":"Interferon gamma receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Indiana University","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Indiana University \/ Amgen Inc","highestDevelopmentStatusID":"8","companyTruncated":"Indiana University \/ Amgen Inc"},{"orgOrder":0,"company":"Children's Hospital of Philadelphia","sponsor":"Friedreich's Ataxia Research Alliance | Vidara Therapeutics Research Ltd","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2013","type":"Inapplicable","leadProduct":"Interferon Gamma-1b","moa":"Interferon gamma receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Children's Hospital of Philadelphia","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Children's Hospital of Philadelphia \/ Friedreich's Ataxia Research Alliance | Vidara Therapeutics Research Ltd","highestDevelopmentStatusID":"8","companyTruncated":"Children's Hospital of Philadelphia \/ Friedreich's Ataxia Research Alliance | Vidara Therapeutics Research Ltd"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2011","type":"Inapplicable","leadProduct":"Canakinumab","moa":"Interleukin-1 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2014","type":"Inapplicable","leadProduct":"Canakinumab","moa":"Interleukin-1 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2010","type":"Inapplicable","leadProduct":"Canakinumab","moa":"Interleukin-1 beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Alvotech","sponsor":"Advanz Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ICELAND","productType":"Antibody, Unconjugated","year":"2025","type":"Agreement","leadProduct":"Canakinumab","moa":"Interleukin-1 beta","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Alvotech","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alvotech \/ Advanz Pharma","highestDevelopmentStatusID":"1","companyTruncated":"Alvotech \/ Advanz Pharma"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2012","type":"Inapplicable","leadProduct":"Canakinumab","moa":"Interleukin-1 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"Canakinumab","moa":"Interleukin-1 beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2010","type":"Inapplicable","leadProduct":"Canakinumab","moa":"Interleukin-1 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2010","type":"Inapplicable","leadProduct":"Canakinumab","moa":"Interleukin-1 beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2010","type":"Inapplicable","leadProduct":"Canakinumab","moa":"Interleukin-1 beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2011","type":"Inapplicable","leadProduct":"Canakinumab","moa":"Interleukin-1 beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Tigermed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Anakinra","moa":"Interleukin-1 receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Tigermed","highestDevelopmentStatusID":"11","companyTruncated":"Swedish Orphan Biovitrum AB \/ Tigermed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Anakinra","moa":"Interleukin-1 receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"The Lundquist Institute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Anakinra","moa":"Interleukin-1 receptor","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"The Lundquist Institute","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"The Lundquist Institute \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"The Lundquist Institute \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Guselkumab","moa":"Interleukin-23","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Guselkumab","moa":"Interleukin-23","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Teva Pharmaceutical Industries","sponsor":"Eton Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Miscellaneous","year":"2025","type":"Acquisition","leadProduct":"Zinc Acetate Dihydrate","moa":"Intestinal cell metallothionein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Teva Pharmaceutical Industries","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Teva Pharmaceutical Industries \/ Eton Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Teva Pharmaceutical Industries \/ Eton Pharmaceuticals"},{"orgOrder":0,"company":"Eton Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Zinc Acetate Dihydrate","moa":"Intestinal cell metallothionein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eton Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Eton Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Eton Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"NKT Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"NKTT120","moa":"Invariant NKT cell","graph1":"Genetic Disease","graph2":"Phase I","graph3":"NKT Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NKT Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"NKT Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Iohexol","moa":"iodine-containing contrast media","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Deferasirox","moa":"Iron","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Deferasirox","moa":"Iron","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Deferasirox","moa":"Iron","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Deferasirox","moa":"Iron","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Deferasirox","moa":"Iron","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Deferasirox","moa":"Iron","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Deferasirox","moa":"Iron","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Inova Health Care Services","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Deferoxamine Mesylate","moa":"Iron","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inova Health Care Services","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inova Health Care Services \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Inova Health Care Services \/ Undisclosed"},{"orgOrder":0,"company":"Theris Medical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Deferoxamine Mesylate","moa":"Iron","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Theris Medical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Patch","sponsorNew":"Theris Medical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Theris Medical \/ Undisclosed"},{"orgOrder":0,"company":"APOPHARMA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Deferiprone","moa":"Iron||Iron (Fe)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"APOPHARMA","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"APOPHARMA \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"APOPHARMA \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Global Rare Diseases","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Deferiprone","moa":"Iron (Fe)","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Chiesi Global Rare Diseases","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chiesi Global Rare Diseases \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Chiesi Global Rare Diseases \/ Undisclosed"},{"orgOrder":0,"company":"Apotex Inc","sponsor":"Chiesi Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2020","type":"Acquisition","leadProduct":"Deferiprone","moa":"Iron (Fe)","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Apotex Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Apotex Inc \/ Chiesi Group","highestDevelopmentStatusID":"15","companyTruncated":"Apotex Inc \/ Chiesi Group"},{"orgOrder":0,"company":"Atlas","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Ciclopirox","moa":"Iron | Aluminium","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atlas","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Atlas \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atlas \/ Undisclosed"},{"orgOrder":0,"company":"Antisense Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ATL1102","moa":"ITGA4 messenger RNA (ITGA4 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Antisense Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Antisense Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Antisense Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Antisense Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"ATL1102","moa":"ITGA4 messenger RNA (ITGA4 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Antisense Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Antisense Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Antisense Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Antisense Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"ATL1102","moa":"ITGA4 messenger RNA (ITGA4 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Antisense Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Antisense Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Antisense Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Percheron Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ATL1102","moa":"ITGA4 messenger RNA (ITGA4 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Percheron Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Percheron Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Percheron Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arnatar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ART4","moa":"JAG1 protein level","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Arnatar Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arnatar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Arnatar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"JNJ-64251330","moa":"JAK","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"NTLA-2002","moa":"Kallikrein B1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"BCX4161","moa":"Kallikrein-2; Kininogen-1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"BCX4161","moa":"Kallikrein-2; Kininogen-1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"BCX4161","moa":"Kallikrein-2; Kininogen-1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"BCX4161","moa":"Kallikrein-2; Kininogen-1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Lenire Biosciences","sponsor":"University College London","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Licensing Agreement","leadProduct":"VSN16R","moa":"KCa channel","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Lenire Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Lenire Biosciences \/ University College London","highestDevelopmentStatusID":"4","companyTruncated":"Lenire Biosciences \/ University College London"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Avrobio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Collaboration","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Avrobio","highestDevelopmentStatusID":"4","companyTruncated":"Jasper Therapeutics \/ Avrobio"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Argo Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"BW-20805","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Argo Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Argo Biopharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Argo Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Public Offering","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0.19,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.19,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Jefferies","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Jefferies"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Public Offering","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.19,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Jefferies","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Jefferies"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Public Offering","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0.16,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.19,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Jefferies","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Jefferies"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Public Offering","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0.059999999999999998,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Kaken Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Licensing Agreement","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Kaken Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Kaken Pharmaceuticals"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Pendopharm","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Licensing Agreement","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Pendopharm Division of De Pharmascience Inc","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Pendopharm Division of De Pharmascience Inc"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OPGx-001","moa":"LCA5","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Opus Genetics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Opus Genetics \/ Undisclosed"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"National Resilience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Collaboration","leadProduct":"OPGx-001","moa":"LCA5","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Opus Genetics \/ National Resilience","highestDevelopmentStatusID":"5","companyTruncated":"Opus Genetics \/ National Resilience"},{"orgOrder":0,"company":"Regenxbio","sponsor":"National Heart, Lung, and Blood Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"Aav-Hldlr Gene Therapy","moa":"LDL-cholesterol oxidation","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regenxbio \/ National Heart, Lung, and Blood Institute","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ National Heart, Lung, and Blood Institute"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Hormone","year":"2024","type":"Inapplicable","leadProduct":"Metreleptin","moa":"Leptin receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Amryt Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amryt Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Global Rare Diseases","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Hormone","year":"2024","type":"Inapplicable","leadProduct":"Metreleptin","moa":"Leptin receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Chiesi Global Rare Diseases","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chiesi Global Rare Diseases \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Chiesi Global Rare Diseases \/ Undisclosed"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Hormone","year":"2021","type":"Inapplicable","leadProduct":"Metreleptin","moa":"Leptin receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Amryt Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amryt Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Metreleptin","moa":"Leptin receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Amryt Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amryt Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Medison Pharma","sponsor":"Amryt Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Hormone","year":"2021","type":"Agreement","leadProduct":"Metreleptin","moa":"Leptin receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Medison Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Medison Pharma \/ Amryt Pharma","highestDevelopmentStatusID":"15","companyTruncated":"Medison Pharma \/ Amryt Pharma"},{"orgOrder":0,"company":"AEGERION","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2019","type":"Inapplicable","leadProduct":"Metreleptin","moa":"Leptin receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"AEGERION","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AEGERION \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"AEGERION \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Mibavademab","moa":"Leptin receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"REGN4461","moa":"Leptin receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Northwell Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2011","type":"Inapplicable","leadProduct":"L-Leucine","moa":"Leucine--tRNA ligase, mitochondrial; tRNA wybutosine-synthesizing protein 4; Leucine carboxyl methyltransferase 1; Leucine--tRNA ligase, cytoplasmic; Branched-chain-amino-acid aminotransferase, mitochondrial; Branched-chain-amino-acid aminotransferase, cytosolic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Northwell Health","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Northwell Health \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Northwell Health \/ Undisclosed"},{"orgOrder":0,"company":"Kamada","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Protein","year":"2020","type":"Agreement","leadProduct":"Alpha-1 Antitrypsin","moa":"ELA2\/HIV-1 protease\/Serine protease||Leukocyte elastase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Kamada","amount2":0.029999999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Infusion","sponsorNew":"Kamada \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Kamada \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Kamada","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Alpha-1 Antitrypsin","moa":"ELA2\/HIV-1 protease\/Serine protease||Leukocyte elastase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Kamada","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kamada \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Kamada \/ Undisclosed"},{"orgOrder":0,"company":"Kamada","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Alpha-1 Antitrypsin","moa":"ELA2\/HIV-1 protease\/Serine protease||Leukocyte elastase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Kamada","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kamada \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Kamada \/ Undisclosed"},{"orgOrder":0,"company":"Kamada","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Alpha-1 Antitrypsin","moa":"ELA2\/HIV-1 protease\/Serine protease||Leukocyte elastase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Kamada","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kamada \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Kamada \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Alpha1-proteinase Inhibitor","moa":"Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Alpha1-proteinase Inhibitor","moa":"Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Syneos Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Alvelestat","moa":"Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Mereo BioPharma Group \/ Syneos Health","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Syneos Health"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Alvelestat","moa":"Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Mereo BioPharma Group \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Undisclosed"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Alvelestat","moa":"Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Mereo BioPharma Group \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Undisclosed"},{"orgOrder":0,"company":"University of Alabama at Birmingham","sponsor":"National Institutes of Health | Mereo BioPharma Group | National Center for Advancing Translational Sciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Alvelestat","moa":"Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of Alabama at Birmingham","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"University of Alabama at Birmingham \/ National Institutes of Health | Mereo BioPharma Group | National Center for Advancing Translational Sciences","highestDevelopmentStatusID":"8","companyTruncated":"University of Alabama at Birmingham \/ National Institutes of Health | Mereo BioPharma Group | National Center for Advancing Translational Sciences"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Human Alpha1-Proteinase Inhibitor","moa":"Leukocyte elastase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Celtaxsys, Inc.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Acebilustat","moa":"Leukotriene A4 hydrolase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Celtaxsys, Inc.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Celtaxsys, Inc. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Celtaxsys, Inc. \/ Undisclosed"},{"orgOrder":0,"company":"Celtaxsys, Inc.","sponsor":"Celerion","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Acebilustat","moa":"Leukotriene A4 hydrolase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Celtaxsys, Inc.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Celtaxsys, Inc. \/ Celerion","highestDevelopmentStatusID":"8","companyTruncated":"Celtaxsys, Inc. \/ Celerion"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"BIIL-284","moa":"Leukotriene B4 receptor 1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"BIIL-284","moa":"Leukotriene B4 receptor 1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"Prothena","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Birtamimab","moa":"||Light chain aggregates","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prothena","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prothena \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prothena \/ Undisclosed"},{"orgOrder":0,"company":"Prothena","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2022","type":"Public Offering","leadProduct":"Birtamimab","moa":"||Light chain aggregates","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prothena","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prothena \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Prothena \/ Jefferies"},{"orgOrder":0,"company":"Prothena","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Birtamimab","moa":"Light chain aggregates","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prothena","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Prothena \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prothena \/ Undisclosed"},{"orgOrder":0,"company":"Prothena","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2014","type":"Inapplicable","leadProduct":"NEOD001","moa":"Light chain aggregates","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prothena","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prothena \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prothena \/ Undisclosed"},{"orgOrder":0,"company":"Prothena","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2015","type":"Inapplicable","leadProduct":"NEOD001","moa":"Light chain aggregates","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prothena","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prothena \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prothena \/ Undisclosed"},{"orgOrder":0,"company":"Prothena","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2015","type":"Inapplicable","leadProduct":"NEOD001","moa":"Light chain aggregates","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prothena","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prothena \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prothena \/ Undisclosed"},{"orgOrder":0,"company":"Prothena","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"NEOD001","moa":"Light chain aggregates","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prothena","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prothena \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prothena \/ Undisclosed"},{"orgOrder":0,"company":"Prothena","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2012","type":"Inapplicable","leadProduct":"NEOD001","moa":"Light chain aggregates","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prothena","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prothena \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prothena \/ Undisclosed"},{"orgOrder":0,"company":"Retrotope","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2020","type":"Inapplicable","leadProduct":"Linoleic Acid","moa":"Lipid peroxidation","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Retrotope","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Retrotope \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Retrotope \/ Undisclosed"},{"orgOrder":0,"company":"Peptris Technologies","sponsor":"Revio Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Undisclosed","year":"2025","type":"Licensing Agreement","leadProduct":"RT001","moa":"Lipid peroxidation","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Peptris Technologies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Peptris Technologies \/ Revio Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Peptris Technologies \/ Revio Therapeutics"},{"orgOrder":0,"company":"Retrotope","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"RT001","moa":"Lipid peroxidation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Retrotope","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Retrotope \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Retrotope \/ Undisclosed"},{"orgOrder":0,"company":"Retrotope","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"RT001","moa":"Lipid peroxidation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Retrotope","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Retrotope \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Retrotope \/ Undisclosed"},{"orgOrder":0,"company":"Cellaion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell & Gene Therapy","year":"2015","type":"Inapplicable","leadProduct":"Heterologous Human Adult Liver Derived Progenitor Cell","moa":"Liver progenitor cells","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cellaion","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cellaion \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cellaion \/ Undisclosed"},{"orgOrder":0,"company":"Cellaion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell & Gene Therapy","year":"2013","type":"Inapplicable","leadProduct":"Heterologous Human Adult Liver Derived Progenitor Cell","moa":"Liver progenitor cells","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cellaion","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Paste","sponsorNew":"Cellaion \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cellaion \/ Undisclosed"},{"orgOrder":0,"company":"Peking Union Medical College Hospital","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Daratumumab","moa":"Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Peking Union Medical College Hospital","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Peking Union Medical College Hospital \/ Johnson & Johnson Innovative Medicine","highestDevelopmentStatusID":"8","companyTruncated":"Peking Union Medical College Hospital \/ Johnson & Johnson Innovative Medicine"},{"orgOrder":0,"company":"Stichting European Myeloma Network","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Daratumumab","moa":"Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Stichting European Myeloma Network","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stichting European Myeloma Network \/ Johnson & Johnson Innovative Medicine","highestDevelopmentStatusID":"8","companyTruncated":"Stichting European Myeloma Network \/ Johnson & Johnson Innovative Medicine"},{"orgOrder":0,"company":"Genmab","sponsor":"GSK","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Daratumumab","moa":"||Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Genmab","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genmab \/ GSK","highestDevelopmentStatusID":"10","companyTruncated":"Genmab \/ GSK"},{"orgOrder":0,"company":"Genmab","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Daratumumab","moa":"||Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Genmab","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genmab \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Genmab \/ Undisclosed"},{"orgOrder":0,"company":"Children's National Research Institute","sponsor":"Alberta Children's Hospital | The Hospital for Sick Children | Levine Children's Hospital | Ann & Robert H Lurie Children's Hospital of Chicago | Nationwide Children\u2019s Hospital | Children's Hospital at Montefiore | Doris Duke Foundation | Johnson & Johnso","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Daratumumab","moa":"Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Children's National Research Institute","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Children's National Research Institute \/ Alberta Children's Hospital | The Hospital for Sick Children | Levine Children's Hospital | Ann & Robert H Lurie Children's Hospital of Chicago | Nationwide Children\u2019s Hospital | Children's Hospital at Montefiore | Doris Duke Foundation | Johnson & Johnso","highestDevelopmentStatusID":"8","companyTruncated":"Children's National Research Institute \/ Alberta Children's Hospital | The Hospital for Sick Children | Levine Children's Hospital | Ann & Robert H Lurie Children's Hospital of Chicago | Nationwide Children\u2019s Hospital | Children's Hospital at Montefiore | Doris Duke Foundation | Johnson & Johnso"},{"orgOrder":0,"company":"Boston Medical Center","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"Daratumumab","moa":"Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Boston Medical Center","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Boston Medical Center \/ Johnson & Johnson Innovative Medicine","highestDevelopmentStatusID":"7","companyTruncated":"Boston Medical Center \/ Johnson & Johnson Innovative Medicine"},{"orgOrder":0,"company":"MD Anderson Cancer Center","sponsor":"Johnson & Johnson Innovative Medicine | Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Daratumumab","moa":"Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Phase I","graph3":"MD Anderson Cancer Center","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MD Anderson Cancer Center \/ Johnson & Johnson Innovative Medicine | Takeda Pharmaceutical","highestDevelopmentStatusID":"6","companyTruncated":"MD Anderson Cancer Center \/ Johnson & Johnson Innovative Medicine | Takeda Pharmaceutical"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Daratumumab","moa":"||Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Daratumumab","moa":"||Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Halozyme Therapeutics","sponsor":"GSK","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Daratumumab","moa":"||Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Halozyme Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Halozyme Therapeutics \/ GSK","highestDevelopmentStatusID":"10","companyTruncated":"Halozyme Therapeutics \/ GSK"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Daratumumab","moa":"||Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Daratumumab","moa":"||Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Daratumumab","moa":"||Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Daratumumab","moa":"||Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Centocor","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Daratumumab","moa":"||Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Centocor","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Centocor \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Centocor \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Daratumumab","moa":"Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Daratumumab","moa":"Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Intergroupe Francophone du Myelome","sponsor":"Sanofi | Bristol Myers Squibb","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Isatuximab","moa":"Lymphocyte differentiation antigen CD38","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Intergroupe Francophone du Myelome","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Intergroupe Francophone du Myelome \/ Sanofi | Bristol Myers Squibb","highestDevelopmentStatusID":"8","companyTruncated":"Intergroupe Francophone du Myelome \/ Sanofi | Bristol Myers Squibb"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PR001","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Prevail Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Acquisition","leadProduct":"Methylprednisolone","moa":"||Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":1.04,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":1.04,"dosageForm":"Injection","sponsorNew":"Prevail Therapeutics \/ Eli Lilly","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Eli Lilly"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Methylprednisolone","moa":"||Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Prevail Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PR001","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Prevail Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Pazopanib Hydrochloride","moa":"Macrophage colony stimulating factor receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"Glycomine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Undisclosed"},{"orgOrder":0,"company":"Glycomine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Undisclosed"},{"orgOrder":0,"company":"Glycomine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Undisclosed"},{"orgOrder":0,"company":"Glycomine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Undisclosed"},{"orgOrder":0,"company":"Glycomine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Undisclosed"},{"orgOrder":0,"company":"Glycomine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Undisclosed"},{"orgOrder":0,"company":"Glycomine","sponsor":"CTI Life Sciences Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Series C Financing","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0.12,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ CTI Life Sciences Fund","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ CTI Life Sciences Fund"},{"orgOrder":0,"company":"Glycomine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Undisclosed"},{"orgOrder":0,"company":"Glycomine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Undisclosed"},{"orgOrder":0,"company":"CervoMed","sponsor":"Voisin Consulting","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Neflamapimod","moa":"MAP kinase p38 alpha","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CervoMed","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"CervoMed \/ Voisin Consulting","highestDevelopmentStatusID":"8","companyTruncated":"CervoMed \/ Voisin Consulting"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"FSHD Society","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Collaboration","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ FSHD Society","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ FSHD Society"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0.13,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Goldman Sachs & Co","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Collaboration","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":1.0600000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":1.0600000000000001,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Sanofi","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Losmapimod","moa":"MAP kinase p38 alpha | MAP kinase p38 beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fulcrum Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fulcrum Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fulcrum Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"AZD0120","moa":"MAPK pathway","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Allomek","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"CIP-137401","moa":"MAPK\/ERK kinase kinase (MAP3K)","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Allomek","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Allomek \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Allomek \/ Undisclosed"},{"orgOrder":0,"company":"Allomek","sponsor":"Camac Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Acquisition","leadProduct":"CIP-137401","moa":"MAPK\/ERK kinase kinase (MAP3K)","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Allomek","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Allomek \/ Camac Partners","highestDevelopmentStatusID":"5","companyTruncated":"Allomek \/ Camac Partners"},{"orgOrder":0,"company":"Aclaris Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"ATI-450","moa":"MAPK2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aclaris Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aclaris Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aclaris Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Unity Health Toronto","sponsor":"Barrow Neurological Institute | Duke University | Germans Trias i Pujol Hospital | The University of Pittsburgh School of Medicine | Sunnybrook Health Sciences Centre","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Doxycycline Hyclate","moa":"Matrix metalloproteinase 8 | Matrix metalloproteinase 13 | Matrix metalloproteinase 7 | Matrix metalloproteinase-1 | Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Unity Health Toronto","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Unity Health Toronto \/ Barrow Neurological Institute | Duke University | Germans Trias i Pujol Hospital | The University of Pittsburgh School of Medicine | Sunnybrook Health Sciences Centre","highestDevelopmentStatusID":"8","companyTruncated":"Unity Health Toronto \/ Barrow Neurological Institute | Duke University | Germans Trias i Pujol Hospital | The University of Pittsburgh School of Medicine | Sunnybrook Health Sciences Centre"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"Andecaliximab","moa":"Matrix metalloproteinase 9","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Gilead Sciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Gilead Sciences \/ Undisclosed"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Undisclosed"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Undisclosed"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Columbus Venture Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2023","type":"Series B Financing","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Columbus Venture Partners","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Columbus Venture Partners"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Undisclosed"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Undisclosed"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Undisclosed"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"EIC Accelerator","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2022","type":"Funding","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ EIC Accelerator","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ EIC Accelerator"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Atumelnant","moa":"ACTH||MC2R","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Atumelnant","moa":"MC2R","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Atumelnant","moa":"MC2R","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Atumelnant","moa":"MC2R","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Atumelnant","moa":"MC2R","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Atumelnant","moa":"MC2R","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Atumelnant","moa":"MC2R","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Atumelnant","moa":"MC2R","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Atumelnant","moa":"MC2R","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Atumelnant","moa":"MC2R","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"MCO-010","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Sonpiretigene Isteparvovec","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Sonpiretigene Isteparvovec","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Sonpiretigene Isteparvovec","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Sonpiretigene Isteparvovec","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Sonpiretigene Isteparvovec","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Sonpiretigene Isteparvovec","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Verana Health","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Collaboration","leadProduct":"Sonpiretigene Isteparvovec","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Verana Health","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Verana Health"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"vMCO-010","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"vMCO-010","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Nanoscope Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Partnership","leadProduct":"vMCO-010","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Charles River Laboratories, Inc \/ Nanoscope Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Charles River Laboratories, Inc \/ Nanoscope Therapeutics"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"vMCO-010","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"vMCO-010","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"vMCO-010","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fosun Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"FCN-159","moa":"MEK1 \/ MEK2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fosun Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fosun Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fosun Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Fosun Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"FCN-159","moa":"MEK1 \/ MEK2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fosun Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fosun Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Fosun Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"REC-4881","moa":"MEK1 receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Recursion Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Recursion Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Recursion Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"REC-4881","moa":"MEK1 receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Recursion Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Recursion Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Recursion Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"REC-4881","moa":"MEK1 receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Recursion Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Recursion Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Recursion Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Pasithea Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"PAS-004","moa":"MEK1\/2 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pasithea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pasithea Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Pasithea Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Pasithea Therapeutics","sponsor":"Novotech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"PAS-004","moa":"MEK1\/2 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pasithea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pasithea Therapeutics \/ Novotech","highestDevelopmentStatusID":"6","companyTruncated":"Pasithea Therapeutics \/ Novotech"},{"orgOrder":0,"company":"Pasithea Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"PAS-004","moa":"MEK1\/2 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pasithea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pasithea Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Pasithea Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Pasithea Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"PAS-004","moa":"MEK1\/2 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pasithea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pasithea Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Pasithea Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Pasithea Therapeutics","sponsor":"Novotech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"PAS-004","moa":"MEK1\/2 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pasithea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pasithea Therapeutics \/ Novotech","highestDevelopmentStatusID":"6","companyTruncated":"Pasithea Therapeutics \/ Novotech"},{"orgOrder":0,"company":"Pasithea Therapeutics","sponsor":"WuXi AppTec","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Agreement","leadProduct":"PAS-004","moa":"MEK1\/2 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pasithea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pasithea Therapeutics \/ WuXi AppTec","highestDevelopmentStatusID":"6","companyTruncated":"Pasithea Therapeutics \/ WuXi AppTec"},{"orgOrder":0,"company":"Fosun Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Luvometinib","moa":"MEK1|MEK2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Fosun Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fosun Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Fosun Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2012","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2010","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Clinuvel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Afamelanotide","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Clinuvel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Clinuvel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Clinuvel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Mitsubishi Tanabe Pharma America","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Dersimelagon","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Mitsubishi Tanabe Pharma America","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mitsubishi Tanabe Pharma America \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Mitsubishi Tanabe Pharma America \/ Undisclosed"},{"orgOrder":0,"company":"Mitsubishi Tanabe Pharma America","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Dersimelagon","moa":"Melanocortin receptor 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Mitsubishi Tanabe Pharma America","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mitsubishi Tanabe Pharma America \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Mitsubishi Tanabe Pharma America \/ Undisclosed"},{"orgOrder":0,"company":"Mallinckrodt Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2018","type":"Inapplicable","leadProduct":"Acthar Gel","moa":"Melanocortin receptor 2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mallinckrodt Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Mallinckrodt Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Mallinckrodt Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2014","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Xinvento","sponsor":"Rhythm Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Peptide, Unconjugated","year":"2023","type":"Acquisition","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Xinvento","amount2":0.20999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20999999999999999,"dosageForm":"Injection","sponsorNew":"Xinvento \/ Rhythm Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Xinvento \/ Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"RareStone Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Licensing Agreement","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rhythm Pharmaceuticals","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ RareStone Group","highestDevelopmentStatusID":"10","companyTruncated":"Rhythm Pharmaceuticals \/ RareStone Group"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Medison Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Partnership","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Medison Pharma","highestDevelopmentStatusID":"15","companyTruncated":"Rhythm Pharmaceuticals \/ Medison Pharma"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Rhythm Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Flex Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"FLX-787","moa":"Melanoma antigen gp75 (TYRP1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Flex Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Flex Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Flex Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Johns Hopkins University","sponsor":"Neurofibromatosis Therapeutic Acceleration Program | Amgen Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Talimogene Laherparepvec","moa":"Melanoma cell","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Johns Hopkins University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johns Hopkins University \/ Neurofibromatosis Therapeutic Acceleration Program | Amgen Inc","highestDevelopmentStatusID":"6","companyTruncated":"Johns Hopkins University \/ Neurofibromatosis Therapeutic Acceleration Program | Amgen Inc"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Tasimelteon","moa":"Melatonin receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vanda Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vanda Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vanda Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tasimelteon","moa":"Melatonin receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vanda Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vanda Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vanda Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"MSN Laboratories","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Licensing Agreement","leadProduct":"Tasimelteon","moa":"Melatonin receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vanda Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vanda Pharmaceuticals \/ MSN Laboratories","highestDevelopmentStatusID":"15","companyTruncated":"Vanda Pharmaceuticals \/ MSN Laboratories"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tasimelteon","moa":"Melatonin receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vanda Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vanda Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vanda Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"30 Technology","sponsor":"ConvaTec","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2023","type":"Divestment","leadProduct":"RESP301","moa":"Membrane fusion protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"30 Technology","amount2":0.22,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.22,"dosageForm":"Solution for Inhalation","sponsorNew":"30 Technology \/ ConvaTec","highestDevelopmentStatusID":"8","companyTruncated":"30 Technology \/ ConvaTec"},{"orgOrder":0,"company":"Hope Biosciences","sponsor":"Hope Biosciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"HB-adMSC","moa":"Mesenchymal stem cells","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Hope Biosciences","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hope Biosciences \/ Hope Biosciences","highestDevelopmentStatusID":"1","companyTruncated":"Hope Biosciences \/ Hope Biosciences"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Basimglurant","moa":"Metabotropic glutamate receptor 5","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Basimglurant","moa":"Metabotropic glutamate receptor 5","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Mavoglurant","moa":"Metabotropic glutamate receptor 5","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Mavoglurant","moa":"Metabotropic glutamate receptor 5","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Mavoglurant","moa":"Metabotropic glutamate receptor 5","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Mavoglurant","moa":"Metabotropic glutamate receptor 5","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Mavoglurant","moa":"Metabotropic glutamate receptor 5","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Seaside Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"STX107","moa":"Metabotropic glutamate receptor 5","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Seaside Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Seaside Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Seaside Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Zafgen, Inc.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Beloranib","moa":"Methionine aminopeptidase 2","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zafgen, Inc.","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zafgen, Inc. \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Zafgen, Inc. \/ Undisclosed"},{"orgOrder":0,"company":"Aevi Genomic Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"NFC-1","moa":"mGluR5","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Aevi Genomic Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Aevi Genomic Medicine \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Aevi Genomic Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Alaxia","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Hypothiocyanite","moa":"Microbial enzymes","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Alaxia","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Inhalation","sponsorNew":"Alaxia \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Alaxia \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Citigroup","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Private Placement","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Solid Biosciences","amount2":0.11,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Citigroup","highestDevelopmentStatusID":"10","companyTruncated":"Solid Biosciences \/ Citigroup"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Forge Biologics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Partnership","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Forge Biologics","highestDevelopmentStatusID":"10","companyTruncated":"Solid Biosciences \/ Forge Biologics"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Perceptive Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Private Placement","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Solid Biosciences","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Perceptive Advisors","highestDevelopmentStatusID":"10","companyTruncated":"Solid Biosciences \/ Perceptive Advisors"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Armatus Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Licensing Agreement","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Solid Biosciences \/ Armatus Bio","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Armatus Bio"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Public Offering","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0.13,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Jefferies"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Collaboration","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0.29999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.29999999999999999,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2016","type":"Inapplicable","leadProduct":"Lademirsen","moa":"microRNA 21","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Collaboration","leadProduct":"Lademirsen","moa":"microRNA 21","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Regulus Therapeutics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Sanofi","highestDevelopmentStatusID":"8","companyTruncated":"Regulus Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Agreement","leadProduct":"Lademirsen","moa":"microRNA 21","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Sanofi","highestDevelopmentStatusID":"8","companyTruncated":"Regulus Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Collaboration","leadProduct":"Lademirsen","moa":"microRNA 21","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Regulus Therapeutics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Sanofi","highestDevelopmentStatusID":"8","companyTruncated":"Regulus Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"RGLS4326","moa":"microRNA hsa-miR-17 (MIR17)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"RGLS4326","moa":"microRNA hsa-miR-17 (MIR17)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"RGLS4326","moa":"microRNA hsa-miR-17 (MIR17)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"RGLS4326","moa":"microRNA hsa-miR-17 (MIR17)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"RGLS4326","moa":"microRNA hsa-miR-17 (MIR17)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Lomitapide","moa":"Microsomal triglyceride transfer protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amryt Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amryt Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Lomitapide","moa":"Microsomal triglyceride transfer protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amryt Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amryt Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AEGERION","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Lomitapide","moa":"Microsomal triglyceride transfer protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AEGERION","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AEGERION \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AEGERION \/ Undisclosed"},{"orgOrder":0,"company":"AEGERION","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Lomitapide","moa":"Microsomal triglyceride transfer protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AEGERION","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AEGERION \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AEGERION \/ Undisclosed"},{"orgOrder":0,"company":"AEGERION","sponsor":"University of Pennsylvania | Doris Duke Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Lomitapide","moa":"Microsomal triglyceride transfer protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AEGERION","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AEGERION \/ University of Pennsylvania | Doris Duke Foundation","highestDevelopmentStatusID":"10","companyTruncated":"AEGERION \/ University of Pennsylvania | Doris Duke Foundation"},{"orgOrder":0,"company":"Sperogenix Therapeutics","sponsor":"Santhera Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sperogenix Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Sperogenix Therapeutics \/ Santhera Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Sperogenix Therapeutics \/ Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"ASTE","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ ASTE","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ ASTE"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"GENESIS Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ GENESIS Pharma","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ GENESIS Pharma"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Highbridge Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Financing","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Highbridge Capital Management","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Highbridge Capital Management"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"ReveraGen BioPharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ ReveraGen BioPharma","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ ReveraGen BioPharma"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"ReveraGen BioPharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ ReveraGen BioPharma","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ ReveraGen BioPharma"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Sperogenix Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Licensing Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Sperogenix Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Sperogenix Therapeutics"},{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"Santhera Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Licensing Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Idorsia Pharmaceuticals","amount2":0.01,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Suspension","sponsorNew":"Idorsia Pharmaceuticals \/ Santhera Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Idorsia Pharmaceuticals \/ Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Catalyst Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Catalyst Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Catalyst Pharmaceuticals"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"R-Bridge Healthcare Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Financing","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0.040000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ R-Bridge Healthcare Fund","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ R-Bridge Healthcare Fund"},{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"R-Bridge Healthcare Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Idorsia Pharmaceuticals","amount2":0.029999999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Suspension","sponsorNew":"Idorsia Pharmaceuticals \/ R-Bridge Healthcare Fund","highestDevelopmentStatusID":"15","companyTruncated":"Idorsia Pharmaceuticals \/ R-Bridge Healthcare Fund"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Clinigen Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Clinigen Group","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Clinigen Group"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Catalyst Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Licensing Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0.23000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Catalyst Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Catalyst Pharmaceuticals"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Catalyst Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Licensing Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0.23000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Catalyst Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Catalyst Pharmaceuticals"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Highbridge Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Private Placement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Highbridge Capital Management","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Highbridge Capital Management"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"ReveraGen BioPharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ ReveraGen BioPharma","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ ReveraGen BioPharma"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"ReveraGen BioPharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ ReveraGen BioPharma","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ ReveraGen BioPharma"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"ReveraGen BioPharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ ReveraGen BioPharma","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ ReveraGen BioPharma"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"ReveraGen BioPharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ ReveraGen BioPharma","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ ReveraGen BioPharma"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"ReveraGen BioPharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ ReveraGen BioPharma","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ ReveraGen BioPharma"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"ReveraGen BioPharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ ReveraGen BioPharma","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ ReveraGen BioPharma"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"ReveraGen BioPharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ ReveraGen BioPharma","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ ReveraGen BioPharma"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"ReveraGen BioPharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ ReveraGen BioPharma","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ ReveraGen BioPharma"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Gen Pharmaceuticals and Health Products","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Gen Pharmaceuticals and Health Products","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Gen Pharmaceuticals and Health Products"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Uniphar","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Uniphar","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Uniphar"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Ikris Pharma Network","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Ikris Pharma Network","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Ikris Pharma Network"},{"orgOrder":0,"company":"ReveraGen BioPharma","sponsor":"The University of Pittsburgh School of Medicine | National Institute of Arthritis and Musculoskeletal and Skin Diseases | National Institute of Neurological Disorders and Stroke | Cooperative International Neuromuscular Research Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ReveraGen BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"ReveraGen BioPharma \/ The University of Pittsburgh School of Medicine | National Institute of Arthritis and Musculoskeletal and Skin Diseases | National Institute of Neurological Disorders and Stroke | Cooperative International Neuromuscular Research Group","highestDevelopmentStatusID":"8","companyTruncated":"ReveraGen BioPharma \/ The University of Pittsburgh School of Medicine | National Institute of Arthritis and Musculoskeletal and Skin Diseases | National Institute of Neurological Disorders and Stroke | Cooperative International Neuromuscular Research Group"},{"orgOrder":0,"company":"ReveraGen BioPharma","sponsor":"The University of Pittsburgh School of Medicine | National Institute of Neurological Disorders and Stroke | Cooperative International Neuromuscular Research Group | National Institute of Arthritis and Musculoskeletal and Skin Diseases","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ReveraGen BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"ReveraGen BioPharma \/ The University of Pittsburgh School of Medicine | National Institute of Neurological Disorders and Stroke | Cooperative International Neuromuscular Research Group | National Institute of Arthritis and Musculoskeletal and Skin Diseases","highestDevelopmentStatusID":"8","companyTruncated":"ReveraGen BioPharma \/ The University of Pittsburgh School of Medicine | National Institute of Neurological Disorders and Stroke | Cooperative International Neuromuscular Research Group | National Institute of Arthritis and Musculoskeletal and Skin Diseases"},{"orgOrder":0,"company":"ReveraGen BioPharma","sponsor":"The University of Pittsburgh School of Medicine | Cooperative International Neuromuscular Research Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ReveraGen BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"ReveraGen BioPharma \/ The University of Pittsburgh School of Medicine | Cooperative International Neuromuscular Research Group","highestDevelopmentStatusID":"8","companyTruncated":"ReveraGen BioPharma \/ The University of Pittsburgh School of Medicine | Cooperative International Neuromuscular Research Group"},{"orgOrder":0,"company":"ReveraGen BioPharma","sponsor":"European Union | Cooperative International Neuromuscular Research Group | Newcastle University | The University of Pittsburgh School of Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ReveraGen BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"ReveraGen BioPharma \/ European Union | Cooperative International Neuromuscular Research Group | Newcastle University | The University of Pittsburgh School of Medicine","highestDevelopmentStatusID":"8","companyTruncated":"ReveraGen BioPharma \/ European Union | Cooperative International Neuromuscular Research Group | Newcastle University | The University of Pittsburgh School of Medicine"},{"orgOrder":0,"company":"ReveraGen BioPharma","sponsor":"Santhera Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ReveraGen BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"ReveraGen BioPharma \/ Santhera Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"ReveraGen BioPharma \/ Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Catalyst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Catalyst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Catalyst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Catalyst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Pharmaceuticals","sponsor":"KYE Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Licensing Agreement","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Catalyst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Catalyst Pharmaceuticals \/ KYE Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Catalyst Pharmaceuticals \/ KYE Pharmaceuticals"},{"orgOrder":0,"company":"KYE Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KYE Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"KYE Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KYE Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"ReveraGen BioPharma","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Funding","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ReveraGen BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"ReveraGen BioPharma \/ National Institutes of Health","highestDevelopmentStatusID":"8","companyTruncated":"ReveraGen BioPharma \/ National Institutes of Health"},{"orgOrder":0,"company":"ReveraGen BioPharma","sponsor":"Santhera Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor | Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ReveraGen BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"ReveraGen BioPharma \/ Santhera Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"ReveraGen BioPharma \/ Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Acquisition","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":1.7,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":1.7,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Acquisition","leadProduct":"Farabursen","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":1.7,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":1.7,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"RGLS8429","moa":"miR-17","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regulus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"RG012","moa":"miR-21","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Indiana Hemophilia &Thrombosis Center, Inc.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Metformin","moa":"Mitochondrial complex I (NADH dehydrogenase)","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Indiana Hemophilia &Thrombosis Center, Inc.","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Indiana Hemophilia &Thrombosis Center, Inc. \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Indiana Hemophilia &Thrombosis Center, Inc. \/ Undisclosed"},{"orgOrder":0,"company":"Baylor College of Medicine","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Metformin","moa":"Mitochondrial complex I (NADH dehydrogenase)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Baylor College of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baylor College of Medicine \/ Pfizer Inc","highestDevelopmentStatusID":"6","companyTruncated":"Baylor College of Medicine \/ Pfizer Inc"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Association Fran\u00e7aise contre les Myopathies","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Olesoxime","moa":"Mitochondrial dysfunction","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid, Suspension","sponsorNew":"F. Hoffmann-La Roche \/ Association Fran\u00e7aise contre les Myopathies","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Association Fran\u00e7aise contre les Myopathies"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Olesoxime","moa":"Mitochondrial dysfunction","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"University of Toronto","sponsor":"Ferring Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Coenzyme Q10","moa":"Mitochondrial electron transport chain","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Toronto","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Toronto \/ Ferring Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"University of Toronto \/ Ferring Pharmaceuticals"},{"orgOrder":0,"company":"Cooperative International Neuromuscular Research Group","sponsor":"US Department of Defense","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Coenzyme Q10","moa":"Mitochondrial electron transport chain","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Cooperative International Neuromuscular Research Group","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cooperative International Neuromuscular Research Group \/ US Department of Defense","highestDevelopmentStatusID":"9","companyTruncated":"Cooperative International Neuromuscular Research Group \/ US Department of Defense"},{"orgOrder":0,"company":"University of Primorska","sponsor":"Biovis d.o.o.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SLOVENIA","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Coenzyme Q10","moa":"Mitochondrial electron transport chain","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Primorska","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Primorska \/ Biovis d.o.o.","highestDevelopmentStatusID":"1","companyTruncated":"University of Primorska \/ Biovis d.o.o."},{"orgOrder":0,"company":"Epirium","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Plant Extract\/Herbal","year":"2020","type":"Inapplicable","leadProduct":"Epicatechin","moa":"Mitochondrial protein unspecific (MP)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Epirium","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Epirium \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Epirium \/ Undisclosed"},{"orgOrder":0,"company":"Epirium","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Plant Extract\/Herbal","year":"2020","type":"Inapplicable","leadProduct":"Epicatechin","moa":"Mitochondrial protein unspecific (MP)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Epirium","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Epirium \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Epirium \/ Undisclosed"},{"orgOrder":0,"company":"Piramal Pharma Solutions","sponsor":"Epirium","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Plant Extract\/Herbal","year":"2020","type":"Collaboration","leadProduct":"Epicatechin","moa":"Mitochondrial protein unspecific (MP)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Piramal Pharma Solutions","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Piramal Pharma Solutions \/ Epirium","highestDevelopmentStatusID":"6","companyTruncated":"Piramal Pharma Solutions \/ Epirium"},{"orgOrder":0,"company":"Epirium","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Plant Extract\/Herbal","year":"2020","type":"Inapplicable","leadProduct":"Epicatechin","moa":"Mitochondrial protein unspecific (MP)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Epirium","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Epirium \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Epirium \/ Undisclosed"},{"orgOrder":0,"company":"Ralitza Gavrilova","sponsor":"Cardero Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Plant Extract\/Herbal","year":"2016","type":"Inapplicable","leadProduct":"Epicatechin","moa":"Mitochondrial protein unspecific (MP)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ralitza Gavrilova","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Ralitza Gavrilova \/ Cardero Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Ralitza Gavrilova \/ Cardero Therapeutics"},{"orgOrder":0,"company":"Craig McDonald","sponsor":"Cardero Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Plant Extract\/Herbal","year":"2017","type":"Inapplicable","leadProduct":"Epicatechin","moa":"Mitochondrial protein unspecific (MP)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Craig McDonald","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Craig McDonald \/ Cardero Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Craig McDonald \/ Cardero Therapeutics"},{"orgOrder":0,"company":"Craig McDonald","sponsor":"Cardero Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Plant Extract\/Herbal","year":"2013","type":"Inapplicable","leadProduct":"Epicatechin","moa":"Mitochondrial protein unspecific (MP)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Craig McDonald","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Craig McDonald \/ Cardero Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Craig McDonald \/ Cardero Therapeutics"},{"orgOrder":0,"company":"Craig McDonald","sponsor":"Cardero Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Plant Extract\/Herbal","year":"2016","type":"Inapplicable","leadProduct":"Epicatechin","moa":"Mitochondrial protein unspecific (MP)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Craig McDonald","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Craig McDonald \/ Cardero Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Craig McDonald \/ Cardero Therapeutics"},{"orgOrder":0,"company":"Shanghai Kechow Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tunlametinib","moa":"ERK activator kinase (MEK)||Mitogen-activated protein kinase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Shanghai Kechow Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Shanghai Kechow Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Shanghai Kechow Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Elizabeth Berry-Kravis","sponsor":"FRAXA Research Foundation | Purposeful IKE","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Dihydroergotoxine Mesylate","moa":"Monoamine oxidase (MAO)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Elizabeth Berry-Kravis","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Elizabeth Berry-Kravis \/ FRAXA Research Foundation | Purposeful IKE","highestDevelopmentStatusID":"8","companyTruncated":"Elizabeth Berry-Kravis \/ FRAXA Research Foundation | Purposeful IKE"},{"orgOrder":0,"company":"Saniona","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Tesofensine","moa":"Monoamine transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Saniona \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Undisclosed"},{"orgOrder":0,"company":"Saniona","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tesofensine","moa":"Monoamine transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Saniona \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Undisclosed"},{"orgOrder":0,"company":"Saniona","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Monoamine transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Saniona \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Undisclosed"},{"orgOrder":0,"company":"Saniona","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Monoamine transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Saniona \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Undisclosed"},{"orgOrder":0,"company":"Saniona","sponsor":"Foundation for Prader-Willi Research","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2021","type":"Partnership","leadProduct":"Tesofensine","moa":"||Monoamine transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Saniona \/ Foundation for Prader-Willi Research","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Foundation for Prader-Willi Research"},{"orgOrder":0,"company":"Saniona","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Monoamine transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Saniona \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Undisclosed"},{"orgOrder":0,"company":"Saniona","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Monoamine transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Saniona \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Undisclosed"},{"orgOrder":0,"company":"Saniona","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Monoamine transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Saniona \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Undisclosed"},{"orgOrder":0,"company":"Saniona","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Monoamine transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Saniona \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Undisclosed"},{"orgOrder":0,"company":"Saniona","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Monoamine transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Saniona \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Undisclosed"},{"orgOrder":0,"company":"Khondrion","sponsor":"University Ghent","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"KH176","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Khondrion","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Khondrion \/ University Ghent","highestDevelopmentStatusID":"6","companyTruncated":"Khondrion \/ University Ghent"},{"orgOrder":0,"company":"Khondrion","sponsor":"Netherlands Enterprise Agency","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2025","type":"Funding","leadProduct":"Sonlicromanol","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Khondrion","amount2":0.01,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Solution","sponsorNew":"Khondrion \/ Netherlands Enterprise Agency","highestDevelopmentStatusID":"10","companyTruncated":"Khondrion \/ Netherlands Enterprise Agency"},{"orgOrder":0,"company":"Khondrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sonlicromanol","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Khondrion","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Khondrion \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Khondrion \/ Undisclosed"},{"orgOrder":0,"company":"Khondrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Sonlicromanol","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Khondrion","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Khondrion \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Khondrion \/ Undisclosed"},{"orgOrder":0,"company":"Khondrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sonlicromanol","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Khondrion","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Khondrion \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Khondrion \/ Undisclosed"},{"orgOrder":0,"company":"Khondrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sonlicromanol","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Khondrion","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Khondrion \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Khondrion \/ Undisclosed"},{"orgOrder":0,"company":"Khondrion","sponsor":"UMDF","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2024","type":"Funding","leadProduct":"Sonlicromanol","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Khondrion","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Khondrion \/ UMDF","highestDevelopmentStatusID":"10","companyTruncated":"Khondrion \/ UMDF"},{"orgOrder":0,"company":"BioLeaders Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Vaccine","year":"2018","type":"Inapplicable","leadProduct":"BLS-M22","moa":"MSTN","graph1":"Genetic Disease","graph2":"Phase I","graph3":"BioLeaders Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioLeaders Corporation \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"BioLeaders Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Taldefgrobep Alpha","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Taldefgrobep Alpha","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Taldefgrobep Alpha","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Taldefgrobep Alpha","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Taldefgrobep Alpha","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Bristol Myers Squibb","sponsor":"Biohaven Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Licensing Agreement","leadProduct":"Taldefgrobep Alpha","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Bristol Myers Squibb","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bristol Myers Squibb \/ Biohaven Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Bristol Myers Squibb \/ Biohaven Pharmaceuticals"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"Resamirigene Bilparvovec","moa":"MTM1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Astellas Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Astellas Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Anakuria","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Acquisition","leadProduct":"AT-20494","moa":"mTORC1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Anakuria","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Anakuria \/ Johnson & Johnson Innovative Medicine","highestDevelopmentStatusID":"4","companyTruncated":"Anakuria \/ Johnson & Johnson Innovative Medicine"},{"orgOrder":0,"company":"Columbia University","sponsor":"Attralus","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Radiolabeled Compound","year":"2022","type":"Inapplicable","leadProduct":"124-I Evuzamitide","moa":"MUC1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Columbia University","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Columbia University \/ Attralus","highestDevelopmentStatusID":"8","companyTruncated":"Columbia University \/ Attralus"},{"orgOrder":0,"company":"Renovion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2020","type":"Inapplicable","leadProduct":"Ascorbic Acid","moa":"||Mucous clearance","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Renovion","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nebulizer","sponsorNew":"Renovion \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Renovion \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Tiotropium Bromide","moa":"Muscarinic acetylcholine receptor M3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Tiotropium Bromide","moa":"Muscarinic acetylcholine receptor M3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"Affiris","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRIA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"mAB C6-17","moa":"Mutant huntingtin","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Affiris","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Affiris \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Affiris \/ Undisclosed"},{"orgOrder":0,"company":"ElevateBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"LETI-101","moa":"Mutant huntingtin protein level","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ElevateBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ElevateBio \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"ElevateBio \/ Undisclosed"},{"orgOrder":0,"company":"Tenaya Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Public Offering","leadProduct":"TN-201","moa":"MYBPC3","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Tenaya Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"Tenaya Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"4","companyTruncated":"Tenaya Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Tenaya Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Public Offering","leadProduct":"TN-201","moa":"MYBPC3","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Tenaya Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"Tenaya Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"4","companyTruncated":"Tenaya Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH||Myosin","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH||Myosin","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Worldwide Clinical Trials","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH||Myosin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Edgewise Therapeutics \/ Worldwide Clinical Trials","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Worldwide Clinical Trials"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Medpace, Inc | ImagingNMD | SYSNAV Healthcare","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Medpace, Inc | ImagingNMD | SYSNAV Healthcare","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Medpace, Inc | ImagingNMD | SYSNAV Healthcare"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Medpace, Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Medpace, Inc","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Medpace, Inc"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Medpace, Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Medpace, Inc","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Medpace, Inc"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Muscular Dystrophy Association","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Muscular Dystrophy Association","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Muscular Dystrophy Association"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Braidwell LP","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Public Offering","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20000000000000001,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Braidwell LP","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Braidwell LP"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Public Offering","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0.14000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14000000000000001,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ J.P. Morgan","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ J.P. Morgan"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Medpace, Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Medpace, Inc","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Medpace, Inc"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Public Offering","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0.23999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23999999999999999,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ RA Capital Management","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ RA Capital Management"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Sevasemten","moa":"MYH","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AAVB-081","moa":"MYO7A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AAVantgarde Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Undisclosed"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AAVB-081","moa":"MYO7A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AAVantgarde Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Undisclosed"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AAVB-081","moa":"MYO7A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AAVantgarde Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Undisclosed"},{"orgOrder":0,"company":"Juvena Therapeutics","sponsor":"Mubadala Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Series A Financing","leadProduct":"JUV-161","moa":"Myogenesis","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Juvena Therapeutics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Injection","sponsorNew":"Juvena Therapeutics \/ Mubadala Capital","highestDevelopmentStatusID":"6","companyTruncated":"Juvena Therapeutics \/ Mubadala Capital"},{"orgOrder":0,"company":"Juvena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"JUV-161","moa":"Myogenesis","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Juvena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Juvena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Juvena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Juvena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"JUV-161","moa":"Myogenesis","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Juvena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Juvena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Juvena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Juvena Therapeutics","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Funding","leadProduct":"JUV-161","moa":"Myogenesis","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Juvena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Juvena Therapeutics \/ California Institute for Regenerative Medicine","highestDevelopmentStatusID":"6","companyTruncated":"Juvena Therapeutics \/ California Institute for Regenerative Medicine"},{"orgOrder":0,"company":"Keros Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"KER-065","moa":"Myostatin\/Activin A","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Keros Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Keros Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Keros Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Keros Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"KER-065","moa":"Myostatin\/Activin A","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Keros Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Keros Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Keros Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Niagen Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2024","type":"Inapplicable","leadProduct":"Nicotinamide Riboside","moa":"NAD","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Niagen Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Niagen Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Niagen Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Niagen Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2021","type":"Inapplicable","leadProduct":"Nicotinamide Riboside","moa":"NAD","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Niagen Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Niagen Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Niagen Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"YUNGJIN PHARM. CO. LTD","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"KL1333","moa":"NAD","graph1":"Genetic Disease","graph2":"Phase I","graph3":"YUNGJIN PHARM. CO. LTD","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"YUNGJIN PHARM. CO. LTD \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"YUNGJIN PHARM. CO. LTD \/ Undisclosed"},{"orgOrder":0,"company":"Abliva","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"KL1333","moa":"NAD","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Abliva","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Encapsulated","sponsorNew":"Abliva \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Abliva \/ Undisclosed"},{"orgOrder":0,"company":"Akershus University Hospital","sponsor":"The Bergesen Foundation | South-Eastern Norway Regional Health Authority | Sykehuset Innlandet HF | Oslo University Hospital | St. Olavs Hospital | Haukeland University Hospital | University Hospital of North Norway | University of Bergen","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NORWAY","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Nicotinamide Riboside","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Akershus University Hospital","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Akershus University Hospital \/ The Bergesen Foundation | South-Eastern Norway Regional Health Authority | Sykehuset Innlandet HF | Oslo University Hospital | St. Olavs Hospital | Haukeland University Hospital | University Hospital of North Norway | University of Bergen","highestDevelopmentStatusID":"8","companyTruncated":"Akershus University Hospital \/ The Bergesen Foundation | South-Eastern Norway Regional Health Authority | Sykehuset Innlandet HF | Oslo University Hospital | St. Olavs Hospital | Haukeland University Hospital | University Hospital of North Norway | University of Bergen"},{"orgOrder":0,"company":"Oslo University Hospital","sponsor":"Haukeland University Hospital | South-Eastern Norway Regional Health Authority | Klinbeforsk | The Dam Foundation | Elysium Health | NKS Olaviken Gerontopsychiatric Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NORWAY","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Nicotinamide Riboside","moa":"NAD","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Oslo University Hospital","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Oslo University Hospital \/ Haukeland University Hospital | South-Eastern Norway Regional Health Authority | Klinbeforsk | The Dam Foundation | Elysium Health | NKS Olaviken Gerontopsychiatric Hospital","highestDevelopmentStatusID":"8","companyTruncated":"Oslo University Hospital \/ Haukeland University Hospital | South-Eastern Norway Regional Health Authority | Klinbeforsk | The Dam Foundation | Elysium Health | NKS Olaviken Gerontopsychiatric Hospital"},{"orgOrder":0,"company":"Metro International Biotech","sponsor":"Children's Hospital of Philadelphia","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"MIB-626","moa":"NAD+","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Metro International Biotech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Metro International Biotech \/ Children's Hospital of Philadelphia","highestDevelopmentStatusID":"8","companyTruncated":"Metro International Biotech \/ Children's Hospital of Philadelphia"},{"orgOrder":0,"company":"Calliditas Therapeutics AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Setanaxib","moa":"NADPH oxidase 4 | NADPH oxidase 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Calliditas Therapeutics AB","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Calliditas Therapeutics AB \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Calliditas Therapeutics AB \/ Undisclosed"},{"orgOrder":0,"company":"Calliditas Therapeutics AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Setanaxib","moa":"NADPH oxidase 4 | NADPH oxidase 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Calliditas Therapeutics AB","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Calliditas Therapeutics AB \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Calliditas Therapeutics AB \/ Undisclosed"},{"orgOrder":0,"company":"Calliditas Therapeutics AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Setanaxib","moa":"NADPH oxidase 4 | NADPH oxidase 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Calliditas Therapeutics AB","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Calliditas Therapeutics AB \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Calliditas Therapeutics AB \/ Undisclosed"},{"orgOrder":0,"company":"Calliditas Therapeutics AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Setanaxib","moa":"NADPH oxidase 4 | NADPH oxidase 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Calliditas Therapeutics AB","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Calliditas Therapeutics AB \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Calliditas Therapeutics AB \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Venn Life Sciences | Institut Pasteur","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"rAAV2\/5-hNAGLU","moa":"NAGLU","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Venn Life Sciences | Institut Pasteur","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Venn Life Sciences | Institut Pasteur"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"rAAV9.CMV.hNAGLU","moa":"NAGLU","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Abeona Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Abeona Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Praxis Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Relutrigine","moa":"NaV Channel","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Praxis Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Praxis Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Praxis Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"Enavate Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Series C Financing","leadProduct":"SION-638","moa":"NBD1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sionna Therapeutics","amount2":0.17999999999999999,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Sionna Therapeutics \/ Enavate Sciences","highestDevelopmentStatusID":"6","companyTruncated":"Sionna Therapeutics \/ Enavate Sciences"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"SION-638","moa":"NBD1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sionna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sionna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sionna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"OrbiMed Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Series B Financing","leadProduct":"SION-638","moa":"||NBD1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sionna Therapeutics","amount2":0.11,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"Sionna Therapeutics \/ OrbiMed Advisors","highestDevelopmentStatusID":"6","companyTruncated":"Sionna Therapeutics \/ OrbiMed Advisors"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Public Offering","leadProduct":"SION\u2011719","moa":"NBD1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sionna Therapeutics","amount2":0.19,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.22,"dosageForm":"Tablet","sponsorNew":"Sionna Therapeutics \/ Goldman Sachs & Co","highestDevelopmentStatusID":"6","companyTruncated":"Sionna Therapeutics \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Public Offering","leadProduct":"SION\u2011719","moa":"NBD1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sionna Therapeutics","amount2":0.22,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.22,"dosageForm":"Tablet","sponsorNew":"Sionna Therapeutics \/ Goldman Sachs & Co","highestDevelopmentStatusID":"6","companyTruncated":"Sionna Therapeutics \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2012","type":"Inapplicable","leadProduct":"Larazotide Acetate","moa":"Haptoglobin (HP)||Neuronal acetylcholine receptor; alpha3\/beta4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Omigapil","moa":"neuronal apoptosis inhibitors","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"aTyr Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"ATYR1940","moa":"Neuropilin-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"aTyr Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"aTyr Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"aTyr Pharma \/ Undisclosed"},{"orgOrder":0,"company":"aTyr Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"ATYR1940","moa":"Neuropilin-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"aTyr Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"aTyr Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"aTyr Pharma \/ Undisclosed"},{"orgOrder":0,"company":"aTyr Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"ATYR1940","moa":"Neuropilin-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"aTyr Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"aTyr Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"aTyr Pharma \/ Undisclosed"},{"orgOrder":0,"company":"aTyr Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"ATYR1940","moa":"Neuropilin-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"aTyr Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"aTyr Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"aTyr Pharma \/ Undisclosed"},{"orgOrder":0,"company":"aTyr Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"ATYR1940","moa":"Neuropilin-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"aTyr Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"aTyr Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"aTyr Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Dompe Farmaceutici","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Recombinant Human Nerve Growth Factor","moa":"Neurotrophic tyrosine kinase receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Dompe Farmaceutici","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Dompe Farmaceutici \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Dompe Farmaceutici \/ Undisclosed"},{"orgOrder":0,"company":"Peak Bio","sponsor":"White Lion Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Agreement","leadProduct":"PHP-303","moa":"Neutrophil elastase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Peak Bio","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Tablet","sponsorNew":"Peak Bio \/ White Lion Capital","highestDevelopmentStatusID":"8","companyTruncated":"Peak Bio \/ White Lion Capital"},{"orgOrder":0,"company":"Peak Bio","sponsor":"White Lion Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Financing","leadProduct":"PHP-303","moa":"Neutrophil elastase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Peak Bio","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Tablet","sponsorNew":"Peak Bio \/ White Lion Capital","highestDevelopmentStatusID":"8","companyTruncated":"Peak Bio \/ White Lion Capital"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"DFV890","moa":"NLRP3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Inflazome","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Inzomelid","moa":"NLRP3","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Inflazome","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inflazome \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Inflazome \/ Undisclosed"},{"orgOrder":0,"company":"Inflazome","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Inzomelid","moa":"NLRP3","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Inflazome","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inflazome \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Inflazome \/ Undisclosed"},{"orgOrder":0,"company":"Inflazome","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"IZD334","moa":"NLRP3","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Inflazome","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Inflazome \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Inflazome \/ Undisclosed"},{"orgOrder":0,"company":"Zydus Lifesciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Usnoflast","moa":"NLRP3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zydus Lifesciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zydus Lifesciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Zydus Lifesciences \/ Undisclosed"},{"orgOrder":0,"company":"Zydus Lifesciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Usnoflast","moa":"NLRP3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zydus Lifesciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zydus Lifesciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Zydus Lifesciences \/ Undisclosed"},{"orgOrder":0,"company":"Zydus Lifesciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Usnoflast","moa":"NLRP3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zydus Lifesciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zydus Lifesciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Zydus Lifesciences \/ Undisclosed"},{"orgOrder":0,"company":"Zomagen Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"VTX2735","moa":"NLRP3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zomagen Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zomagen Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Zomagen Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Jupiter Neurosciences","sponsor":"Spartan Capital Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Plant Extract\/Herbal","year":"2022","type":"Public Offering","leadProduct":"Resveratrol","moa":"NLRP3\/SIRT1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Jupiter Neurosciences","amount2":0.02,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Undisclosed","sponsorNew":"Jupiter Neurosciences \/ Spartan Capital Securities","highestDevelopmentStatusID":"8","companyTruncated":"Jupiter Neurosciences \/ Spartan Capital Securities"},{"orgOrder":0,"company":"Sage Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"SAGE-718","moa":"N-methyl-D-aspartate receptor (NMDAR)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sage Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sage Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sage Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"SAGE-718","moa":"N-methyl-D-aspartate receptor (NMDAR)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sage Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sage Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sage Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"SAGE-718","moa":"N-methyl-D-aspartate receptor (NMDAR)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sage Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sage Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sage Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"SAGE-718","moa":"N-methyl-D-aspartate receptor (NMDAR)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sage Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sage Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sage Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"SAGE-718","moa":"N-methyl-D-aspartate receptor (NMDAR)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sage Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sage Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sage Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"SAGE-718","moa":"N-methyl-D-aspartate receptor (NMDAR)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sage Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sage Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sage Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"SAGE-718","moa":"N-methyl-D-aspartate receptor (NMDAR)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Sage Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sage Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Exsar Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Ambroxol","moa":"NO dependent soluble guanylate cyclase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Exsar Corporation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Exsar Corporation \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Exsar Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Wake Forest University Health Sciences","sponsor":"Gilead Sciences | National Institute of Diabetes and Digestive and Kidney Diseases | National Institutes of Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Ledipasvir","moa":"Nonstructural protein 5A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Wake Forest University Health Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Wake Forest University Health Sciences \/ Gilead Sciences | National Institute of Diabetes and Digestive and Kidney Diseases | National Institutes of Health","highestDevelopmentStatusID":"8","companyTruncated":"Wake Forest University Health Sciences \/ Gilead Sciences | National Institute of Diabetes and Digestive and Kidney Diseases | National Institutes of Health"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Bupropion Hydrochloride","moa":"Norepinephrine transporter | Dopamine transporter","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Visen Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Visen Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Visen Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Visen Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Visen Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Visen Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Visen Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Visen Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ascendis Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ascendis Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ascendis Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ascendis Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ascendis Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ascendis Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ascendis Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ascendis Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Ascendis Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Ascendis Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Ascendis Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ascendis Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Bocidelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Astellas Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Astellas Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Bocidelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Astellas Pharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Astellas Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Jefferies","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Jefferies"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Novo Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Series B Financing","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Novo Ventures","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Novo Ventures"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"OnKure Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"OnKure Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"OnKure Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"OnKure Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Reneo Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Reneo Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Reneo Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Reneo Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Laboratoires CTRS","sponsor":"Ethypharm","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2020","type":"Agreement","leadProduct":"Cholic Acid","moa":"NR1H4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Laboratoires CTRS","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Laboratoires CTRS \/ Ethypharm","highestDevelopmentStatusID":"15","companyTruncated":"Laboratoires CTRS \/ Ethypharm"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"NR2E3-AAV","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Abeona Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"ABO-102","moa":"N-sulphoglucosamine sulphohydrolase (SGSH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Abeona Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Abeona Therapeutics"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2018","type":"Inapplicable","leadProduct":"SOBI003","moa":"N-sulphoglucosamine sulphohydrolase (SGSH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"SOBI003","moa":"N-sulphoglucosamine sulphohydrolase (SGSH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"AbbVie Inc | Friedreich's Ataxia Research Alliance","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biogen \/ AbbVie Inc | Friedreich's Ataxia Research Alliance","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ AbbVie Inc | Friedreich's Ataxia Research Alliance"},{"orgOrder":0,"company":"Biogen","sponsor":"AbbVie Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biogen \/ AbbVie Inc","highestDevelopmentStatusID":"8","companyTruncated":"Biogen \/ AbbVie Inc"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Acquisition","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":7.2999999999999998,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":7.2999999999999998,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Biogen","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Biogen"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Acquisition","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":7.2999999999999998,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":7.2999999999999998,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Biogen","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Biogen"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Omaveloxolone","moa":"Nuclear factor erythroid 2-related factor 2","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Edasalonexent","moa":"Nuclear factor NF-kappa-B complex","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Catabasis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catabasis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Edasalonexent","moa":"Nuclear factor NF-kappa-B complex","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Catabasis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catabasis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Edasalonexent","moa":"Nuclear factor NF-kappa-B complex","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Catabasis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catabasis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Edasalonexent","moa":"Nuclear factor NF-kappa-B complex","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Catabasis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catabasis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Duchenne Uk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Partnership","leadProduct":"Edasalonexent","moa":"Nuclear factor NF-kappa-B complex","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Catabasis Pharmaceuticals \/ Duchenne Uk","highestDevelopmentStatusID":"10","companyTruncated":"Catabasis Pharmaceuticals \/ Duchenne Uk"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Edasalonexent","moa":"Nuclear factor NF-kappa-B complex","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Catabasis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catabasis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Edasalonexent","moa":"Nuclear factor NF-kappa-B complex","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Catabasis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catabasis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Edasalonexent","moa":"Nuclear factor NF-kappa-B complex","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Catabasis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catabasis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Edasalonexent","moa":"Nuclear factor NF-kappa-B complex","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Catabasis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catabasis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Edasalonexent","moa":"Nuclear factor NF-kappa-B complex","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Catabasis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catabasis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Oppenheimer & Co. Inc.","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Public Offering","leadProduct":"Edasalonexent","moa":"Nuclear factor NF-kappa-B complex","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catabasis Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Capsule","sponsorNew":"Catabasis Pharmaceuticals \/ Oppenheimer & Co. Inc.","highestDevelopmentStatusID":"10","companyTruncated":"Catabasis Pharmaceuticals \/ Oppenheimer & Co. Inc."},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"PYC-001","moa":"OPA1\/Tubulin\/VEGF","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"PYC-001","moa":"OPA1\/Tubulin\/VEGF","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"STK-002","moa":"Optic atrophy-1 protein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Stoke Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stoke Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Stoke Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"STK-002","moa":"Optic atrophy-1 protein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Stoke Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stoke Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Stoke Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"STK-002","moa":"Optic atrophy-1 protein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Stoke Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stoke Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Stoke Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Cancer Prevention Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2010","type":"Inapplicable","leadProduct":"Eflornithine Hydrochloride","moa":"Ornithine decarboxylase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Cancer Prevention Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cancer Prevention Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Cancer Prevention Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Cancer Prevention Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2011","type":"Inapplicable","leadProduct":"Eflornithine Hydrochloride","moa":"Ornithine decarboxylase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Cancer Prevention Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cancer Prevention Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Cancer Prevention Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Panbela Therapeutics","sponsor":"One-Two Therapeutics Assets Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2023","type":"Termination","leadProduct":"Eflornithine Hydrochloride","moa":"||Ornithine decarboxylase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Panbela Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Panbela Therapeutics \/ One-Two Therapeutics Assets Limited","highestDevelopmentStatusID":"15","companyTruncated":"Panbela Therapeutics \/ One-Two Therapeutics Assets Limited"},{"orgOrder":0,"company":"Cancer Prevention Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"Eflornithine Hydrochloride","moa":"||Ornithine decarboxylase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Cancer Prevention Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cancer Prevention Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Cancer Prevention Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Panbela Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2022","type":"Inapplicable","leadProduct":"Eflornithine Hydrochloride","moa":"||Ornithine decarboxylase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Panbela Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Panbela Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Panbela Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Cancer Prevention Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"Eflornithine Hydrochloride","moa":"||Ornithine decarboxylase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Cancer Prevention Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cancer Prevention Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Cancer Prevention Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Cancer Prevention Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"Eflornithine Hydrochloride","moa":"||Ornithine decarboxylase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Cancer Prevention Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cancer Prevention Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Cancer Prevention Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Cancer Prevention Pharmaceuticals","sponsor":"Panbela Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2022","type":"Acquisition","leadProduct":"Eflornithine Hydrochloride","moa":"||Ornithine decarboxylase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Cancer Prevention Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Tablet","sponsorNew":"Cancer Prevention Pharmaceuticals \/ Panbela Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Cancer Prevention Pharmaceuticals \/ Panbela Therapeutics"},{"orgOrder":0,"company":"Cancer Prevention Pharmaceuticals","sponsor":"Panbela Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2022","type":"Acquisition","leadProduct":"Eflornithine Hydrochloride","moa":"||Ornithine decarboxylase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Cancer Prevention Pharmaceuticals","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Tablet","sponsorNew":"Cancer Prevention Pharmaceuticals \/ Panbela Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Cancer Prevention Pharmaceuticals \/ Panbela Therapeutics"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ARCT-810","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ARCT-810","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARCT-810","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ARCT-810","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"ARCT-810","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"ARCT-810","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"ARCT-810","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"ARCT-810","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"ARCT-810","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Arcturus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arcturus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Avalotcagene Ontaparvovec","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Avalotcagene Ontaparvovec","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Avalotcagene Ontaparvovec","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"DTX301","moa":"Ornithine transcarbamylase (OTC)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2016","type":"Inapplicable","leadProduct":"scAAV8OTC","moa":"OTC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Acadia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Carbetocin Acetate","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Acadia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"Acadia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Acadia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Acadia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Carbetocin Acetate","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Acadia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"Acadia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Acadia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Levo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2018","type":"Inapplicable","leadProduct":"Carbetocin Acetate","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Levo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Levo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Levo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Acadia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Carbetocin Acetate","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Acadia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"Acadia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Acadia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Acadia Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Carbetocin Acetate","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Acadia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"Acadia Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Acadia Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Levo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Carbetocin Acetate","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Levo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Levo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Levo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ferring Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2013","type":"Inapplicable","leadProduct":"FE 992097","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ferring Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"Ferring Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ferring Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"French National Institute of Health and Medical Research","sponsor":"Tonix Pharmaceuticals Holding Corp","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Hormone","year":"2021","type":"Licensing Agreement","leadProduct":"Magnesium Potentiated Oxytocin","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"French National Institute of Health and Medical Research","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"French National Institute of Health and Medical Research \/ Tonix Pharmaceuticals Holding Corp","highestDevelopmentStatusID":"5","companyTruncated":"French National Institute of Health and Medical Research \/ Tonix Pharmaceuticals Holding Corp"},{"orgOrder":0,"company":"Tonix Pharmaceuticals Holding Corp","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2023","type":"Inapplicable","leadProduct":"Magnesium","moa":"||Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tonix Pharmaceuticals Holding Corp","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tonix Pharmaceuticals Holding Corp \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Tonix Pharmaceuticals Holding Corp \/ Undisclosed"},{"orgOrder":0,"company":"Tonix Pharmaceuticals Holding Corp","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2024","type":"Inapplicable","leadProduct":"Magnesium Potentiated Oxytocin","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tonix Pharmaceuticals Holding Corp","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tonix Pharmaceuticals Holding Corp \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Tonix Pharmaceuticals Holding Corp \/ Undisclosed"},{"orgOrder":0,"company":"Tonix Pharmaceuticals Holding Corp","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2023","type":"Inapplicable","leadProduct":"Magnesium","moa":"||Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tonix Pharmaceuticals Holding Corp","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tonix Pharmaceuticals Holding Corp \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Tonix Pharmaceuticals Holding Corp \/ Undisclosed"},{"orgOrder":0,"company":"Tonix Pharmaceuticals Holding Corp","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Magnesium Potentiated Oxytocin","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tonix Pharmaceuticals Holding Corp","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tonix Pharmaceuticals Holding Corp \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Tonix Pharmaceuticals Holding Corp \/ Undisclosed"},{"orgOrder":0,"company":"BiomX","sponsor":"Laidlaw & Company","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Microorganism","year":"2025","type":"Private Placement","leadProduct":"BX004","moa":"P. aeruginosa biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BiomX","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Inhalation","sponsorNew":"BiomX \/ Laidlaw & Company","highestDevelopmentStatusID":"7","companyTruncated":"BiomX \/ Laidlaw & Company"},{"orgOrder":0,"company":"BiomX","sponsor":"Deerfield Management Company","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Microorganism","year":"2024","type":"Private Placement","leadProduct":"BX004","moa":"P. aeruginosa biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BiomX","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Solution for Nebulizer","sponsorNew":"BiomX \/ Deerfield Management Company","highestDevelopmentStatusID":"7","companyTruncated":"BiomX \/ Deerfield Management Company"},{"orgOrder":0,"company":"BiomX","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Microorganism","year":"2021","type":"Inapplicable","leadProduct":"BX004","moa":"P. aeruginosa biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BiomX","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"BiomX \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BiomX \/ Undisclosed"},{"orgOrder":0,"company":"BiomX","sponsor":"Deerfield Management Company","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Microorganism","year":"2024","type":"Private Placement","leadProduct":"BX004","moa":"P. aeruginosa biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BiomX","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Solution for Nebulizer","sponsorNew":"BiomX \/ Deerfield Management Company","highestDevelopmentStatusID":"7","companyTruncated":"BiomX \/ Deerfield Management Company"},{"orgOrder":0,"company":"Adaptive Phage Therapeutics","sponsor":"BiomX","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2024","type":"Merger","leadProduct":"BX004","moa":"P. aeruginosa biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Adaptive Phage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Adaptive Phage Therapeutics \/ BiomX","highestDevelopmentStatusID":"7","companyTruncated":"Adaptive Phage Therapeutics \/ BiomX"},{"orgOrder":0,"company":"Adaptive Phage Therapeutics","sponsor":"BiomX","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2024","type":"Merger","leadProduct":"BX004","moa":"P. aeruginosa biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Adaptive Phage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Adaptive Phage Therapeutics \/ BiomX","highestDevelopmentStatusID":"7","companyTruncated":"Adaptive Phage Therapeutics \/ BiomX"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-103","moa":"PAH gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"HMI-103","moa":"PAH gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-103","moa":"PAH gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-103","moa":"PAH gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"HMI-103","moa":"PAH gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-103","moa":"PAH gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"HMI-103","moa":"PAH gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-103","moa":"PAH gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"ASP0367","moa":"PARP delta","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Astellas Pharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Astellas Pharma \/ Undisclosed"},{"orgOrder":0,"company":"OnKure Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"REN001","moa":"PARP delta","graph1":"Genetic Disease","graph2":"Phase I","graph3":"OnKure Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"OnKure Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"OnKure Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"OnKure Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"REN001","moa":"PARP delta","graph1":"Genetic Disease","graph2":"Phase I","graph3":"OnKure Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"OnKure Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"OnKure Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"OnKure Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"REN001","moa":"PARP delta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"OnKure Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"OnKure Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"OnKure Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"OnKure Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"REN001","moa":"PARP delta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"OnKure Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"OnKure Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"OnKure Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"EpiDestiny","sponsor":"National Institutes of Health | National Heart, Lung, and Blood Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Nicotinamide","moa":"Poly [ADP-ribose] polymerase 1 (PARP1)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"EpiDestiny","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"EpiDestiny \/ National Institutes of Health | National Heart, Lung, and Blood Institute","highestDevelopmentStatusID":"6","companyTruncated":"EpiDestiny \/ National Institutes of Health | National Heart, Lung, and Blood Institute"},{"orgOrder":0,"company":"Humanigen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"KB001-A","moa":"PcrV (Pseudomonas)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Humanigen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Humanigen \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Humanigen \/ Undisclosed"},{"orgOrder":0,"company":"iECURE","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"GTP-506A","moa":"||PCSK9","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"iECURE \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"iECURE \/ Undisclosed"},{"orgOrder":0,"company":"iECURE","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"GTP-506A","moa":"||PCSK9","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"iECURE \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"iECURE \/ Undisclosed"},{"orgOrder":0,"company":"Verve Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"VERVE-101","moa":"PCSK9","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Verve Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Verve Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Verve Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"The Medicines Company","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2016","type":"Inapplicable","leadProduct":"Cemdisiran","moa":"PCSK9 mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"The Medicines Company","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"The Medicines Company \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"The Medicines Company \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Inclisiran","moa":"PCSK9 mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Inclisiran","moa":"PCSK9 mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Inclisiran Sodium","moa":"PCSK9 mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"QBW276","moa":"PD-1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"CTx-PDE6b","moa":"PDE6B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Coave Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Laboratoires Thea","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"CTx-PDE6b","moa":"PDE6B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Injection","sponsorNew":"Coave Therapeutics \/ Laboratoires Thea","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Laboratoires Thea"},{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"CTx-PDE6b","moa":"PDE6B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Coave Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2011","type":"Inapplicable","leadProduct":"CR002","moa":"PDGFD","graph1":"Genetic Disease","graph2":"Phase I","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"ASC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"ASC61","moa":"PD-L1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ASC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ASC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ASC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Children's Hospital of Philadelphia","sponsor":"Friedreich's Ataxia Research Alliance | Stealth Biotherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Elamipretide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Children's Hospital of Philadelphia","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Children's Hospital of Philadelphia \/ Friedreich's Ataxia Research Alliance | Stealth Biotherapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Children's Hospital of Philadelphia \/ Friedreich's Ataxia Research Alliance | Stealth Biotherapeutics"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2015","type":"Inapplicable","leadProduct":"Elamipretide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Stealth Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Elamipretide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stealth Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Elamipretide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stealth Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Elamipretide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stealth Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Stealth Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Elamipretide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stealth Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Stealth Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Elamipretide","moa":"||peptides","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stealth Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Stealth Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Elamipretide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Stealth Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Stealth Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Millendo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Livoletide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Millendo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Millendo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Millendo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Millendo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Livoletide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Millendo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Millendo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Millendo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Protagonist Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"PTG-300","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Protagonist Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Protagonist Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Protagonist Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Protagonist Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"PTG-300","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Protagonist Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Protagonist Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Protagonist Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Protagonist Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Rusfertide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Protagonist Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Protagonist Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Protagonist Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Neuren Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Peptide, Unconjugated","year":"2013","type":"Inapplicable","leadProduct":"Trofinetide","moa":"IGF-1 receptor||peptides: neurologic indications","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Neuren Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Neuren Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Neuren Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Neupharma","sponsor":"Sintesi Research Srl | Evotec | Pari","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Antibiotic","year":"2019","type":"Inapplicable","leadProduct":"Teicoplanin","moa":"Peptidoglycan","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Neupharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Neupharma \/ Sintesi Research Srl | Evotec | Pari","highestDevelopmentStatusID":"6","companyTruncated":"Neupharma \/ Sintesi Research Srl | Evotec | Pari"},{"orgOrder":0,"company":"Savara","sponsor":"INC Research Limited","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2012","type":"Inapplicable","leadProduct":"Vancomycin HCl","moa":"Peptidoglycan","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Savara","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"Savara \/ INC Research Limited","highestDevelopmentStatusID":"8","companyTruncated":"Savara \/ INC Research Limited"},{"orgOrder":0,"company":"Savara","sponsor":"Syneos Health | Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2012","type":"Inapplicable","leadProduct":"Vancomycin HCl","moa":"Peptidoglycan","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Savara","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Savara \/ Syneos Health | Cystic Fibrosis Foundation","highestDevelopmentStatusID":"8","companyTruncated":"Savara \/ Syneos Health | Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Polaryx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Gemfibrozil","moa":"Peroxisome proliferator-activated receptor alpha","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Polaryx Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Polaryx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Polaryx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Micelle BioPharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"DHA","moa":"Peroxisome proliferator-activated receptor alpha; Prostaglandin G\/H synthase 1; Prostaglandin G\/H synthase 2; Peroxisome proliferator-activated receptor gamma; Retinoic acid receptor RXR-alpha; Retinoic acid receptor RXR-beta; Retinoic acid receptor RXR-gamma; Sterol regulatory element-binding protein 1; Myc proto-oncogene protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Micelle BioPharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Micelle BioPharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Micelle BioPharma \/ Undisclosed"},{"orgOrder":0,"company":"Minoryx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Leriglitazone","moa":"Peroxisome proliferator-activated receptor gamma","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Minoryx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Minoryx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Minoryx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Bardoxolone Methyl","moa":"Peroxisome proliferator-activated receptor gamma | Keap1\/Nrf2 | Inhibitor of nuclear factor kappa B kinase beta subunit","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Blackstone Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Financing","leadProduct":"Bardoxolone Methyl","moa":"Peroxisome proliferator-activated receptor gamma | Keap1\/Nrf2 | Inhibitor of nuclear factor kappa B kinase beta subunit","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Reata Pharmaceuticals","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Blackstone Life Sciences","highestDevelopmentStatusID":"10","companyTruncated":"Reata Pharmaceuticals \/ Blackstone Life Sciences"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Bardoxolone Methyl","moa":"Peroxisome proliferator-activated receptor gamma | Keap1\/Nrf2 | Inhibitor of nuclear factor kappa B kinase beta subunit","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Bardoxolone Methyl","moa":"Peroxisome proliferator-activated receptor gamma | Keap1\/Nrf2 | Inhibitor of nuclear factor kappa B kinase beta subunit","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Bardoxolone Methyl","moa":"Peroxisome proliferator-activated receptor gamma | Keap1\/Nrf2 | Inhibitor of nuclear factor kappa B kinase beta subunit","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Bardoxolone Methyl","moa":"Peroxisome proliferator-activated receptor gamma | Keap1\/Nrf2 | Inhibitor of nuclear factor kappa B kinase beta subunit","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Reata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Bardoxolone Methyl","moa":"Peroxisome proliferator-activated receptor gamma | Keap1\/Nrf2 | Inhibitor of nuclear factor kappa B kinase beta subunit","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Thomas Jefferson University","sponsor":"Drexel University | GSK","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Omega-3-Acid Ethyl Esters 90","moa":"Peroxisome proliferator-activated receptor gamma; Peroxisome proliferator-activated receptor alpha; Sterol regulatory element-binding protein 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Thomas Jefferson University","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Thomas Jefferson University \/ Drexel University | GSK","highestDevelopmentStatusID":"8","companyTruncated":"Thomas Jefferson University \/ Drexel University | GSK"},{"orgOrder":0,"company":"Synlogic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Labafenogene Marselecobac","moa":"Phe level","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Synlogic \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Synlogic \/ Undisclosed"},{"orgOrder":0,"company":"Synlogic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Labafenogene Marselecobac","moa":"Phe level","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Synlogic \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Synlogic \/ Undisclosed"},{"orgOrder":0,"company":"Synlogic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Labafenogene Marselecobac","moa":"Phe level","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Synlogic \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Synlogic \/ Undisclosed"},{"orgOrder":0,"company":"Synlogic","sponsor":"Science 37 Holdings","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Partnership","leadProduct":"Labafenogene Marselecobac","moa":"Phe level","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Synlogic \/ Science 37 Holdings","highestDevelopmentStatusID":"10","companyTruncated":"Synlogic \/ Science 37 Holdings"},{"orgOrder":0,"company":"Synlogic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Labafenogene Marselecobac","moa":"Phe level","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Synlogic \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Synlogic \/ Undisclosed"},{"orgOrder":0,"company":"Synlogic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Labafenogene Marselecobac","moa":"Phe level","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Synlogic \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Synlogic \/ Undisclosed"},{"orgOrder":0,"company":"Synlogic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"SYNB1934","moa":"Phe level","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Synlogic \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Synlogic \/ Undisclosed"},{"orgOrder":0,"company":"Nestle Health Sciences SA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"CDX6114","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nestle Health Sciences SA","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Nestle Health Sciences SA \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nestle Health Sciences SA \/ Undisclosed"},{"orgOrder":0,"company":"Nestle Health Sciences SA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"CDX-6114","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nestle Health Sciences SA","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Nestle Health Sciences SA \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nestle Health Sciences SA \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2013","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2012","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2018","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Taipei Veterans General Hospital","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"TAIWAN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"BH4","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Taipei Veterans General Hospital","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Taipei Veterans General Hospital \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"Taipei Veterans General Hospital \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"HLB Cell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"Hepastem","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"HLB Cell","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"HLB Cell \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"HLB Cell \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Financing","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Pfizer Inc","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Pfizer Inc"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"University of Texas Health Science Center at Houston","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Texas Health Science Center at Houston","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Texas Health Science Center at Houston \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"University of Texas Health Science Center at Houston \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"University of Utah","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"University of Utah","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Utah \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"University of Utah \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Merck Group","sponsor":"Merck Group | Smerud Medical Research International AS","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Merck Group","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Merck Group \/ Merck Group | Smerud Medical Research International AS","highestDevelopmentStatusID":"11","companyTruncated":"Merck Group \/ Merck Group | Smerud Medical Research International AS"},{"orgOrder":0,"company":"Merck Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Merck Group","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Merck Group \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Merck Group \/ Undisclosed"},{"orgOrder":0,"company":"University of Southern California","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"University of Southern California","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Southern California \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"University of Southern California \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"University Hospitals Cleveland Medical Center","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"University Hospitals Cleveland Medical Center","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University Hospitals Cleveland Medical Center \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"University Hospitals Cleveland Medical Center \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Phosphatidylserine","moa":"Phosphatidylserine decarboxylase proenzyme, mitochondrial; Scavenger receptor class B member 1; Protein kinase C alpha type; Phosphatidylserine synthase 1; Diacylglycerol kinase gamma; Diacylglycerol kinase delta; Phosphatidylserine synthase 2; Sphingomyelin phosphodiesterase 4; Sphingomyelin phosphodiesterase 3; Phospholipid-transporting ATPase IA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NYU Langone Health \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NYU Langone Health \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"PF-02545920","moa":"Phosphodiesterase 10A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"PF-02545920","moa":"Phosphodiesterase 10A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"PF-02545920","moa":"Phosphodiesterase 10A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Verona Pharma","sponsor":"Cystic Fibrosis Trust","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Ensifentrine","moa":"Phosphodiesterase 3 | Phosphodiesterase 4","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Verona Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Verona Pharma \/ Cystic Fibrosis Trust","highestDevelopmentStatusID":"8","companyTruncated":"Verona Pharma \/ Cystic Fibrosis Trust"},{"orgOrder":0,"company":"Shionogi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Zatolmilast","moa":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Shionogi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Shionogi \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Shionogi \/ Undisclosed"},{"orgOrder":0,"company":"Tetra Discovery Partners","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Zatolmilast","moa":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Tetra Discovery Partners","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Tetra Discovery Partners \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Tetra Discovery Partners \/ Undisclosed"},{"orgOrder":0,"company":"Tetra Discovery Partners","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Zatolmilast","moa":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Tetra Discovery Partners","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Tetra Discovery Partners \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Tetra Discovery Partners \/ Undisclosed"},{"orgOrder":0,"company":"Shionogi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Zatolmilast","moa":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Shionogi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Shionogi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Shionogi \/ Undisclosed"},{"orgOrder":0,"company":"Shionogi","sponsor":"Jordan\u2019s Guardian Angels","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2025","type":"Collaboration","leadProduct":"Zatolmilast","moa":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Shionogi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Shionogi \/ Jordan\u2019s Guardian Angels","highestDevelopmentStatusID":"8","companyTruncated":"Shionogi \/ Jordan\u2019s Guardian Angels"},{"orgOrder":0,"company":"Tetra Discovery Partners","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Zatolmilast","moa":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Tetra Discovery Partners","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tetra Discovery Partners \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Tetra Discovery Partners \/ Undisclosed"},{"orgOrder":0,"company":"Tetra Discovery Partners","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Zatolmilast","moa":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Tetra Discovery Partners","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tetra Discovery Partners \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Tetra Discovery Partners \/ Undisclosed"},{"orgOrder":0,"company":"Tetra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Zatolmilast","moa":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Tetra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Tetra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Tetra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Tetra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Zatolmilast","moa":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Tetra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Tetra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Tetra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Tetra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Zatolmilast","moa":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Tetra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Tetra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Tetra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"National Jewish Health","sponsor":"Augusta University | Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Sildenafil Citrate","moa":"Phosphodiesterase 5A","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"National Jewish Health","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"National Jewish Health \/ Augusta University | Cystic Fibrosis Foundation","highestDevelopmentStatusID":"9","companyTruncated":"National Jewish Health \/ Augusta University | Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"National Jewish Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Sildenafil Citrate","moa":"Phosphodiesterase 5A","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"National Jewish Health","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"National Jewish Health \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"National Jewish Health \/ Undisclosed"},{"orgOrder":0,"company":"National Jewish Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Sildenafil Citrate","moa":"Phosphodiesterase 5A","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"National Jewish Health","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"National Jewish Health \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"National Jewish Health \/ Undisclosed"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Tadalafil","moa":"Phosphodiesterase 5A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eli Lilly \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Eli Lilly \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"PF-04447943","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Cardurion Pharmaceutical","sponsor":"Imara","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Tovinontrine","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cardurion Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cardurion Pharmaceutical \/ Imara","highestDevelopmentStatusID":"8","companyTruncated":"Cardurion Pharmaceutical \/ Imara"},{"orgOrder":0,"company":"Imara","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Tovinontrine","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Imara","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Imara \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Imara \/ Undisclosed"},{"orgOrder":0,"company":"Cardurion Pharmaceutical","sponsor":"Imara","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tovinontrine","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cardurion Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cardurion Pharmaceutical \/ Imara","highestDevelopmentStatusID":"8","companyTruncated":"Cardurion Pharmaceutical \/ Imara"},{"orgOrder":0,"company":"Cardurion Pharmaceutical","sponsor":"Quintiles Inc | Imara","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Tovinontrine","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cardurion Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cardurion Pharmaceutical \/ Quintiles Inc | Imara","highestDevelopmentStatusID":"8","companyTruncated":"Cardurion Pharmaceutical \/ Quintiles Inc | Imara"},{"orgOrder":0,"company":"Imara","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Tovinontrine","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Imara","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Imara \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Imara \/ Undisclosed"},{"orgOrder":0,"company":"Imara","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tovinontrine","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Imara","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Imara \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Imara \/ Undisclosed"},{"orgOrder":0,"company":"Imara","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Tovinontrine","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Imara","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Imara \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Imara \/ Undisclosed"},{"orgOrder":0,"company":"Imara","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Public Offering","leadProduct":"Tovinontrine","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Imara","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Imara \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Imara \/ Undisclosed"},{"orgOrder":0,"company":"Imara","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tovinontrine","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Imara","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Imara \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Imara \/ Undisclosed"},{"orgOrder":0,"company":"Imara","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tovinontrine","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Imara","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Imara \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Imara \/ Undisclosed"},{"orgOrder":0,"company":"Imara","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tovinontrine","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Imara","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Imara \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Imara \/ Undisclosed"},{"orgOrder":0,"company":"Imara","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Public Offering","leadProduct":"Tovinontrine","moa":"Phosphodiesterase 9A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Imara","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Imara \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Imara \/ Undisclosed"},{"orgOrder":0,"company":"Anthera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Varespladib","moa":"Phospholipase A2 group IIA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Anthera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Anthera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Anthera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"phosphoro-derivatives","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Agreement","leadProduct":"Fosdenopterin","moa":"phosphoro-derivatives","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"phosphoro-derivatives","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"phosphoro-derivatives","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"phosphoro-derivatives","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"phosphoro-derivatives","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Relay Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"RLY-2608","moa":"PI3K-alpha","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Relay Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Relay Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Relay Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Alpelisib","moa":"PI3-kinase p110-alpha subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Alpelisib","moa":"PI3-kinase p110-alpha subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Alpelisib","moa":"PI3-kinase p110-alpha subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Alpelisib","moa":"PI3-kinase p110-alpha subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Pharming","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Leniolisib Phosphate","moa":"PI3-kinase p110-delta subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Pharming \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pharming \/ Undisclosed"},{"orgOrder":0,"company":"Pharming","sponsor":"Aixial Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Leniolisib Phosphate","moa":"PI3-kinase p110-delta subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Pharming \/ Aixial Group","highestDevelopmentStatusID":"8","companyTruncated":"Pharming \/ Aixial Group"},{"orgOrder":0,"company":"Pharming","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Leniolisib Phosphate","moa":"PI3-kinase p110-delta subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pharming \/ Undisclosed"},{"orgOrder":0,"company":"Pharming","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Leniolisib Phosphate","moa":"PI3-kinase p110-delta subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pharming \/ Undisclosed"},{"orgOrder":0,"company":"HaiHe Biopharma Co., Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"CYH33","moa":"PI3K\u03b1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"HaiHe Biopharma Co., Ltd","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"HaiHe Biopharma Co., Ltd \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"HaiHe Biopharma Co., Ltd \/ Undisclosed"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ADX-324","moa":"PKK","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ADARx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ADARx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"ADARx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"Avance Clinical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ADX-324","moa":"PKK","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ADARx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ADARx Pharmaceuticals \/ Avance Clinical","highestDevelopmentStatusID":"10","companyTruncated":"ADARx Pharmaceuticals \/ Avance Clinical"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"Bain Capital Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Series C Financing","leadProduct":"ADX-324","moa":"PKK","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ADARx Pharmaceuticals","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20000000000000001,"dosageForm":"Injection","sponsorNew":"ADARx Pharmaceuticals \/ Bain Capital Life Sciences","highestDevelopmentStatusID":"10","companyTruncated":"ADARx Pharmaceuticals \/ Bain Capital Life Sciences"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"Ascenta Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Series B Financing","leadProduct":"ADX-324","moa":"PKK","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ADARx Pharmaceuticals","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Injection","sponsorNew":"ADARx Pharmaceuticals \/ Ascenta Capital","highestDevelopmentStatusID":"10","companyTruncated":"ADARx Pharmaceuticals \/ Ascenta Capital"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ADX-324","moa":"PKK","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ADARx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ADARx Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"ADARx Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"RP-L301","moa":"PKLR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"RP-L301","moa":"PKLR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tebapivat","moa":"PKLR||Pyruvate kinase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Tebapivat","moa":"PKLR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Pint Pharma","sponsor":"BioCryst Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"AUSTRIA","productType":"Miscellaneous","year":"2022","type":"Partnership","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pint Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pint Pharma \/ BioCryst Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Pint Pharma \/ BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Pint Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRIA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pint Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pint Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pint Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Pint Pharma","sponsor":"BioCryst Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRIA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pint Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pint Pharma \/ BioCryst Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Pint Pharma \/ BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Pint Pharma","sponsor":"BioCryst Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRIA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pint Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pint Pharma \/ BioCryst Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Pint Pharma \/ BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Pint Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRIA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pint Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pint Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pint Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"enzyme inhibitors||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"enzyme inhibitors||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"enzyme inhibitors||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"enzyme inhibitors||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"enzyme inhibitors||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"enzyme inhibitors||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"enzyme inhibitors||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"enzyme inhibitors||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"enzyme inhibitors||Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Erkim","sponsor":"BioCryst Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"TURKEY","productType":"Miscellaneous","year":"2023","type":"Collaboration","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Erkim","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Erkim \/ BioCryst Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Erkim \/ BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Torii Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Agreement","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0.040000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Torii Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Torii Pharmaceutical"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Pint Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Pint Pharma","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Pint Pharma"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Swixx BioPharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Collaboration","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Swixx BioPharma","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Swixx BioPharma"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Torii Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Collaboration","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Torii Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Torii Pharmaceutical"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Neopharmed Gentili","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Divestment","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0.26000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.26000000000000001,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Neopharmed Gentili","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Neopharmed Gentili"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Funding","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0.33000000000000002,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.33000000000000002,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Royalty Pharma","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Royalty Pharma"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Avoralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Berotralstat Hydrochloride","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BioCryst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2015","type":"Inapplicable","leadProduct":"DX-2930","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SHIRE PLC \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"DX-2930","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2014","type":"Inapplicable","leadProduct":"DX-2930","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2013","type":"Inapplicable","leadProduct":"DX-2930","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Ecallantide","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Protein","year":"2013","type":"Inapplicable","leadProduct":"Ecallantide","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2013","type":"Inapplicable","leadProduct":"Ecallantide","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NYU Langone Health \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"NYU Langone Health \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Charite University, Berlin, Germany","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Charite University, Berlin, Germany","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Charite University, Berlin, Germany \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Charite University, Berlin, Germany \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SHIRE PLC \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"Ypsomed","sponsor":"Astria Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2024","type":"Partnership","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ypsomed","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ypsomed \/ Astria Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Ypsomed \/ Astria Therapeutics"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Public Offering","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Jefferies"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Public Offering","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0.13,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ RA Capital Management","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ RA Capital Management"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Navenibart","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Kaken Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Licensing Agreement","leadProduct":"Navenibart","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Kaken Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Kaken Pharmaceuticals"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Perceptive Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Private Placement","leadProduct":"QLS-215","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Catabasis Pharmaceuticals","amount2":0.11,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"Catabasis Pharmaceuticals \/ Perceptive Advisors","highestDevelopmentStatusID":"7","companyTruncated":"Catabasis Pharmaceuticals \/ Perceptive Advisors"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"QLS-215","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Catabasis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Catabasis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Quellis Biosciences Inc","sponsor":"Catabasis Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Acquisition","leadProduct":"QLS-215","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Quellis Biosciences Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Quellis Biosciences Inc \/ Catabasis Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Quellis Biosciences Inc \/ Catabasis Pharmaceuticals"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"STAR-0215","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"STAR-0215","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"STAR-0215","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"TAK-743","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"KVD824","moa":"Plasma kallikrein (KLKB1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"KVD824","moa":"Plasma kallikrein (KLKB1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"KVD824","moa":"Plasma kallikrein (KLKB1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"KVD824","moa":"Plasma kallikrein (KLKB1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"KVD824","moa":"Plasma kallikrein (KLKB1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"KVD824","moa":"Plasma kallikrein (KLKB1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"KVD824","moa":"Plasma kallikrein (KLKB1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"KVD824","moa":"Plasma kallikrein (KLKB1)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor","moa":"C1 esterase||Plasma protease C1 inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Parexel","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor","moa":"Plasma protease C1 inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Parexel","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Parexel"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor","moa":"Plasma protease C1 inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor","moa":"Plasma protease C1 inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor","moa":"Plasma protease C1 inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Halozyme Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor","moa":"Plasma protease C1 inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Halozyme Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Halozyme Therapeutics"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor","moa":"Plasma protease C1 inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor","moa":"Plasma protease C1 inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Kedrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"Plasminogen (Human)","moa":"Plasminogen","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Kedrion","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kedrion \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Kedrion \/ Undisclosed"},{"orgOrder":0,"company":"Kedrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Plasminogen (Human)","moa":"Plasminogen","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Kedrion","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kedrion \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Kedrion \/ Undisclosed"},{"orgOrder":0,"company":"Kedrion","sponsor":"Cedars-Sinai | Vanderbilt University Medical Center | Indiana Hemophilia &Thrombosis Center, Inc.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Plasminogen (Human)","moa":"Plasminogen","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Kedrion","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kedrion \/ Cedars-Sinai | Vanderbilt University Medical Center | Indiana Hemophilia &Thrombosis Center, Inc.","highestDevelopmentStatusID":"9","companyTruncated":"Kedrion \/ Cedars-Sinai | Vanderbilt University Medical Center | Indiana Hemophilia &Thrombosis Center, Inc."},{"orgOrder":0,"company":"Kedrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2018","type":"Inapplicable","leadProduct":"Plasminogen (Human)","moa":"Plasminogen","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Kedrion","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kedrion \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Kedrion \/ Undisclosed"},{"orgOrder":0,"company":"Nexus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Tranexamic Acid","moa":"Plasminogen","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Nexus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Nexus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Avenacy","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Tranexamic Acid","moa":"Plasminogen","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Avenacy","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Avenacy \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Avenacy \/ Undisclosed"},{"orgOrder":0,"company":"DTx Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"DTx-1252","moa":"PMP22","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"DTx Pharma","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"DTx Pharma \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"DTx Pharma \/ Undisclosed"},{"orgOrder":0,"company":"DTx Pharma","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Acquisition","leadProduct":"DTx-1252","moa":"PMP22","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"DTx Pharma","amount2":1,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":1,"dosageForm":"Injection","sponsorNew":"DTx Pharma \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"5","companyTruncated":"DTx Pharma \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Tianjin Tasly Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"PXT3003","moa":"PMP22 overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Tianjin Tasly Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Tianjin Tasly Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Tianjin Tasly Group \/ Undisclosed"},{"orgOrder":0,"company":"Pharnext","sponsor":"Syneos Health | Premier Research Group | Greenphire | Theradis | Amarex | Eurofins CDMO","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"PXT3003","moa":"PMP22 overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Pharnext \/ Syneos Health | Premier Research Group | Greenphire | Theradis | Amarex | Eurofins CDMO","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Syneos Health | Premier Research Group | Greenphire | Theradis | Amarex | Eurofins CDMO"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Parexel","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"AZD2693","moa":"PNPLA3","graph1":"Genetic Disease","graph2":"Phase I","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AstraZeneca \/ Parexel","highestDevelopmentStatusID":"6","companyTruncated":"AstraZeneca \/ Parexel"},{"orgOrder":0,"company":"Pretzel Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"PX578","moa":"POLG","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pretzel Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pretzel Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Pretzel Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Savara","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"MST-188","moa":"polymers","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Savara","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Savara \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Savara \/ Undisclosed"},{"orgOrder":0,"company":"Savara","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"MST-188","moa":"polymers","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Savara","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Savara \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Savara \/ Undisclosed"},{"orgOrder":0,"company":"University of Illinois, Urbana-Champaign","sponsor":"BIO-CAT","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Protease","moa":"Polypeptides","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Illinois, Urbana-Champaign","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Illinois, Urbana-Champaign \/ BIO-CAT","highestDevelopmentStatusID":"1","companyTruncated":"University of Illinois, Urbana-Champaign \/ BIO-CAT"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Folia Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Folia Health","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Folia Health"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Guggenheim Securities","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Guggenheim Securities"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Oppenheimer & Co. Inc.","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Public Offering","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Oppenheimer & Co. Inc.","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Oppenheimer & Co. Inc."},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Piper Sandler","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Public Offering","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0.14000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14000000000000001,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Piper Sandler","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Piper Sandler"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Adage Capital Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0.12,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Adage Capital Partners","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Adage Capital Partners"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Public Offering","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Guggenheim Securities","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Guggenheim Securities"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Guggenheim Securities","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Guggenheim Securities"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Nantahala Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Agreement","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Nantahala Capital Management","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Nantahala Capital Management"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Oppenheimer & Co. Inc.","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Public Offering","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Oppenheimer & Co. Inc.","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Oppenheimer & Co. Inc."},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Oxford Finance","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Financing","leadProduct":"Diazoxide","moa":"Potassium channel, inwardly rectifying, subfamily J, member 8 | Potassium channel, inwardly rectifying, subfamily J, member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Oxford Finance","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Oxford Finance"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Diazoxide","moa":"ABCC8(SUR1)\/KCNJ11(KIR6.2)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Diazoxide","moa":"ABCC8(SUR1)\/KCNJ11(KIR6.2)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Diazoxide","moa":"ABCC8(SUR1)\/KCNJ11(KIR6.2)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Diazoxide","moa":"ABCC8(SUR1)\/KCNJ11(KIR6.2)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Soleno Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Soleno Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Essentialis","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Diazoxide","moa":"ABCC8(SUR1)\/KCNJ11(KIR6.2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Essentialis","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Essentialis \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Essentialis \/ Undisclosed"},{"orgOrder":0,"company":"Essentialis","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Diazoxide","moa":"ABCC8(SUR1)\/KCNJ11(KIR6.2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Essentialis","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Essentialis \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Essentialis \/ Undisclosed"},{"orgOrder":0,"company":"Polaryx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"PLX-300","moa":"PPA-alpha receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Polaryx Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Polaryx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Polaryx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Seladelpar Lysine","moa":"Peroxisome proliferator-activated receptor delta||PPA-delta receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Gilead Sciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Gilead Sciences \/ Undisclosed"},{"orgOrder":0,"company":"SLA Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Icosapent Ethyl","moa":"Prostaglandin G\/H synthase 2; Prostaglandin G\/H synthase 1; Peroxisome proliferator-activated receptor gamma; Peroxisome proliferator-activated receptor delta; Free fatty acid receptor 1; Sodium\/calcium exchanger 1; Fatty acid-binding protein, brain; Acyl-CoA (8-3)-desaturase; Long-chain-fatty-acid--CoA ligase 4; Transient receptor potential cation channel subfamily V member 1; Peroxisome proliferator-activated receptor alpha; Polyunsaturated fatty acid 5-lipoxygenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SLA Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"SLA Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SLA Pharma \/ Undisclosed"},{"orgOrder":0,"company":"1cBio","sponsor":"Alesta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Licensing Agreement","leadProduct":"ALE1","moa":"PPi","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"1cBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"1cBio \/ Alesta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"1cBio \/ Alesta Therapeutics"},{"orgOrder":0,"company":"University of Chicago","sponsor":"Inflectis Bioscience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Licensing Agreement","leadProduct":"Sephin1","moa":"PPP1R15A\/PP1c phosphatase complex","graph1":"Genetic Disease","graph2":"Phase I","graph3":"University of Chicago","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"University of Chicago \/ Inflectis Bioscience","highestDevelopmentStatusID":"6","companyTruncated":"University of Chicago \/ Inflectis Bioscience"},{"orgOrder":0,"company":"Endogena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"EA-2353","moa":"Progenitor cell","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Endogena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Endogena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Endogena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Endogena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"EA-2353","moa":"Progenitor cell","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Endogena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Endogena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Endogena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Endogena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"EA-2353","moa":"Progenitor cell","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Endogena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Endogena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Endogena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Endogena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"EA-2353","moa":"Progenitor cell","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Endogena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Endogena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Endogena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Fate Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"ProHema-CB","moa":"Progenitor cell","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Fate Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Fate Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Fate Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Meta Healthcare Ltd","sponsor":"Birmingham Women's and Children's NHS Foundation Trust","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Amino Acid","year":"2024","type":"Inapplicable","leadProduct":"L-VALINE","moa":"Propionyl-CoA carboxylase beta chain, mitochondrial; Branched-chain-amino-acid aminotransferase, cytosolic; Valine--tRNA ligase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Meta Healthcare Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Meta Healthcare Ltd \/ Birmingham Women's and Children's NHS Foundation Trust","highestDevelopmentStatusID":"1","companyTruncated":"Meta Healthcare Ltd \/ Birmingham Women's and Children's NHS Foundation Trust"},{"orgOrder":0,"company":"Tempest Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"TPST-1495","moa":"Prostaglandin E2 receptor EP2 (PTGER2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tempest Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tempest Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Tempest Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Tempest Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"TPST-1495","moa":"Prostaglandin E2 receptor EP2 (PTGER2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tempest Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tempest Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Tempest Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"R-Tech Ueno","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Plant Extract\/Herbal","year":"2013","type":"Inapplicable","leadProduct":"Isopropyl Unoprostone","moa":"prostaglandins","graph1":"Genetic Disease","graph2":"Phase III","graph3":"R-Tech Ueno","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"R-Tech Ueno \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"R-Tech Ueno \/ Undisclosed"},{"orgOrder":0,"company":"United Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Treprostinil Sodium","moa":"Prostanoid IP receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"United Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"United Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"United Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ApcinteX","sponsor":"Centessa Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"SerpinPC","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ApcinteX","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ApcinteX \/ Centessa Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"ApcinteX \/ Centessa Pharmaceuticals"},{"orgOrder":0,"company":"ApcinteX","sponsor":"Centessa Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"SerpinPC","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ApcinteX","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ApcinteX \/ Centessa Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"ApcinteX \/ Centessa Pharmaceuticals"},{"orgOrder":0,"company":"ApcinteX","sponsor":"Centessa Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"SerpinPC","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ApcinteX","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ApcinteX \/ Centessa Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"ApcinteX \/ Centessa Pharmaceuticals"},{"orgOrder":0,"company":"ApcinteX","sponsor":"Centessa Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"SerpinPC","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ApcinteX","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ApcinteX \/ Centessa Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"ApcinteX \/ Centessa Pharmaceuticals"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Public Offering","leadProduct":"Serpinpc","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Centessa Pharmaceuticals","amount2":0.23000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Infusion","sponsorNew":"Centessa Pharmaceuticals \/ Goldman Sachs & Co","highestDevelopmentStatusID":"10","companyTruncated":"Centessa Pharmaceuticals \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Serpinpc","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Centessa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Centessa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Centessa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Serpinpc","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Centessa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Centessa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Centessa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Oberland Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Agreement","leadProduct":"Serpinpc","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Centessa Pharmaceuticals","amount2":0.29999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.29999999999999999,"dosageForm":"Infusion","sponsorNew":"Centessa Pharmaceuticals \/ Oberland Capital Management","highestDevelopmentStatusID":"7","companyTruncated":"Centessa Pharmaceuticals \/ Oberland Capital Management"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Serpinpc","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Centessa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Centessa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Centessa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Public Offering","leadProduct":"Serpinpc","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Centessa Pharmaceuticals","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Infusion","sponsorNew":"Centessa Pharmaceuticals \/ Goldman Sachs & Co","highestDevelopmentStatusID":"10","companyTruncated":"Centessa Pharmaceuticals \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"ApcinteX","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Serpinpc","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Centessa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Centessa Pharmaceuticals \/ ApcinteX","highestDevelopmentStatusID":"10","companyTruncated":"Centessa Pharmaceuticals \/ ApcinteX"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"AnGes","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Partnership","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ AnGes","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ AnGes"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Acquisition","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"AnGes","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ AnGes","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ AnGes"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"The Progeria Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Divestment","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0.10000000000000001,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ The Progeria Research Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ The Progeria Research Foundation"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"The Progeria Research Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ The Progeria Research Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ The Progeria Research Foundation"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eiger BioPharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Eiger BioPharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Aytu BioPharma","sponsor":"Canaccord Genuity","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Public Offering","leadProduct":"Enzastaurin","moa":"Protein kinase C beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Aytu BioPharma","amount2":0.01,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Tablet","sponsorNew":"Aytu BioPharma \/ Canaccord Genuity","highestDevelopmentStatusID":"10","companyTruncated":"Aytu BioPharma \/ Canaccord Genuity"},{"orgOrder":0,"company":"Aytu BioPharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Enzastaurin","moa":"Protein kinase C beta","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Aytu BioPharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Aytu BioPharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Aytu BioPharma \/ Undisclosed"},{"orgOrder":0,"company":"Rumpus Therapeutics","sponsor":"Aytu BioScience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Licensing Agreement","leadProduct":"Enzastaurin","moa":"Protein kinase C beta","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rumpus Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rumpus Therapeutics \/ Aytu BioScience","highestDevelopmentStatusID":"4","companyTruncated":"Rumpus Therapeutics \/ Aytu BioScience"},{"orgOrder":0,"company":"Denovo Biopharma","sponsor":"Aytu BioScience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Licensing Agreement","leadProduct":"Enzastaurin","moa":"Protein kinase C beta","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Denovo Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Denovo Biopharma \/ Aytu BioScience","highestDevelopmentStatusID":"4","companyTruncated":"Denovo Biopharma \/ Aytu BioScience"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"VP-001","moa":"PRPF31","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"VP-001","moa":"PRPF31","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"VP-001","moa":"PRPF31","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"VP-001","moa":"PRPF31","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"VP-001","moa":"PRPF31","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"NICE","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"NICE","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"NICE \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"NICE \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Children's Hospital Los Angeles","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Children's Hospital Los Angeles","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Children's Hospital Los Angeles \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"7","companyTruncated":"Children's Hospital Los Angeles \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Inclacumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Inclacumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Inclacumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Inclacumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Inclacumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Global Blood Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Global Blood Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Reprixys Pharmaceutical","sponsor":"National Heart, Lung, and Blood Institute | Food and Drug Administration","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Antibody, Unconjugated","year":"2013","type":"Inapplicable","leadProduct":"SelG1","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Reprixys Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Reprixys Pharmaceutical \/ National Heart, Lung, and Blood Institute | Food and Drug Administration","highestDevelopmentStatusID":"8","companyTruncated":"Reprixys Pharmaceutical \/ National Heart, Lung, and Blood Institute | Food and Drug Administration"},{"orgOrder":0,"company":"Crescent Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Rivipansel","moa":"Leukocyte adhesion molecule-1 | P-selectin | Selectin E||P-selectin | Leukocyte adhesion molecule-1 | Selectin E","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crescent Biopharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Crescent Biopharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crescent Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Crescent Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Rivipansel","moa":"P-selectin | Leukocyte adhesion molecule-1 | Selectin E","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crescent Biopharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Crescent Biopharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crescent Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Crescent Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Rivipansel","moa":"P-selectin | Leukocyte adhesion molecule-1 | Selectin E","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crescent Biopharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Crescent Biopharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crescent Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Crescent Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Rivipansel","moa":"P-selectin | Leukocyte adhesion molecule-1 | Selectin E","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crescent Biopharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Crescent Biopharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crescent Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Crescent Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Rivipansel","moa":"P-selectin | Leukocyte adhesion molecule-1 | Selectin E","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crescent Biopharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Crescent Biopharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crescent Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Crescent Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Rivipansel","moa":"P-selectin | Leukocyte adhesion molecule-1 | Selectin E","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crescent Biopharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Crescent Biopharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crescent Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Biossil","sponsor":"Crescent Biopharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Rivipansel","moa":"P-selectin | Leukocyte adhesion molecule-1 | Selectin E","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biossil","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Biossil \/ GlycoMimetics","highestDevelopmentStatusID":"10","companyTruncated":"Biossil \/ GlycoMimetics"},{"orgOrder":0,"company":"ContraFect Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"CF-370","moa":"Pseudomonas aeruginosa cell wall","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"ContraFect Corporation","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ContraFect Corporation \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"ContraFect Corporation \/ Undisclosed"},{"orgOrder":0,"company":"ContraFect Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"CF-370","moa":"Pseudomonas aeruginosa cell wall","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"ContraFect Corporation","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ContraFect Corporation \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"ContraFect Corporation \/ Undisclosed"},{"orgOrder":0,"company":"DepYmed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"DPM-1001","moa":"PTP1B","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"DepYmed","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"DepYmed \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"DepYmed \/ Undisclosed"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Daiichi Sankyo","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Prasugrel","moa":"Purinergic receptor P2Y12","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Eli Lilly \/ Daiichi Sankyo","highestDevelopmentStatusID":"10","companyTruncated":"Eli Lilly \/ Daiichi Sankyo"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Daiichi Sankyo","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Prasugrel","moa":"Purinergic receptor P2Y12","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Eli Lilly \/ Daiichi Sankyo","highestDevelopmentStatusID":"10","companyTruncated":"Eli Lilly \/ Daiichi Sankyo"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Daiichi Sankyo","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Prasugrel","moa":"Purinergic receptor P2Y12","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Eli Lilly \/ Daiichi Sankyo","highestDevelopmentStatusID":"10","companyTruncated":"Eli Lilly \/ Daiichi Sankyo"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Daiichi Sankyo","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Prasugrel","moa":"Purinergic receptor P2Y12","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Eli Lilly \/ Daiichi Sankyo","highestDevelopmentStatusID":"10","companyTruncated":"Eli Lilly \/ Daiichi Sankyo"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Prasugrel","moa":"Purinergic receptor P2Y12","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Eli Lilly \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Eli Lilly \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Parexel","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Ticagrelor","moa":"Purinergic receptor P2Y12","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Parexel","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Parexel"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Ticagrelor","moa":"Purinergic receptor P2Y12","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"IQVIA","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Ticagrelor","moa":"Purinergic receptor P2Y12","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AstraZeneca \/ IQVIA","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ IQVIA"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"IQVIA","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ticagrelor","moa":"Purinergic receptor P2Y12","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AstraZeneca \/ IQVIA","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ IQVIA"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Ticagrelor","moa":"Purinergic receptor P2Y12","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Ticagrelor","moa":"Purinergic receptor P2Y12","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"Merck & Co","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Denufosol Tetrasodium","moa":"Purinergic receptor P2Y2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Merck & Co","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Merck & Co \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Merck & Co \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2015","type":"Inapplicable","leadProduct":"Asfotase Alfa","moa":"Pyrophosphate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Asfotase Alfa","moa":"Pyrophosphate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Asfotase Alfa","moa":"Pyrophosphate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Asfotase Alfa","moa":"Pyrophosphate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Asfotase Alfa","moa":"Pyrophosphate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2015","type":"Inapplicable","leadProduct":"Asfotase Alfa","moa":"Pyrophosphate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2019","type":"Inapplicable","leadProduct":"Asfotase Alfa","moa":"Pyrophosphate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2016","type":"Inapplicable","leadProduct":"Asfotase Alfa","moa":"Pyrophosphate","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Medpace, Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Forma Therapeutics \/ Medpace, Inc","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Medpace, Inc"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Novo Nordisk","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Novo Nordisk","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Novo Nordisk"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Gabrail Cancer Center Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Gabrail Cancer Center Research","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Gabrail Cancer Center Research"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Novo Nordisk","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Novo Nordisk","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Novo Nordisk"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Public Offering","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Jefferies"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Acquisition","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":1.1000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":1.1000000000000001,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Novo Nordisk","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Novo Nordisk"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Acquisition","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":1.1000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":1.1000000000000001,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Novo Nordisk","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Novo Nordisk"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Newco","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Divestment","leadProduct":"Etavopivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Forma Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Forma Therapeutics \/ Newco","highestDevelopmentStatusID":"10","companyTruncated":"Forma Therapeutics \/ Newco"},{"orgOrder":0,"company":"Centogene","sponsor":"Agios Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2021","type":"Partnership","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Centogene","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Centogene \/ Agios Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Centogene \/ Agios Pharmaceuticals"},{"orgOrder":0,"company":"Centogene","sponsor":"Agios Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2022","type":"Expanded Collaboration","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Centogene","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Centogene \/ Agios Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Centogene \/ Agios Pharmaceuticals"},{"orgOrder":0,"company":"National Institutes of Health Clinical Center","sponsor":"National Heart, Lung, and Blood Institute | Agios Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase I","graph3":"National Institutes of Health Clinical Center","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Institutes of Health Clinical Center \/ National Heart, Lung, and Blood Institute | Agios Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"National Institutes of Health Clinical Center \/ National Heart, Lung, and Blood Institute | Agios Pharmaceuticals"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase isozymes R\/L","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Agios Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"University of South Florida","sponsor":"EDISON PHARMACEUTICALS INC | Friedreich's Ataxia Research Alliance","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"Quinone reductase 1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of South Florida","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of South Florida \/ EDISON PHARMACEUTICALS INC | Friedreich's Ataxia Research Alliance","highestDevelopmentStatusID":"8","companyTruncated":"University of South Florida \/ EDISON PHARMACEUTICALS INC | Friedreich's Ataxia Research Alliance"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"Quinone reductase 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"Quinone reductase 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"Quinone reductase 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"Quinone reductase 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"Quinone reductase 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"Quinone reductase 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"Quinone reductase 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"Quinone reductase 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stargazer Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"STG-001","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Stargazer Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Stargazer Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Stargazer Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Stargazer Pharmaceuticals","sponsor":"Organovo Holdings","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Series A Financing","leadProduct":"STG-001","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Stargazer Pharmaceuticals","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Stargazer Pharmaceuticals \/ Organovo Holdings","highestDevelopmentStatusID":"8","companyTruncated":"Stargazer Pharmaceuticals \/ Organovo Holdings"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Benchmark Company","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Public Offering","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Benchmark Company","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Benchmark Company"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"The Benchmark Company","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Public Offering","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ The Benchmark Company","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ The Benchmark Company"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Belite Bio","sponsor":"SVB Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ SVB Securities","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ SVB Securities"},{"orgOrder":0,"company":"Belite Bio","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Private Placement","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belite Bio","amount2":0.28000000000000003,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.28000000000000003,"dosageForm":"Tablet","sponsorNew":"Belite Bio \/ RA Capital Management","highestDevelopmentStatusID":"10","companyTruncated":"Belite Bio \/ RA Capital Management"},{"orgOrder":0,"company":"RBP4","sponsor":"Belite Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"RBP4","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"RBP4 \/ Belite Bio","highestDevelopmentStatusID":"7","companyTruncated":"RBP4 \/ Belite Bio"},{"orgOrder":0,"company":"SparingVision","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Undisclosed"},{"orgOrder":0,"company":"SparingVision","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Undisclosed"},{"orgOrder":0,"company":"SparingVision","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Undisclosed"},{"orgOrder":0,"company":"SparingVision","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Undisclosed"},{"orgOrder":0,"company":"SparingVision","sponsor":"4BIO Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Financing","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Injection","sponsorNew":"SparingVision \/ 4BIO Capital","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ 4BIO Capital"},{"orgOrder":0,"company":"SparingVision","sponsor":"Jeito Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2022","type":"Series B Financing","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Jeito Capital","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Jeito Capital"},{"orgOrder":0,"company":"Agtc","sponsor":"SparingVision","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ SparingVision","highestDevelopmentStatusID":"5","companyTruncated":"Agtc \/ SparingVision"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Recifercept","moa":"receptor molecules","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Recifercept","moa":"receptor molecules","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"RO7239361","moa":"receptor molecules","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"RO7239361","moa":"receptor molecules","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"R-Pharm","sponsor":"Data Management 365 | Atlant Clinical | R-Pharm | UNIMED Laboratories | Exacte Labs | Key Stat | Inpharmus | Ascot E\u011fitim ve Dan\u0131\u015fmanl\u0131k","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"RPH-104","moa":"receptor molecules","graph1":"Genetic Disease","graph2":"Phase II","graph3":"R-Pharm","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"R-Pharm \/ Data Management 365 | Atlant Clinical | R-Pharm | UNIMED Laboratories | Exacte Labs | Key Stat | Inpharmus | Ascot E\u011fitim ve Dan\u0131\u015fmanl\u0131k","highestDevelopmentStatusID":"8","companyTruncated":"R-Pharm \/ Data Management 365 | Atlant Clinical | R-Pharm | UNIMED Laboratories | Exacte Labs | Key Stat | Inpharmus | Ascot E\u011fitim ve Dan\u0131\u015fmanl\u0131k"},{"orgOrder":0,"company":"R-Pharm","sponsor":"Atlant Clinical | UNIMED Laboratories | Data Management 365 | R-Pharm | Inpharmus | Exacte Labs | Key Stat","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"RPH-104","moa":"receptor molecules","graph1":"Genetic Disease","graph2":"Phase II","graph3":"R-Pharm","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"R-Pharm \/ Atlant Clinical | UNIMED Laboratories | Data Management 365 | R-Pharm | Inpharmus | Exacte Labs | Key Stat","highestDevelopmentStatusID":"8","companyTruncated":"R-Pharm \/ Atlant Clinical | UNIMED Laboratories | Data Management 365 | R-Pharm | Inpharmus | Exacte Labs | Key Stat"},{"orgOrder":0,"company":"Biohaven Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Taldefgrobep Alfa","moa":"receptor molecules","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Biohaven Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biohaven Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Biohaven Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Tesevatinib","moa":"Receptor protein-tyrosine kinase erbB-2 | Epidermal growth factor receptor erbB1 | Ephrin type-B receptor 4","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Tesevatinib","moa":"Receptor protein-tyrosine kinase erbB-2 | Epidermal growth factor receptor erbB1 | Ephrin type-B receptor 4","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Tesevatinib","moa":"Receptor protein-tyrosine kinase erbB-2 | Epidermal growth factor receptor erbB1 | Ephrin type-B receptor 4","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"The National Comprehensive Cancer Network","sponsor":"Puma Biotechnology","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Collaboration","leadProduct":"Neratinib","moa":"Receptor protein-tyrosine kinase erbB-4 | Epidermal growth factor receptor erbB1 | Receptor protein-tyrosine kinase erbB-2","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"The National Comprehensive Cancer Network","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"The National Comprehensive Cancer Network \/ Puma Biotechnology","highestDevelopmentStatusID":"1","companyTruncated":"The National Comprehensive Cancer Network \/ Puma Biotechnology"},{"orgOrder":0,"company":"Afimmune","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Epeleuton","moa":"Reperfusion stress","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Afimmune","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Afimmune \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Afimmune \/ Undisclosed"},{"orgOrder":0,"company":"Laurent Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"LAU-7b","moa":"Retinoic acid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Laurent Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Laurent Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"8","companyTruncated":"Laurent Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Rege Nephro Co., Ltd.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2024","type":"Inapplicable","leadProduct":"Tamibarotene","moa":"Retinoic acid receptor alpha | Retinoic acid receptor beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rege Nephro Co., Ltd.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rege Nephro Co., Ltd. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Rege Nephro Co., Ltd. \/ Undisclosed"},{"orgOrder":0,"company":"Rege Nephro","sponsor":"DCI Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2024","type":"Series B Financing","leadProduct":"Tamibarotene","moa":"Retinoic acid receptor alpha | Retinoic acid receptor beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rege Nephro","amount2":0.02,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Undisclosed","sponsorNew":"Rege Nephro \/ DCI Partners","highestDevelopmentStatusID":"8","companyTruncated":"Rege Nephro \/ DCI Partners"},{"orgOrder":0,"company":"Rege Nephro","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2024","type":"Inapplicable","leadProduct":"Tamibarotene","moa":"Retinoic acid receptor alpha | Retinoic acid receptor beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rege Nephro","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rege Nephro \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Rege Nephro \/ Undisclosed"},{"orgOrder":0,"company":"Syros Pharmaceuticals","sponsor":"Rege Nephro","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2025","type":"Acquisition","leadProduct":"Tamibarotene","moa":"Retinoic acid receptor alpha | Retinoic acid receptor beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Syros Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Syros Pharmaceuticals \/ Rege Nephro","highestDevelopmentStatusID":"8","companyTruncated":"Syros Pharmaceuticals \/ Rege Nephro"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Food and Drug Administration","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Emixustat","moa":"Retinoid isomerohydrolase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kubota Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Kubota Pharmaceutical \/ Food and Drug Administration","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Food and Drug Administration"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Emixustat","moa":"Retinoid isomerohydrolase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kubota Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Kubota Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Emixustat Hydrochloride","moa":"Retinoid isomerohydrolase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kubota Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Kubota Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Emixustat Hydrochloride","moa":"Retinoid isomerohydrolase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kubota Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Kubota Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Emixustat Hydrochloride","moa":"Retinoid isomerohydrolase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kubota Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Kubota Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Emixustat Hydrochloride","moa":"Retinoid isomerohydrolase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kubota Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Kubota Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Emixustat Hydrochloride","moa":"Retinoid isomerohydrolase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kubota Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Kubota Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Laurent Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Fenretinide","moa":"retinol derivatives","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Laurent Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Laurent Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Laurent Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"QLT Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Plant Extract\/Herbal","year":"2012","type":"Inapplicable","leadProduct":"Vitamin A Acetate","moa":"retinol derivatives","graph1":"Genetic Disease","graph2":"Phase I","graph3":"QLT Inc","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"QLT Inc \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"QLT Inc \/ Undisclosed"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"QR-1123","moa":"RHO (Rhodopsin)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ProQR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aurion Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Neltependocel","moa":"Rho-associated protein kinase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Aurion Biotech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aurion Biotech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Aurion Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Clinical Research Consultants","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2025","type":"Inapplicable","leadProduct":"Riboflavin","moa":"Riboflavin kinase; Riboflavin synthase; Flavin reductase (NADPH)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Clinical Research Consultants","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Clinical Research Consultants \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Clinical Research Consultants \/ Undisclosed"},{"orgOrder":0,"company":"Theravia","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Theravia","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Theravia \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Theravia \/ Undisclosed"},{"orgOrder":0,"company":"Theravia","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Theravia","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Theravia \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Theravia \/ Undisclosed"},{"orgOrder":0,"company":"Theravia","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Theravia","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Theravia \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Theravia \/ Undisclosed"},{"orgOrder":0,"company":"Global Health Uganda","sponsor":"Columbia University | The University of Pittsburgh School of Medicine | Gabrail Cancer Center Research | Mulago Hospital, Uganda | Makerere University | Gansu Provincial Maternal and Child Health Care Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UGANDA","productType":"Cytotoxic Drug","year":"2021","type":"Inapplicable","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Global Health Uganda","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Global Health Uganda \/ Columbia University | The University of Pittsburgh School of Medicine | Gabrail Cancer Center Research | Mulago Hospital, Uganda | Makerere University | Gansu Provincial Maternal and Child Health Care Hospital","highestDevelopmentStatusID":"10","companyTruncated":"Global Health Uganda \/ Columbia University | The University of Pittsburgh School of Medicine | Gabrail Cancer Center Research | Mulago Hospital, Uganda | Makerere University | Gansu Provincial Maternal and Child Health Care Hospital"},{"orgOrder":0,"company":"Nova Laboratories","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cytotoxic Drug","year":"2018","type":"Inapplicable","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nova Laboratories","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Nova Laboratories \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nova Laboratories \/ Undisclosed"},{"orgOrder":0,"company":"Theravia","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cytotoxic Drug","year":"2015","type":"Inapplicable","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Theravia","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Theravia \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Theravia \/ Undisclosed"},{"orgOrder":0,"company":"Theravia","sponsor":"Norgine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cytotoxic Drug","year":"2025","type":"Acquisition","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Theravia","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Theravia \/ Norgine","highestDevelopmentStatusID":"15","companyTruncated":"Theravia \/ Norgine"},{"orgOrder":0,"company":"Theravia","sponsor":"Norgine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cytotoxic Drug","year":"2025","type":"Acquisition","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Theravia","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Theravia \/ Norgine","highestDevelopmentStatusID":"15","companyTruncated":"Theravia \/ Norgine"},{"orgOrder":0,"company":"Children's Hospital Medical Center, Cincinnati","sponsor":"National Heart, Lung, and Blood Institute | Boston Children's Hospital | UT Southwestern Medical Center | Children's Healthcare of Atlanta | Children's Hospital of Philadelphia | The Hospital for Sick Children | Children's National Research Institute | Co","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cytotoxic Drug","year":"2011","type":"Inapplicable","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Children's Hospital Medical Center, Cincinnati","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Children's Hospital Medical Center, Cincinnati \/ National Heart, Lung, and Blood Institute | Boston Children's Hospital | UT Southwestern Medical Center | Children's Healthcare of Atlanta | Children's Hospital of Philadelphia | The Hospital for Sick Children | Children's National Research Institute | Co","highestDevelopmentStatusID":"10","companyTruncated":"Children's Hospital Medical Center, Cincinnati \/ National Heart, Lung, and Blood Institute | Boston Children's Hospital | UT Southwestern Medical Center | Children's Healthcare of Atlanta | Children's Hospital of Philadelphia | The Hospital for Sick Children | Children's National Research Institute | Co"},{"orgOrder":0,"company":"Brown University","sponsor":"National Heart, Lung, and Blood Institute | Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cytotoxic Drug","year":"2022","type":"Inapplicable","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Brown University","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Brown University \/ National Heart, Lung, and Blood Institute | Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"10","companyTruncated":"Brown University \/ National Heart, Lung, and Blood Institute | Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"ASIS Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Gadolinium","moa":"Ribonucleoside-diphosphate reductase RR1 | Thioredoxin reductase 1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ASIS Corporation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ASIS Corporation \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ASIS Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Vico Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Vico Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Vico Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vico Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vico Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Seroba","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2024","type":"Series B Financing","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Undisclosed","sponsorNew":"Vico Therapeutics \/ Seroba","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Seroba"},{"orgOrder":0,"company":"Neurelis","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"NRL-1049","moa":"ROCK","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Neurelis","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurelis \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Neurelis \/ Undisclosed"},{"orgOrder":0,"company":"Neurelis","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"NRL-1049","moa":"ROCK","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Neurelis","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurelis \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Neurelis \/ Undisclosed"},{"orgOrder":0,"company":"BioAxone BioSciences","sponsor":"Neurelis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Acquisition","leadProduct":"BA-1049","moa":"ROCK2","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"BioAxone BioSciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioAxone BioSciences \/ Neurelis","highestDevelopmentStatusID":"4","companyTruncated":"BioAxone BioSciences \/ Neurelis"},{"orgOrder":0,"company":"Ovid Therapeutics","sponsor":"Graviton Bioscience","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"GV101","moa":"ROCK2","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Ovid Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ovid Therapeutics \/ Graviton Bioscience","highestDevelopmentStatusID":"6","companyTruncated":"Ovid Therapeutics \/ Graviton Bioscience"},{"orgOrder":0,"company":"Redx Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"RXC007","moa":"ROCK2","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Redx Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Redx Pharma \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Redx Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"CPK850","moa":"RPE65","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"4D-125","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Public Offering","leadProduct":"4D-125","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"4D Molecular Therapeutics \/ Goldman Sachs & Co","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"4D-125","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"4D-125","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Financing","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0.12,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Injection","sponsorNew":"Beacon Therapeutics \/ Syncona Limited","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Syncona Limited"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"TCGX","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Series B Financing","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0.17000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17000000000000001,"dosageForm":"Injection","sponsorNew":"Beacon Therapeutics \/ TCGX","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ TCGX"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"TCGX","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Series B Financing","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17000000000000001,"dosageForm":"Injection","sponsorNew":"Beacon Therapeutics \/ TCGX","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ TCGX"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"Cantor Fitzgerald","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Public Offering","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0.01,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Injection","sponsorNew":"Agtc \/ Cantor Fitzgerald","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Cantor Fitzgerald"},{"orgOrder":0,"company":"Agtc","sponsor":"H.C. Wainwright & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Public Offering","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ H.C. Wainwright & Co","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ H.C. Wainwright & Co"},{"orgOrder":0,"company":"Agtc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Acquisition","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0.02,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Injection","sponsorNew":"Agtc \/ Syncona Limited","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Syncona Limited"},{"orgOrder":0,"company":"Agtc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Undisclosed"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"rAAV2tYF-GRK1-RPGR","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"rAAV2tYF-GRK1-RPGR","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"rAAV2tYF-GRK1-RPGR","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Agtc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Agtc \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"SRP-9004","moa":"Sarcoglycan alpha (SGCA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Nationwide Children\u2019s Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2013","type":"Inapplicable","leadProduct":"SRP-9004","moa":"Sarcoglycan alpha (SGCA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Nationwide Children\u2019s Hospital","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Nationwide Children\u2019s Hospital"},{"orgOrder":0,"company":"Lingyi Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"LY-M001","moa":"SARS-CoV-2 spike protein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lingyi Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lingyi Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lingyi Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2011","type":"Inapplicable","leadProduct":"BPS804","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"BPS804","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"8","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group | Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2011","type":"Inapplicable","leadProduct":"BPS804","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group | Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"8","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group | Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Romosozumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Romosozumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mereo BioPharma Group \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Undisclosed"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mereo BioPharma Group \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Undisclosed"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibody, Unconjugated","year":"2024","type":"Public Offering","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mereo BioPharma Group \/ Jefferies","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Jefferies"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibody, Unconjugated","year":"2020","type":"Collaboration","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0.29999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.29999999999999999,"dosageForm":"Infusion","sponsorNew":"Mereo BioPharma Group \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Public Offering","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ J.P. Morgan"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Public Offering","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ J.P. Morgan"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Public Offering","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0.29999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ J.P. Morgan"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Public Offering","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0.33000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ J.P. Morgan"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Crescent Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Uproleselan","moa":"Selectin E","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Crescent Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Crescent Biopharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Crescent Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Evotec","sponsor":"Bayer AG","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2023","type":"Collaboration","leadProduct":"BAY401016","moa":"Sema3A","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Evotec","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Evotec \/ Bayer AG","highestDevelopmentStatusID":"6","companyTruncated":"Evotec \/ Bayer AG"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Pepinemab","moa":"Semaphorin-4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vaccinex","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vaccinex \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vaccinex \/ Undisclosed"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Pepinemab","moa":"Semaphorin-4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vaccinex","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vaccinex \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vaccinex \/ Undisclosed"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Pepinemab","moa":"Semaphorin-4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vaccinex","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vaccinex \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vaccinex \/ Undisclosed"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Pepinemab","moa":"Semaphorin-4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vaccinex","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vaccinex \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vaccinex \/ Undisclosed"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Pepinemab","moa":"Semaphorin-4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vaccinex","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vaccinex \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vaccinex \/ Undisclosed"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Pepinemab","moa":"Semaphorin-4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vaccinex","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vaccinex \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vaccinex \/ Undisclosed"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Pepinemab","moa":"Semaphorin-4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vaccinex","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vaccinex \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vaccinex \/ Undisclosed"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Pepinemab","moa":"Semaphorin-4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vaccinex","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vaccinex \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vaccinex \/ Undisclosed"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Keystone Capital Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Financing","leadProduct":"Pepinemab","moa":"Semaphorin-4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vaccinex","amount2":0.02,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Infusion","sponsorNew":"Vaccinex \/ Keystone Capital Partners","highestDevelopmentStatusID":"8","companyTruncated":"Vaccinex \/ Keystone Capital Partners"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Private Placement","leadProduct":"Pepinemab","moa":"Semaphorin-4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vaccinex","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vaccinex \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vaccinex \/ Undisclosed"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Huntington Study Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2015","type":"Inapplicable","leadProduct":"VX15\/2503","moa":"Semaphorin-4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vaccinex","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vaccinex \/ Huntington Study Group","highestDevelopmentStatusID":"8","companyTruncated":"Vaccinex \/ Huntington Study Group"},{"orgOrder":0,"company":"Lixte Biotechnology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"LB-100","moa":"||Serine\/threonine protein phosphatase 2A, catalytic subunit, beta isoform | Serine\/threonine protein phosphatase 2A, catalytic subunit, alpha isoform | Serine\/threonine-protein phosphatase 5","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Lixte Biotechnology","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Lixte Biotechnology \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Lixte Biotechnology \/ Undisclosed"},{"orgOrder":0,"company":"Massachusetts General Hospital","sponsor":"AstraZeneca | US Department of Defense","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Vistusertib","moa":"Serine\/threonine-protein kinase mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Massachusetts General Hospital","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Massachusetts General Hospital \/ AstraZeneca | US Department of Defense","highestDevelopmentStatusID":"8","companyTruncated":"Massachusetts General Hospital \/ AstraZeneca | US Department of Defense"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Bayer AG","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Sorafenib","moa":"Serine\/threonine-protein kinase RAF","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"NYU Langone Health \/ Bayer AG","highestDevelopmentStatusID":"8","companyTruncated":"NYU Langone Health \/ Bayer AG"},{"orgOrder":0,"company":"Nova Mentis Life Science","sponsor":"KGK Science","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Psilocybine","moa":"Serotonin 2a (5-HT2a) receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nova Mentis Life Science","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Nova Mentis Life Science \/ KGK Science","highestDevelopmentStatusID":"8","companyTruncated":"Nova Mentis Life Science \/ KGK Science"},{"orgOrder":0,"company":"Nova Mentis Life Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Controlled Substance","year":"2021","type":"Inapplicable","leadProduct":"Psilocybine","moa":"Serotonin 2a (5-HT2a) receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nova Mentis Life Science","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Nova Mentis Life Science \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nova Mentis Life Science \/ Undisclosed"},{"orgOrder":0,"company":"KGK Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Controlled Substance","year":"2023","type":"Inapplicable","leadProduct":"Psilocybine","moa":"Serotonin 2a (5-HT2a) receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"KGK Science","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"KGK Science \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"KGK Science \/ Undisclosed"},{"orgOrder":0,"company":"Nova Mentis Life Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Controlled Substance","year":"2021","type":"Inapplicable","leadProduct":"Psilocybine","moa":"Serotonin 2a (5-HT2a) receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nova Mentis Life Science","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Nova Mentis Life Science \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nova Mentis Life Science \/ Undisclosed"},{"orgOrder":0,"company":"KGK Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Controlled Substance","year":"2023","type":"Inapplicable","leadProduct":"Psilocybine","moa":"Serotonin 2a (5-HT2a) receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"KGK Science","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"KGK Science \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"KGK Science \/ Undisclosed"},{"orgOrder":0,"company":"Wellbeing Digital Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Controlled Substance","year":"2022","type":"Inapplicable","leadProduct":"Psilocybine","moa":"Serotonin 2a (5-HT2a) receptor","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Wellbeing Digital Sciences","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Wellbeing Digital Sciences \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Wellbeing Digital Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wellbeing Digital Sciences","sponsor":"Nova Mentis Life Science","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Controlled Substance","year":"2022","type":"Agreement","leadProduct":"Psilocybine","moa":"Serotonin 2a (5-HT2a) receptor","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Wellbeing Digital Sciences","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Wellbeing Digital Sciences \/ Nova Mentis Life Science","highestDevelopmentStatusID":"5","companyTruncated":"Wellbeing Digital Sciences \/ Nova Mentis Life Science"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"ARO-AAT","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2017","type":"Inapplicable","leadProduct":"ARO-AAT","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Belcesiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Dicerna Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Dicerna Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Dicerna Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Belcesiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Dicerna Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Dicerna Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Dicerna Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Belcesiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Dicerna Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Dicerna Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Dicerna Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Belcesiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Dicerna Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dicerna Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Dicerna Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Belcesiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Dicerna Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dicerna Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Dicerna Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Arrowhead Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Arrowhead Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Arrowhead Pharmaceuticals"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Public Offering","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0.45000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.45000000000000001,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Jefferies"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Public Offering","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0.45000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.45000000000000001,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Jefferies"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Collaboration","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":1.04,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":1.04,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"9","companyTruncated":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Collaboration","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":1.04,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":1.04,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Licensing Agreement","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":1.04,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":1.04,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"9","companyTruncated":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"GSK2315698","moa":"Serum amyloid P-component","graph1":"Genetic Disease","graph2":"Phase II","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"GSK2315698","moa":"Serum amyloid P-component","graph1":"Genetic Disease","graph2":"Phase II","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"GSK2315698","moa":"Serum amyloid P-component","graph1":"Genetic Disease","graph2":"Phase II","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"GSK2315698","moa":"Serum amyloid P-component","graph1":"Genetic Disease","graph2":"Phase II","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"GSK2315698","moa":"Serum amyloid P-component","graph1":"Genetic Disease","graph2":"Phase II","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Imperial College London | Heart Hospital | Royal Free Hospital NHS FT | Quintiles Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibody, Unconjugated","year":"2013","type":"Inapplicable","leadProduct":"GSK2398852","moa":"Serum amyloid P-component","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"GSK \/ Imperial College London | Heart Hospital | Royal Free Hospital NHS FT | Quintiles Inc","highestDevelopmentStatusID":"6","companyTruncated":"GSK \/ Imperial College London | Heart Hospital | Royal Free Hospital NHS FT | Quintiles Inc"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SRP-9005","moa":"SGCG","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"FRAXA Research Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Blarcamesine","moa":"sigma receptor ligands","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Anavex Life Sciences \/ FRAXA Research Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Anavex Life Sciences \/ FRAXA Research Foundation"},{"orgOrder":0,"company":"Electra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"ELA026","moa":"SIRP-alpha","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Electra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Electra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Electra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Electra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"ELA026","moa":"SIRP-alpha","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Electra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Electra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Electra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Electra Therapeutics","sponsor":"Westlake Village BioPartners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Series B Financing","leadProduct":"ELA026","moa":"SIRP-alpha","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Electra Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Electra Therapeutics \/ Westlake Village BioPartners","highestDevelopmentStatusID":"9","companyTruncated":"Electra Therapeutics \/ Westlake Village BioPartners"},{"orgOrder":0,"company":"CSL Vifor","sponsor":"Fortrea","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Vamifeport","moa":"Ferroportin||SLC40A1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CSL Vifor","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"CSL Vifor \/ Fortrea","highestDevelopmentStatusID":"8","companyTruncated":"CSL Vifor \/ Fortrea"},{"orgOrder":0,"company":"CSL","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Vamifeport","moa":"SLC40A1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CSL","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"CSL \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"CSL \/ Undisclosed"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"JNT-517","moa":"SLC6A19","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jnana Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Jnana Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Jnana Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JNT-517","moa":"SLC6A19","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jnana Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Jnana Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Jnana Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"Otsuka Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"JNT-517","moa":"SLC6A19","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jnana Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Jnana Therapeutics \/ Otsuka Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Jnana Therapeutics \/ Otsuka Pharmaceutical"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JNT-517","moa":"SLC6A19","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jnana Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Jnana Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Jnana Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JNT-517","moa":"SLC6A19","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jnana Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jnana Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Jnana Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"Otsuka Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Acquisition","leadProduct":"JNT-517","moa":"SLC6A19","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jnana Therapeutics","amount2":1.1299999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":1.1299999999999999,"dosageForm":"Undisclosed","sponsorNew":"Jnana Therapeutics \/ Otsuka Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Jnana Therapeutics \/ Otsuka Pharmaceutical"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"JNT-517","moa":"SLC6A19","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jnana Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jnana Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Jnana Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"JNT-517","moa":"SLC6A19","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jnana Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jnana Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Jnana Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"Bain Capital Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Series C Financing","leadProduct":"JNT-517","moa":"SLC6A19","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jnana Therapeutics","amount2":0.11,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"Jnana Therapeutics \/ Bain Capital Life Sciences","highestDevelopmentStatusID":"10","companyTruncated":"Jnana Therapeutics \/ Bain Capital Life Sciences"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"JNT-517","moa":"SLC6A19","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jnana Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jnana Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Jnana Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"OAV101","moa":"SMN1","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"OAV101","moa":"SMN1","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"OAV101","moa":"SMN1","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"OAV101","moa":"SMN1","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"OAV-101","moa":"SMN1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Licensing Agreement","leadProduct":"BIIB115","moa":"SMN1 messenger RNA (SMN1 mRNA)","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ Biogen","highestDevelopmentStatusID":"4","companyTruncated":"Ionis Pharmaceuticals \/ Biogen"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2013","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2011","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2013","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2016","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2012","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Winthrop University Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase I","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NYU Langone Health \/ Winthrop University Hospital","highestDevelopmentStatusID":"6","companyTruncated":"NYU Langone Health \/ Winthrop University Hospital"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Generium","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Generium \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Generium \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Agreement","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":1.1299999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":1.1299999999999999,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Royalty Pharma","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Royalty Pharma"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Collaboration","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alcyone Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Injection","sponsorNew":"Alcyone Therapeutics \/ Biogen","highestDevelopmentStatusID":"15","companyTruncated":"Alcyone Therapeutics \/ Biogen"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Biogen","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alcyone Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alcyone Therapeutics \/ Biogen","highestDevelopmentStatusID":"15","companyTruncated":"Alcyone Therapeutics \/ Biogen"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Chugai Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Chugai Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Chugai Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Chugai Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Natco Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Natco Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Natco Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Natco Pharma \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ PTC Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ PTC Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ PTC Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ PTC Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ PTC Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ PTC Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"Clinic for Special Children","sponsor":"Genentech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Clinic for Special Children","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Insert","sponsorNew":"Clinic for Special Children \/ Genentech","highestDevelopmentStatusID":"11","companyTruncated":"Clinic for Special Children \/ Genentech"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Spinal Muscular Atrophy Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Partnership","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Spinal Muscular Atrophy Foundation","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Spinal Muscular Atrophy Foundation"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Collaboration","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0.48999999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.48999999999999999,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Agreement","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":1.5,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":1.5,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Royalty Pharma","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Royalty Pharma"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Licensing Agreement","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0.48999999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.48999999999999999,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Acquisition","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0.65000000000000002,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.65000000000000002,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Royalty Pharma","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Royalty Pharma"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"Genentech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genentech \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"WVE-003","moa":"SNP3","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-003","moa":"SNP3","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"WVE-003","moa":"SNP3","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Termination","leadProduct":"WVE-003","moa":"SNP3","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0.26000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.26000000000000001,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Washington University School of Medicine","sponsor":"Novartis Pharmaceuticals Corporation | National Institutes of Health | National Institute of Diabetes and Digestive and Kidney Diseases | The University of Pittsburgh School of Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Carbamazepine","moa":"Sodium channel alpha subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Washington University School of Medicine","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Washington University School of Medicine \/ Novartis Pharmaceuticals Corporation | National Institutes of Health | National Institute of Diabetes and Digestive and Kidney Diseases | The University of Pittsburgh School of Medicine","highestDevelopmentStatusID":"8","companyTruncated":"Washington University School of Medicine \/ Novartis Pharmaceuticals Corporation | National Institutes of Health | National Institute of Diabetes and Digestive and Kidney Diseases | The University of Pittsburgh School of Medicine"},{"orgOrder":0,"company":"Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation","sponsor":"Universitaire Ziekenhuizen KU Leuven | ZonMw | Hospital Sant Joan de Deu","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Lamotrigine","moa":"Sodium channel alpha subunit","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation \/ Universitaire Ziekenhuizen KU Leuven | ZonMw | Hospital Sant Joan de Deu","highestDevelopmentStatusID":"9","companyTruncated":"Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation \/ Universitaire Ziekenhuizen KU Leuven | ZonMw | Hospital Sant Joan de Deu"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Mexiletine","moa":"Sodium channel alpha subunit","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Lupin Ltd \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Lupin Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Lupin Ltd","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Mexiletine","moa":"Sodium channel alpha subunit","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Lupin Ltd \/ Lupin Ltd","highestDevelopmentStatusID":"10","companyTruncated":"Lupin Ltd \/ Lupin Ltd"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Lupin Ltd","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Mexiletine","moa":"Sodium channel alpha subunit","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Lupin Ltd \/ Lupin Ltd","highestDevelopmentStatusID":"10","companyTruncated":"Lupin Ltd \/ Lupin Ltd"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Mexiletine","moa":"Sodium channel alpha subunit","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Lupin Ltd \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Lupin Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Exeltis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2020","type":"Agreement","leadProduct":"Mexiletine","moa":"Sodium channel alpha subunit","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Lupin Ltd \/ Exeltis","highestDevelopmentStatusID":"15","companyTruncated":"Lupin Ltd \/ Exeltis"},{"orgOrder":0,"company":"Ohio State University","sponsor":"Gilead Sciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Ranolazine","moa":"Sodium channel protein type V alpha subunit | Sodium channel protein type IV alpha subunit","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Ohio State University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ohio State University \/ Gilead Sciences","highestDevelopmentStatusID":"6","companyTruncated":"Ohio State University \/ Gilead Sciences"},{"orgOrder":0,"company":"Hannover Medical School","sponsor":"Boehringer Ingelheim GmbH","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Empagliflozin","moa":"Sodium\/glucose cotransporter 2","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Hannover Medical School","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hannover Medical School \/ Boehringer Ingelheim GmbH","highestDevelopmentStatusID":"11","companyTruncated":"Hannover Medical School \/ Boehringer Ingelheim GmbH"},{"orgOrder":0,"company":"Massachusetts General Hospital","sponsor":"Ardelyx","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Tenapanor","moa":"Sodium\/hydrogen exchanger 3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Massachusetts General Hospital","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Massachusetts General Hospital \/ Ardelyx","highestDevelopmentStatusID":"10","companyTruncated":"Massachusetts General Hospital \/ Ardelyx"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Digoxin","moa":"Sodium\/potassium-transporting ATPase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Sun Yat-sen University","sponsor":"Zhujiang Hospital | Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | Jeeyor Medical Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Ginkgo Biloba","moa":"Sodium-dependent noradrenaline transporter; Phospholipase A2, membrane associated; Glycine receptor subunit alpha-1; Gamma-aminobutyric acid receptor subunit alpha-1; Gamma-aminobutyric acid receptor subunit beta-2; Gamma-aminobutyric acid receptor subunit gamma-2; Amine oxidase [flavin-containing] A; Nitric oxide synthase, inducible","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sun Yat-sen University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sun Yat-sen University \/ Zhujiang Hospital | Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | Jeeyor Medical Research","highestDevelopmentStatusID":"6","companyTruncated":"Sun Yat-sen University \/ Zhujiang Hospital | Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | Jeeyor Medical Research"},{"orgOrder":0,"company":"Novoteris","sponsor":"Cystic Fibrosis Foundation | Mallinckrodt Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Nitric Oxide","moa":"Soluble guanylate cyclase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novoteris","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novoteris \/ Cystic Fibrosis Foundation | Mallinckrodt Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Novoteris \/ Cystic Fibrosis Foundation | Mallinckrodt Pharmaceuticals"},{"orgOrder":0,"company":"Beyond Air","sponsor":"Soroka University Medical Center | Schneider Children's Medical Center","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Nitric Oxide","moa":"Soluble guanylate cyclase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beyond Air","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beyond Air \/ Soroka University Medical Center | Schneider Children's Medical Center","highestDevelopmentStatusID":"7","companyTruncated":"Beyond Air \/ Soroka University Medical Center | Schneider Children's Medical Center"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Olinciguat","moa":"Soluble guanylate cyclase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cyclerion Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cyclerion Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cyclerion Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Olinciguat","moa":"Soluble guanylate cyclase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cyclerion Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cyclerion Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cyclerion Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Merck & Co","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Riociguat","moa":"Soluble guanylate cyclase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Merck & Co","highestDevelopmentStatusID":"8","companyTruncated":"Bayer AG \/ Merck & Co"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"CTI-1601","moa":"Soluble guanylate cyclase (GCS)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"CTI-1601","moa":"Soluble guanylate cyclase (GCS)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Larimar Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Veristat | Metrum Research Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"CTI-1601","moa":"Soluble guanylate cyclase (GCS)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Larimar Therapeutics \/ Veristat | Metrum Research Group","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Veristat | Metrum Research Group"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Veristat | Metrum Research Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"CTI-1601","moa":"Soluble guanylate cyclase (GCS)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Larimar Therapeutics \/ Veristat | Metrum Research Group","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Veristat | Metrum Research Group"},{"orgOrder":0,"company":"Mayo Clinic","sponsor":"Otsuka Holdings","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Probenecid","moa":"Solute carrier family 22 member 6 | Solute carrier family 22 member 8 | Solute carrier family 22 member 11","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mayo Clinic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mayo Clinic \/ Otsuka Holdings","highestDevelopmentStatusID":"8","companyTruncated":"Mayo Clinic \/ Otsuka Holdings"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Camurus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Octreotide Acetate","moa":"Somatostatin receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Camurus","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Camurus \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Camurus \/ Undisclosed"},{"orgOrder":0,"company":"Universitaire Ziekenhuizen KU Leuven","sponsor":"Ipsen","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Lanreotide Acetate","moa":"Somatostatin receptor 5","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Universitaire Ziekenhuizen KU Leuven","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Universitaire Ziekenhuizen KU Leuven \/ Ipsen","highestDevelopmentStatusID":"9","companyTruncated":"Universitaire Ziekenhuizen KU Leuven \/ Ipsen"},{"orgOrder":0,"company":"Assistance Publique \u2013 H\u00f4pitaux de Paris","sponsor":"Ipsen","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Lanreotide Acetate","moa":"Somatostatin receptor 5","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Assistance Publique \u2013 H\u00f4pitaux de Paris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Ipsen","highestDevelopmentStatusID":"10","companyTruncated":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Ipsen"},{"orgOrder":0,"company":"University of Colorado, Denver","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Pasireotide","moa":"Somatostatin receptor 5","graph1":"Genetic Disease","graph2":"Phase I","graph3":"University of Colorado, Denver","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Colorado, Denver \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"6","companyTruncated":"University of Colorado, Denver \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Montefiore Medical Center","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Pasireotide","moa":"Somatostatin receptor 5","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Montefiore Medical Center","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Montefiore Medical Center \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"8","companyTruncated":"Montefiore Medical Center \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Enzyme","year":"2025","type":"Inapplicable","leadProduct":"Olipudase Alfa","moa":"Sphingomyelin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"CAT-2003","moa":"SREBP-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Catabasis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Catabasis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Camurus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"CAM2029","moa":"SST receptor","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Camurus","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Camurus \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Camurus \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"CRN04777","moa":"SST5 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"CRN04777","moa":"SST5 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"CRN04777","moa":"SST5 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"CRN04777","moa":"SST5 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"CRN04777","moa":"SST5 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BBP-631","moa":"Steroid 21-hydroxylase (CYP21A2)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"BBP-631","moa":"Steroid 21-hydroxylase (CYP21A2)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Millendo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Nevanimibe","moa":"Sterol O-acyltransferase (SOAT)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Millendo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Millendo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Millendo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Alirocumab","moa":"Subtilisin\/kexin type 9","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regeneron Pharmaceuticals \/ Sanofi","highestDevelopmentStatusID":"15","companyTruncated":"Regeneron Pharmaceuticals \/ Sanofi"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Alirocumab","moa":"Subtilisin\/kexin type 9","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Evolocumab","moa":"Subtilisin\/kexin type 9","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2012","type":"Inapplicable","leadProduct":"Evolocumab","moa":"Subtilisin\/kexin type 9","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Innovent Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"IBI306","moa":"Subtilisin\/kexin type 9","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Innovent Biologics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Innovent Biologics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Innovent Biologics \/ Undisclosed"},{"orgOrder":0,"company":"LIB Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Lerodalcibep","moa":"Subtilisin\/kexin type 9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"LIB Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"LIB Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"LIB Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2013","type":"Inapplicable","leadProduct":"Antihemophilic Factor [Recombinant]","moa":"Sucrose","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Antihemophilic Factor [Recombinant]","moa":"Sucrose","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"QOL Medical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2016","type":"Inapplicable","leadProduct":"Sacrosidase","moa":"Sucrose","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"QOL Medical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"QOL Medical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"QOL Medical \/ Undisclosed"},{"orgOrder":0,"company":"QOL Medical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Sacrosidase","moa":"Sucrose","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"QOL Medical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"QOL Medical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"QOL Medical \/ Undisclosed"},{"orgOrder":0,"company":"QOL Medical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Sacrosidase","moa":"Sucrose","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"QOL Medical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"QOL Medical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"QOL Medical \/ Undisclosed"},{"orgOrder":0,"company":"QOL Medical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Inapplicable","leadProduct":"Sacrosidase","moa":"Sucrose","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"QOL Medical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"QOL Medical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"QOL Medical \/ Undisclosed"},{"orgOrder":0,"company":"QOL Medical","sponsor":"Optum Frontier Therapies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Partnership","leadProduct":"Sacrosidase","moa":"Sucrose","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"QOL Medical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"QOL Medical \/ Optum Frontier Therapies","highestDevelopmentStatusID":"15","companyTruncated":"QOL Medical \/ Optum Frontier Therapies"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"REC-994","moa":"Superoxide Radical","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Recursion Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Recursion Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Recursion Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Bayer AG","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Series D Financing","leadProduct":"REC-994","moa":"Superoxide Radical","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Recursion Pharma","amount2":0.23999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23999999999999999,"dosageForm":"Tablet","sponsorNew":"Recursion Pharma \/ Bayer AG","highestDevelopmentStatusID":"8","companyTruncated":"Recursion Pharma \/ Bayer AG"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Branaplam","moa":"survival motor neurin SMN1 and SMN2 splicing modulators","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Branaplam","moa":"survival motor neurin SMN1 and SMN2 splicing modulators","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Branaplam","moa":"survival motor neurin SMN1 and SMN2 splicing modulators","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Branaplam","moa":"survival motor neurin SMN1 and SMN2 splicing modulators","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Branaplam","moa":"survival motor neurin SMN1 and SMN2 splicing modulators","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"United BioSource","sponsor":"AveXis, Inc.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"AVXS-101","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"United BioSource","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"United BioSource \/ AveXis, Inc.","highestDevelopmentStatusID":"1","companyTruncated":"United BioSource \/ AveXis, Inc."},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2014","type":"Inapplicable","leadProduct":"AVXS-101","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"PRA Health Sciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ PRA Health Sciences","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ PRA Health Sciences"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"PRA Health Sciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ PRA Health Sciences","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ PRA Health Sciences"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Agreement","leadProduct":"Onasemnogene Abeparvovec","moa":"||Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Regenxbio","amount2":0.080000000000000002,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"15","companyTruncated":"Regenxbio \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Healthcare Royalty Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Agreement","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Healthcare Royalty Management","highestDevelopmentStatusID":"15","companyTruncated":"Regenxbio \/ Healthcare Royalty Management"},{"orgOrder":0,"company":"SOM Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"SOM3355","moa":"Synaptic vesicle amine transporter (SLC18A2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SOM Biotech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"SOM Biotech \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SOM Biotech \/ Undisclosed"},{"orgOrder":0,"company":"SOM Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"SOM3355","moa":"Synaptic vesicle amine transporter (SLC18A2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SOM Biotech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"SOM Biotech \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SOM Biotech \/ Undisclosed"},{"orgOrder":0,"company":"SOM Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"SOM3355","moa":"Synaptic vesicle amine transporter (SLC18A2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SOM Biotech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"SOM Biotech \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SOM Biotech \/ Undisclosed"},{"orgOrder":0,"company":"SOM Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"SOM3355","moa":"Synaptic vesicle amine transporter (SLC18A2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SOM Biotech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"SOM Biotech \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SOM Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Jiangsu Nhwa Pharmaceutical","sponsor":"Teva Pharmaceutical Industries","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2024","type":"Partnership","leadProduct":"Deutetrabenazine","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Jiangsu Nhwa Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Jiangsu Nhwa Pharmaceutical \/ Teva Pharmaceutical Industries","highestDevelopmentStatusID":"15","companyTruncated":"Jiangsu Nhwa Pharmaceutical \/ Teva Pharmaceutical Industries"},{"orgOrder":0,"company":"Teva Pharmaceutical Industries","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Deutetrabenazine","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Teva Pharmaceutical Industries","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Teva Pharmaceutical Industries \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Teva Pharmaceutical Industries \/ Undisclosed"},{"orgOrder":0,"company":"Vanderbilt University Medical Center","sponsor":"Teva Pharmaceutical Industries","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Deutetrabenazine","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Vanderbilt University Medical Center","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vanderbilt University Medical Center \/ Teva Pharmaceutical Industries","highestDevelopmentStatusID":"9","companyTruncated":"Vanderbilt University Medical Center \/ Teva Pharmaceutical Industries"},{"orgOrder":0,"company":"Auspex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Deutetrabenazine","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Auspex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Auspex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Auspex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Huntington Study Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Valbenazine Tosylate","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Huntington Study Group","highestDevelopmentStatusID":"10","companyTruncated":"Neurocrine Biosciences \/ Huntington Study Group"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Huntington Study Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Valbenazine Tosylate","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Huntington Study Group","highestDevelopmentStatusID":"10","companyTruncated":"Neurocrine Biosciences \/ Huntington Study Group"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Valbenazine Tosylate","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Valbenazine Tosylate","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Valbenazine Tosylate","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Valbenazine Tosylate","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Valbenazine Tosylate","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Valbenazine Tosylate","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"CAMP4 Therapeutics","sponsor":"Coastlands Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Private Placement","leadProduct":"CMP-SYNGAP","moa":"SYNGAP1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CAMP4 Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Undisclosed","sponsorNew":"CAMP4 Therapeutics \/ Coastlands Capital","highestDevelopmentStatusID":"4","companyTruncated":"CAMP4 Therapeutics \/ Coastlands Capital"},{"orgOrder":0,"company":"Gedeon Richter","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"HUNGARY","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"RGH-706","moa":"TAAR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Gedeon Richter","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Gedeon Richter \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Gedeon Richter \/ Undisclosed"},{"orgOrder":0,"company":"Aardvark Therapeutics","sponsor":"Children's Hospital Colorado | Stanford University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Denatonium Acetate","moa":"TAS2 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aardvark Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aardvark Therapeutics \/ Children's Hospital Colorado | Stanford University","highestDevelopmentStatusID":"8","companyTruncated":"Aardvark Therapeutics \/ Children's Hospital Colorado | Stanford University"},{"orgOrder":0,"company":"Aardvark Therapeutics","sponsor":"Cantor Fitzgerald","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Series C Financing","leadProduct":"Denatonium Acetate","moa":"TAS2 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aardvark Therapeutics","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Pill","sponsorNew":"Aardvark Therapeutics \/ Cantor Fitzgerald","highestDevelopmentStatusID":"8","companyTruncated":"Aardvark Therapeutics \/ Cantor Fitzgerald"},{"orgOrder":0,"company":"Aardvark Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Denatonium Acetate","moa":"TAS2 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aardvark Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pill","sponsorNew":"Aardvark Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Aardvark Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aardvark Therapeutics","sponsor":"University of California","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Denatonium Acetate","moa":"TAS2 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aardvark Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pill","sponsorNew":"Aardvark Therapeutics \/ University of California","highestDevelopmentStatusID":"8","companyTruncated":"Aardvark Therapeutics \/ University of California"},{"orgOrder":0,"company":"Cumberland Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Ifetroban Sodium","moa":"TBXA2R","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cumberland Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Cumberland Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cumberland Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Cumberland Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Ifetroban Sodium","moa":"TBXA2R","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cumberland Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cumberland Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cumberland Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharma","sponsor":"California Institute for Regenerative Medicine (CIRM)","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"RP-L401","moa":"T-cell immune regulator 1 (TCIRG1)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Rocket Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rocket Pharma \/ California Institute for Regenerative Medicine (CIRM)","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharma \/ California Institute for Regenerative Medicine (CIRM)"},{"orgOrder":0,"company":"Gabrail Cancer Center Research","sponsor":"Children's Healthcare of Atlanta","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Alefacept","moa":"T-cell surface antigen CD2","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Gabrail Cancer Center Research","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Insert","sponsorNew":"Gabrail Cancer Center Research \/ Children's Healthcare of Atlanta","highestDevelopmentStatusID":"1","companyTruncated":"Gabrail Cancer Center Research \/ Children's Healthcare of Atlanta"},{"orgOrder":0,"company":"Columbia University","sponsor":"ITBMed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Siplizumab","moa":"T-cell surface antigen CD2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Columbia University","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Columbia University \/ ITBMed","highestDevelopmentStatusID":"7","companyTruncated":"Columbia University \/ ITBMed"},{"orgOrder":0,"company":"Bellicum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2014","type":"Inapplicable","leadProduct":"BPX-501","moa":"T-cells (T-cells)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Bellicum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bellicum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Bellicum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2020","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Seelos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Seelos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2021","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Seelos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Seelos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2021","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Seelos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Seelos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2022","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Seelos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Seelos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2020","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Seelos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Seelos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2020","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Seelos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Seelos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2020","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Seelos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Seelos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2020","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Seelos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Seelos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2020","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Seelos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Seelos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2020","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Seelos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Seelos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Bioblast Pharma Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Trehalose API","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bioblast Pharma Ltd","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bioblast Pharma Ltd \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bioblast Pharma Ltd \/ Undisclosed"},{"orgOrder":0,"company":"LFB Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Eptacog Beta","moa":"Thioredoxin reductase (PRDX5)","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"LFB Group","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"LFB Group \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"LFB Group \/ Undisclosed"},{"orgOrder":0,"company":"LFB Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Eptacog Beta","moa":"Thioredoxin reductase (PRDX5)","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"LFB Group","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"LFB Group \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"LFB Group \/ Undisclosed"},{"orgOrder":0,"company":"Sumitomo Pharma Switzerland","sponsor":"National Institutes of Health | National Institute of Allergy and Infectious Diseases | Gansu Provincial Maternal and Child Health Care Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2010","type":"Inapplicable","leadProduct":"Cultured Thymus Tissue","moa":"Thymus tissue","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sumitomo Pharma Switzerland","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Sumitomo Pharma Switzerland \/ National Institutes of Health | National Institute of Allergy and Infectious Diseases | Gansu Provincial Maternal and Child Health Care Hospital","highestDevelopmentStatusID":"1","companyTruncated":"Sumitomo Pharma Switzerland \/ National Institutes of Health | National Institute of Allergy and Infectious Diseases | Gansu Provincial Maternal and Child Health Care Hospital"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Antibody, Unconjugated","year":"2015","type":"Inapplicable","leadProduct":"BAY1093884","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"BAY1093884","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"BAY1093884","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Befovacimab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Concizumab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Concizumab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Concizumab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Concizumab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Concizumab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Concizumab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Concizumab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Concizumab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Concizumab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Concizumab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Concizumab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Marstacimab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Marstacimab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Marstacimab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Marstacimab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Marstacimab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Marstacimab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Marstacimab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"PF-06741086","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"PF-06741086","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"PF-06741086","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"PF-06741086","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"PF-06741086","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"IONIS-TMPRSS6-L RX","moa":"TMPRSS6","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"MG1113","moa":"TMPRSS6","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GC Biopharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"GC Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Dream CIS","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"MG1113","moa":"TMPRSS6","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Green Cross Lab Cell \/ Dream CIS","highestDevelopmentStatusID":"6","companyTruncated":"Green Cross Lab Cell \/ Dream CIS"},{"orgOrder":0,"company":"Silence Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"SLN124","moa":"TMPRSS6 messenger RNA (TMPRSS6 mRNA)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Silence Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Silence Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Silence Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Silence Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"SLN124","moa":"TMPRSS6 messenger RNA (TMPRSS6 mRNA)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Silence Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Silence Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Silence Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Linvoseltamab","moa":"TNFRSF17","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"AZD8848","moa":"Toll-like receptor 7","graph1":"Genetic Disease","graph2":"Phase I","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Forest Laboratories","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2010","type":"Inapplicable","leadProduct":"Levofloxacin","moa":"Bacterial ribosome||Topoisomerase IV | DNA gyrase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Amgen Inc \/ Forest Laboratories","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Forest Laboratories"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Forest Laboratories","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2011","type":"Inapplicable","leadProduct":"Levofloxacin","moa":"Topoisomerase IV | DNA gyrase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Inhalation","sponsorNew":"Amgen Inc \/ Forest Laboratories","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Forest Laboratories"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Pabinafusp Alpha","moa":"Transferrin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Pabinafusp Alpha","moa":"Transferrin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Pabinafusp Alpha","moa":"Transferrin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2021","type":"Collaboration","leadProduct":"Pabinafusp Alpha","moa":"Transferrin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"JCR Pharmaceuticals \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Inapplicable","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Inapplicable","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Myotonic Dystrophy Clinical Research Network","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2020","type":"Collaboration","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Myotonic Dystrophy Clinical Research Network","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Myotonic Dystrophy Clinical Research Network"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Adage Capital Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Private Placement","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0.40000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.40000000000000002,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Adage Capital Partners","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Adage Capital Partners"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Cowen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Public Offering","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0.20999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20999999999999999,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Cowen","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Cowen"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2021","type":"Inapplicable","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2021","type":"Inapplicable","leadProduct":"Del-Desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"Del-desiran","moa":"Transferrin-1 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Acceleron Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Luspatercept","moa":"Transforming growth factor beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Acceleron Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Acceleron Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Acceleron Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Bristol Myers Squibb","sponsor":"Acceleron Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Luspatercept","moa":"Transforming growth factor beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bristol Myers Squibb","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bristol Myers Squibb \/ Acceleron Pharma","highestDevelopmentStatusID":"8","companyTruncated":"Bristol Myers Squibb \/ Acceleron Pharma"},{"orgOrder":0,"company":"Bristol Myers Squibb","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Luspatercept","moa":"Transforming growth factor beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bristol Myers Squibb","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bristol Myers Squibb \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bristol Myers Squibb \/ Undisclosed"},{"orgOrder":0,"company":"Bristol Myers Squibb","sponsor":"Acceleron Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Luspatercept","moa":"Transforming growth factor beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bristol Myers Squibb","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bristol Myers Squibb \/ Acceleron Pharma","highestDevelopmentStatusID":"8","companyTruncated":"Bristol Myers Squibb \/ Acceleron Pharma"},{"orgOrder":0,"company":"Baylor College of Medicine","sponsor":"Sanofi | Oregon Health and Science University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2017","type":"Inapplicable","leadProduct":"Fresolimumab","moa":"Transforming growth factor beta-1 | Transforming growth factor beta-3 | Transforming growth factor beta-2","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Baylor College of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baylor College of Medicine \/ Sanofi | Oregon Health and Science University","highestDevelopmentStatusID":"6","companyTruncated":"Baylor College of Medicine \/ Sanofi | Oregon Health and Science University"},{"orgOrder":0,"company":"Eidos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Acoramidis Hydrochloride","moa":"Transthyretin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eidos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eidos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Eidos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Eidos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Acoramidis Hydrochloride","moa":"Transthyretin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eidos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eidos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Eidos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Eidos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Acoramidis Hydrochloride","moa":"Transthyretin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eidos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eidos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Eidos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Eidos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Acoramidis Hydrochloride","moa":"Transthyretin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eidos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eidos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Eidos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Eidos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Acoramidis Hydrochloride","moa":"Transthyretin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eidos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eidos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Eidos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Brigham and Women's Hospital","sponsor":"GSK | Ionis Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"Inotersen","moa":"Transthyretin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Brigham and Women's Hospital","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Brigham and Women's Hospital \/ GSK | Ionis Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Brigham and Women's Hospital \/ GSK | Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"Inotersen","moa":"Transthyretin","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"Inotersen","moa":"Transthyretin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2012","type":"Inapplicable","leadProduct":"Inotersen","moa":"Transthyretin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Neurimmune","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"NI006","moa":"Transthyretin (TTR)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Neurimmune","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurimmune \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Neurimmune \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NTLA-2001","moa":"Transthyretin (TTR)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2010","type":"Inapplicable","leadProduct":"ALN-TTR01","moa":"Transthyretin messenger RNA (TTR mRNA)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2012","type":"Inapplicable","leadProduct":"ALN-TTR02","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2016","type":"Inapplicable","leadProduct":"ALN-TTRSC02","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Eplontersen","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase I","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Eplontersen","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Eplontersen","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AstraZeneca \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Eplontersen","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Eplontersen","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Eplontersen","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Eplontersen","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Eplontersen","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Eplontersen","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Expanded Collaboration","leadProduct":"Eplontersen","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":3.7999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":3.7999999999999998,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Eplontersen","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"ION-682884","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"American Porphyria Foundation","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Funding","leadProduct":"Patisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"American Porphyria Foundation","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"American Porphyria Foundation \/ Alnylam Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"American Porphyria Foundation \/ Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2013","type":"Inapplicable","leadProduct":"Patisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Patisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Patisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2012","type":"Inapplicable","leadProduct":"Patisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2013","type":"Inapplicable","leadProduct":"Patisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"Patisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2016","type":"Inapplicable","leadProduct":"Patisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Taiba Healthcare","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Agreement","leadProduct":"Patisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alnylam Pharmaceuticals \/ Taiba Healthcare","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Taiba Healthcare"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Patisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"Patisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Gen Pharmaceuticals and Health Products","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Agreement","leadProduct":"Patisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alnylam Pharmaceuticals \/ Gen Pharmaceuticals and Health Products","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Gen Pharmaceuticals and Health Products"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"Revusiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2013","type":"Inapplicable","leadProduct":"Revusiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2013","type":"Inapplicable","leadProduct":"Revusiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2014","type":"Inapplicable","leadProduct":"Revusiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Vutrisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Vutrisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"Vutrisiran","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Partnership","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Orsini Specialty Pharmacy \/ Alnylam Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Vutrisiran Sodium","moa":"Transthyretin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vigil Neuroscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"VGL101","moa":"TREM2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vigil Neuroscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vigil Neuroscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vigil Neuroscience \/ Undisclosed"},{"orgOrder":0,"company":"Vigil Neuroscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"VGL101","moa":"TREM2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vigil Neuroscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vigil Neuroscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vigil Neuroscience \/ Undisclosed"},{"orgOrder":0,"company":"Amsterdam Molecular Therapeutics","sponsor":"International Antiviral Therapy Evaluation Center","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2010","type":"Inapplicable","leadProduct":"Alipogene Tiparvovec","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Amsterdam Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Amsterdam Molecular Therapeutics \/ International Antiviral Therapy Evaluation Center","highestDevelopmentStatusID":"9","companyTruncated":"Amsterdam Molecular Therapeutics \/ International Antiviral Therapy Evaluation Center"},{"orgOrder":0,"company":"Uniqure","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2016","type":"Inapplicable","leadProduct":"Alipogene Tiparvovec","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Chiesi Group","highestDevelopmentStatusID":"8","companyTruncated":"Uniqure \/ Chiesi Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Fatty acid oxidation||Triglyceride","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Fatty acid oxidation||Triglyceride","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Fatty acid oxidation||Triglyceride","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Fatty acid oxidation||Triglyceride","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Rigshospitalet","sponsor":"Groupe Hospitalier Pitie-Salpetriere | UT Southwestern Medical Center | Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rigshospitalet","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oil","sponsorNew":"Rigshospitalet \/ Groupe Hospitalier Pitie-Salpetriere | UT Southwestern Medical Center | Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Rigshospitalet \/ Groupe Hospitalier Pitie-Salpetriere | UT Southwestern Medical Center | Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Rigshospitalet","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rigshospitalet","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oil","sponsorNew":"Rigshospitalet \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Rigshospitalet \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Inserm","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inserm","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oil","sponsorNew":"Inserm \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Inserm \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"University of British Columbia","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of British Columbia","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oil","sponsorNew":"University of British Columbia \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"University of British Columbia \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Partnership","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orsini Specialty Pharmacy \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Jerry Vockley","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Jerry Vockley","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jerry Vockley \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Jerry Vockley \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"ConSynance Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"CSTI-500","moa":"Triple monoamine reuptake","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ConSynance Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"ConSynance Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ConSynance Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ConSynance Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"CSTI-500","moa":"Triple monoamine reuptake","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ConSynance Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"ConSynance Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ConSynance Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ConSynance Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"CSTI-500","moa":"Triple monoamine reuptake","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ConSynance Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"ConSynance Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ConSynance Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Actio Biosciences","sponsor":"Saniona","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"ABS-0871","moa":"TRPV4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Actio Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Actio Biosciences \/ Saniona","highestDevelopmentStatusID":"6","companyTruncated":"Actio Biosciences \/ Saniona"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Bayer AG","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Licensing Agreement","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Bayer AG","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Bayer AG"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Public Offering","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0.25,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.25,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ J.P. Morgan","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ J.P. Morgan"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Blue Owl Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Financing","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0.5,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.5,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Blue Owl Capital","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Blue Owl Capital"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Acoramidis","moa":"TTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Eidos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Acoramidis HCl","moa":"TTR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Eidos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eidos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Eidos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"YolTech Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"YOLT-201","moa":"TTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"YolTech Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"YolTech Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"YolTech Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Nexiguran Ziclumeran","moa":"TTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aurobindo Pharma Limited","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Plant Extract\/Herbal","year":"2022","type":"Inapplicable","leadProduct":"Colchicine","moa":"Tubulin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Aurobindo Pharma Limited","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Aurobindo Pharma Limited \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Aurobindo Pharma Limited \/ Undisclosed"},{"orgOrder":0,"company":"Mutual Pharmaceutical Company, Inc.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Plant Extract\/Herbal","year":"2010","type":"Inapplicable","leadProduct":"Colchicine","moa":"Tubulin","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Mutual Pharmaceutical Company, Inc.","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Mutual Pharmaceutical Company, Inc. \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Mutual Pharmaceutical Company, Inc. \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2015","type":"Inapplicable","leadProduct":"Denosumab","moa":"Tumor necrosis factor ligand superfamily member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Denosumab","moa":"Tumor necrosis factor ligand superfamily member 11","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Suzanne Lentzsch, MD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Teclistamab","moa":"Tumor necrosis factor receptor superfamily member 17 | T cell surface glycoprotein CD3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Suzanne Lentzsch, MD","highestDevelopmentStatusID":"8","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Suzanne Lentzsch, MD"},{"orgOrder":0,"company":"Stichting European Myeloma Network","sponsor":"GSK","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Antibody-drug Conjugate","year":"2020","type":"Inapplicable","leadProduct":"Belantamab Mafodotin","moa":"Tumor necrosis factor receptor superfamily member 17 | Tubulin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Stichting European Myeloma Network","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Stichting European Myeloma Network \/ GSK","highestDevelopmentStatusID":"8","companyTruncated":"Stichting European Myeloma Network \/ GSK"},{"orgOrder":0,"company":"UT Southwestern Medical Center","sponsor":"GSK","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2021","type":"Inapplicable","leadProduct":"Belantamab Mafodotin","moa":"Tumor necrosis factor receptor superfamily member 17 | Tubulin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"UT Southwestern Medical Center","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"UT Southwestern Medical Center \/ GSK","highestDevelopmentStatusID":"7","companyTruncated":"UT Southwestern Medical Center \/ GSK"},{"orgOrder":0,"company":"Bausch Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Methyltyrosine","moa":"Tyrosine 3-hydroxylase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bausch Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Bausch Health \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bausch Health \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Rilzabrutinib","moa":"Tyrosine-protein kinase BTK","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"St. Jude Children","sponsor":"Incyte Corporation | North American Consortium for Histiocytosis | Cures Within Reach","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ruxolitinib Phosphate","moa":"Tyrosine-protein kinase JAK1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"St. Jude Children","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"St. Jude Children \/ Incyte Corporation | North American Consortium for Histiocytosis | Cures Within Reach","highestDevelopmentStatusID":"7","companyTruncated":"St. Jude Children \/ Incyte Corporation | North American Consortium for Histiocytosis | Cures Within Reach"},{"orgOrder":0,"company":"Assistance Publique \u2013 H\u00f4pitaux de Paris","sponsor":"Incyte Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Itacitinib","moa":"Tyrosine-protein kinase JAK1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Assistance Publique \u2013 H\u00f4pitaux de Paris","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Incyte Corporation","highestDevelopmentStatusID":"8","companyTruncated":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Incyte Corporation"},{"orgOrder":0,"company":"Washington University School of Medicine","sponsor":"Incyte Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Ruxolitinib Phosphate","moa":"Tyrosine-protein kinase JAK1","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Washington University School of Medicine","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Washington University School of Medicine \/ Incyte Corporation","highestDevelopmentStatusID":"1","companyTruncated":"Washington University School of Medicine \/ Incyte Corporation"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Bosutinib","moa":"Tyrosine-protein kinase SRC | Tyrosine-protein kinase ABL | Tyrosine-protein kinase Lyn | Tyrosine-protein kinase HCK","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Rigel Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Fostamatinib Disodium Hexahydrate","moa":"Tyrosine-protein kinase SYK","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Rigel Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Rigel Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Rigel Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Grace Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2022","type":"Inapplicable","leadProduct":"GTX-102","moa":"UBE3A-AS","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Grace Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"Grace Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Grace Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Azidus","sponsor":"Sichuan Yuanda Shuyang Pharmaceutical Co., Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BRAZIL","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Immunoglobulins||Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Azidus","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Azidus \/ Sichuan Yuanda Shuyang Pharmaceutical Co., Ltd.","highestDevelopmentStatusID":"10","companyTruncated":"Azidus \/ Sichuan Yuanda Shuyang Pharmaceutical Co., Ltd."},{"orgOrder":0,"company":"Azidus","sponsor":"Cellavita","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BRAZIL","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Human Dental Pulp Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Azidus","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Azidus \/ Cellavita","highestDevelopmentStatusID":"10","companyTruncated":"Azidus \/ Cellavita"},{"orgOrder":0,"company":"Azidus","sponsor":"Cellavita","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BRAZIL","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Mesenchymal Stem Cell","moa":"Stem cell proliferation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Azidus","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Azidus \/ Cellavita","highestDevelopmentStatusID":"10","companyTruncated":"Azidus \/ Cellavita"},{"orgOrder":0,"company":"Azidus","sponsor":"Cellavita","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BRAZIL","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"Mesenchymal Stem Cell","moa":"Stem cell proliferation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Azidus","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Azidus \/ Cellavita","highestDevelopmentStatusID":"10","companyTruncated":"Azidus \/ Cellavita"},{"orgOrder":0,"company":"Azidus","sponsor":"Cellavita | Azidus Brazil","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"BRAZIL","productType":"Cell & Gene Therapy","year":"2016","type":"Inapplicable","leadProduct":"Mesenchymal Stem Cell","moa":"Stem cell proliferation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Azidus","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Azidus \/ Cellavita | Azidus Brazil","highestDevelopmentStatusID":"10","companyTruncated":"Azidus \/ Cellavita | Azidus Brazil"},{"orgOrder":0,"company":"Shanghai BDgene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"BD211","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shanghai BDgene","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Shanghai BDgene \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Shanghai BDgene \/ Undisclosed"},{"orgOrder":0,"company":"Shanghai BDgene","sponsor":"Shanghai Children's Medical Center","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"BD211","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shanghai BDgene","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Shanghai BDgene \/ Shanghai Children's Medical Center","highestDevelopmentStatusID":"6","companyTruncated":"Shanghai BDgene \/ Shanghai Children's Medical Center"},{"orgOrder":0,"company":"Shanghai Vitalgen BioPharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"VGB-R04","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Shanghai Vitalgen BioPharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Shanghai Vitalgen BioPharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Shanghai Vitalgen BioPharma \/ Undisclosed"},{"orgOrder":0,"company":"Shanghai Vitalgen BioPharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"VGM-R02b","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shanghai Vitalgen BioPharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Shanghai Vitalgen BioPharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Shanghai Vitalgen BioPharma \/ Undisclosed"},{"orgOrder":0,"company":"Sinocelltech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Recombinant Human Coagulation FVIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sinocelltech","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sinocelltech \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Sinocelltech \/ Undisclosed"},{"orgOrder":0,"company":"Sinocelltech","sponsor":"Chinese Academy of Medical Sciences | Parexel | Q2 Solutions","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Recombinant Human Coagulation FVIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sinocelltech","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sinocelltech \/ Chinese Academy of Medical Sciences | Parexel | Q2 Solutions","highestDevelopmentStatusID":"11","companyTruncated":"Sinocelltech \/ Chinese Academy of Medical Sciences | Parexel | Q2 Solutions"},{"orgOrder":0,"company":"Sinocelltech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Recombinant Human Coagulation FVIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sinocelltech","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sinocelltech \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Sinocelltech \/ Undisclosed"},{"orgOrder":0,"company":"Thiogenesis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"TTI-0102","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Thiogenesis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Thiogenesis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Thiogenesis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Attralus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Radiolabeled Compound","year":"2022","type":"Inapplicable","leadProduct":"124-I AT03","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Attralus \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Attralus \/ Undisclosed"},{"orgOrder":0,"company":"University of Alberta","sponsor":"Alberta Health services","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Radiolabeled Compound","year":"2017","type":"Inapplicable","leadProduct":"18-F DOPA","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"University of Alberta","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"University of Alberta \/ Alberta Health services","highestDevelopmentStatusID":"10","companyTruncated":"University of Alberta \/ Alberta Health services"},{"orgOrder":0,"company":"Cook Children's Health Care System","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Radiolabeled Compound","year":"2013","type":"Inapplicable","leadProduct":"18-F Fluorodopa","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Cook Children's Health Care System","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Cook Children's Health Care System \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Cook Children's Health Care System \/ Undisclosed"},{"orgOrder":0,"company":"University of Tennessee Graduate School of Medicine","sponsor":"Attralus","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Radiolabeled Compound","year":"2023","type":"Inapplicable","leadProduct":"99m-Tc p5+14","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"University of Tennessee Graduate School of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Tennessee Graduate School of Medicine \/ Attralus","highestDevelopmentStatusID":"6","companyTruncated":"University of Tennessee Graduate School of Medicine \/ Attralus"},{"orgOrder":0,"company":"Genethon","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"AAV1-Gamma-Sarcoglycan Vector","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genethon \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Undisclosed"},{"orgOrder":0,"company":"eyeDNA Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"AAV2\/5-hPDE6B","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"eyeDNA Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"eyeDNA Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"eyeDNA Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"AAV5-hRKp.RPGR","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"AAV5-hRKp.RPGR","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Syne Qua Non Limited | Bionical Emas","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"AAV5-RPGR","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"MeiraGTx \/ Syne Qua Non Limited | Bionical Emas","highestDevelopmentStatusID":"7","companyTruncated":"MeiraGTx \/ Syne Qua Non Limited | Bionical Emas"},{"orgOrder":0,"company":"VegaVect, Inc.","sponsor":"National Eye Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"AAV8-scRS","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"VegaVect, Inc.","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"VegaVect, Inc. \/ National Eye Institute","highestDevelopmentStatusID":"7","companyTruncated":"VegaVect, Inc. \/ National Eye Institute"},{"orgOrder":0,"company":"AskBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"AB-1001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AskBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Undisclosed"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ABO-504","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Abeona Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Abeona Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ascidian Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"ACDN-01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ascidian Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ascidian Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ascidian Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ascidian Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"ACDN-01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ascidian Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ascidian Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ascidian Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ace Cells Lab","sponsor":"European Wellness Academy","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2025","type":"Inapplicable","leadProduct":"Ace Retino","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ace Cells Lab","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ace Cells Lab \/ European Wellness Academy","highestDevelopmentStatusID":"7","companyTruncated":"Ace Cells Lab \/ European Wellness Academy"},{"orgOrder":0,"company":"AC Immune","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Vaccine","year":"2024","type":"Inapplicable","leadProduct":"ACI-24.060","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AC Immune","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AC Immune \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AC Immune \/ Undisclosed"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ACTX-401","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alcyone Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alcyone Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Alcyone Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Angitia Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"AGA2115","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Angitia Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Angitia Biopharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Angitia Biopharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Angitia Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"AGA2115","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Angitia Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Angitia Biopharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Angitia Biopharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Airna","sponsor":"Venrock Healthcare Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Series B Financing","leadProduct":"AIR-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Airna","amount2":0.16,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Undisclosed","sponsorNew":"Airna \/ Venrock Healthcare Capital","highestDevelopmentStatusID":"4","companyTruncated":"Airna \/ Venrock Healthcare Capital"},{"orgOrder":0,"company":"Akeso","sponsor":"AD Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"AK102","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Akeso","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Akeso \/ AD Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Akeso \/ AD Pharmaceuticals"},{"orgOrder":0,"company":"AceLink Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"AL00804","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"AceLink Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AceLink Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"AceLink Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AlgiPharma ","sponsor":"Eurostars","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NORWAY","productType":"Carbohydrate","year":"2014","type":"Inapplicable","leadProduct":"Alginate Oligosaccharide","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AlgiPharma ","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"AlgiPharma \/ Eurostars","highestDevelopmentStatusID":"9","companyTruncated":"AlgiPharma \/ Eurostars"},{"orgOrder":0,"company":"AlgiPharma ","sponsor":"Imperial College London","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NORWAY","productType":"Carbohydrate","year":"2018","type":"Inapplicable","leadProduct":"Alginate Oligosaccharide","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AlgiPharma ","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"AlgiPharma \/ Imperial College London","highestDevelopmentStatusID":"9","companyTruncated":"AlgiPharma \/ Imperial College London"},{"orgOrder":0,"company":"AlgiPharma ","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NORWAY","productType":"Carbohydrate","year":"2015","type":"Inapplicable","leadProduct":"Alginate Oligosaccharide","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AlgiPharma ","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"AlgiPharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AlgiPharma \/ Undisclosed"},{"orgOrder":0,"company":"AlgiPharma ","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NORWAY","productType":"Carbohydrate","year":"2019","type":"Inapplicable","leadProduct":"Alginate Oligosaccharide","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AlgiPharma ","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"AlgiPharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AlgiPharma \/ Undisclosed"},{"orgOrder":0,"company":"AlgiPharma ","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NORWAY","productType":"Carbohydrate","year":"2011","type":"Inapplicable","leadProduct":"Alginate Oligosaccharide","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AlgiPharma ","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AlgiPharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AlgiPharma \/ Undisclosed"},{"orgOrder":0,"company":"Steminent US, Inc.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Allogeneic Adipose-Derived Mesenchymal Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Steminent US, Inc.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Steminent US, Inc. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Steminent US, Inc. \/ Undisclosed"},{"orgOrder":0,"company":"Capricor","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Allogeneic Cardiosphere-Derived Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Capricor","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Capricor \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Capricor \/ Undisclosed"},{"orgOrder":0,"company":"Capricor","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2015","type":"Inapplicable","leadProduct":"Allogeneic Cardiosphere-Derived Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Capricor","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Capricor \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Capricor \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"ALN-TTRSC04","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Avalo Therapeutics","sponsor":"AUG Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2023","type":"Divestment","leadProduct":"Alpha-D-Galactose","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avalo Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Avalo Therapeutics \/ AUG Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Avalo Therapeutics \/ AUG Therapeutics"},{"orgOrder":0,"company":"CDG Care","sponsor":"Cerecor","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2020","type":"Collaboration","leadProduct":"Alpha-D-Galactose","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CDG Care","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CDG Care \/ Cerecor","highestDevelopmentStatusID":"8","companyTruncated":"CDG Care \/ Cerecor"},{"orgOrder":0,"company":"University Health Network, Toronto","sponsor":"Ozmosis Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Cell & Gene Therapy","year":"2016","type":"Inapplicable","leadProduct":"Alpha-Galactosidase A Transduced Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"University Health Network, Toronto","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University Health Network, Toronto \/ Ozmosis Research","highestDevelopmentStatusID":"6","companyTruncated":"University Health Network, Toronto \/ Ozmosis Research"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"Alpha-PI","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Amarna Therapeutics","sponsor":"C4 holding BV","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2021","type":"Financing","leadProduct":"AMA005","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Amarna Therapeutics","amount2":0.01,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"Amarna Therapeutics \/ C4 holding BV","highestDevelopmentStatusID":"5","companyTruncated":"Amarna Therapeutics \/ C4 holding BV"},{"orgOrder":0,"company":"Amplo Biotechnology","sponsor":"NIH-NIAMS","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Funding","leadProduct":"AMP-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Amplo Biotechnology","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amplo Biotechnology \/ NIH-NIAMS","highestDevelopmentStatusID":"4","companyTruncated":"Amplo Biotechnology \/ NIH-NIAMS"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"AMT-191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AMT-191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Biocad","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"ANB-004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Biocad","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Biocad \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Biocad \/ Undisclosed"},{"orgOrder":0,"company":"Biocad","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"ANB-010","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Biocad","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Biocad \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Biocad \/ Undisclosed"},{"orgOrder":0,"company":"Kind Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"AND017","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Kind Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kind Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Kind Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Atum Bio","sponsor":"Anagram Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Expanded Collaboration","leadProduct":"ANG003","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Atum Bio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Atum Bio \/ Anagram Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Atum Bio \/ Anagram Therapeutics"},{"orgOrder":0,"company":"Nektar Therapeutics","sponsor":"HealthCare Royalty","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Agreement","leadProduct":"Antihemophilic Factor Recombinant, PEGylated","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Nektar Therapeutics","amount2":0.14999999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14999999999999999,"dosageForm":"Injection","sponsorNew":"Nektar Therapeutics \/ HealthCare Royalty","highestDevelopmentStatusID":"15","companyTruncated":"Nektar Therapeutics \/ HealthCare Royalty"},{"orgOrder":0,"company":"ALPS Biotech CO. LTD","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Antrodia Cinnamomea","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"ALPS Biotech CO. LTD","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ALPS Biotech CO. LTD \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"ALPS Biotech CO. LTD \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"AOC-1020","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"AOC-1020","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"AOC-1020","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Alebund Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"AP303","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Alebund Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Alebund Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Alebund Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2020","type":"Inapplicable","leadProduct":"AP-PA02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Armata Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2023","type":"Inapplicable","leadProduct":"AP-PA02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Armata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2020","type":"Funding","leadProduct":"AP-PA02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Inhalation","sponsorNew":"Armata Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Innoviva","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2020","type":"Financing","leadProduct":"AP-PA02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Inhalation","sponsorNew":"Armata Pharmaceuticals \/ Innoviva","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Innoviva"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Innoviva","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2020","type":"Agreement","leadProduct":"AP-PA02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Inhalation","sponsorNew":"Armata Pharmaceuticals \/ Innoviva","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Innoviva"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Innoviva","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2020","type":"Financing","leadProduct":"AP-PA02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Inhalation","sponsorNew":"Armata Pharmaceuticals \/ Innoviva","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Innoviva"},{"orgOrder":0,"company":"APR Applied Pharma Research","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"APR-OD031","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"APR Applied Pharma Research","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"APR Applied Pharma Research \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"APR Applied Pharma Research \/ Undisclosed"},{"orgOrder":0,"company":"Kobe University","sponsor":"Japan Medical Association | Nobelpharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"Arbekacin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Kobe University","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kobe University \/ Japan Medical Association | Nobelpharma","highestDevelopmentStatusID":"8","companyTruncated":"Kobe University \/ Japan Medical Association | Nobelpharma"},{"orgOrder":0,"company":"Argenx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"ARGX-119","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Argenx","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Argenx \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Argenx \/ Undisclosed"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Sickle Cell Disease Association Of America","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Partnership","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Sickle Cell Disease Association Of America","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Sickle Cell Disease Association Of America"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Lonza Group","sponsor":"Aruvant Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2021","type":"Agreement","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lonza Group","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lonza Group \/ Aruvant Sciences","highestDevelopmentStatusID":"7","companyTruncated":"Lonza Group \/ Aruvant Sciences"},{"orgOrder":0,"company":"Aldevron","sponsor":"Aruvant Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Partnership","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aldevron","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aldevron \/ Aruvant Sciences","highestDevelopmentStatusID":"7","companyTruncated":"Aldevron \/ Aruvant Sciences"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"ASP2016","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Astellas Pharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Astellas Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ASP2957","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Astellas Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Astellas Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Attralus","sponsor":"Novotech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"AT02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Attralus \/ Novotech","highestDevelopmentStatusID":"8","companyTruncated":"Attralus \/ Novotech"},{"orgOrder":0,"company":"Attralus","sponsor":"Novotech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"AT-02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Attralus \/ Novotech","highestDevelopmentStatusID":"8","companyTruncated":"Attralus \/ Novotech"},{"orgOrder":0,"company":"Attralus","sponsor":"Alpha Wave Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Financing","leadProduct":"AT-02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Attralus","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Infusion","sponsorNew":"Attralus \/ Alpha Wave Ventures","highestDevelopmentStatusID":"8","companyTruncated":"Attralus \/ Alpha Wave Ventures"},{"orgOrder":0,"company":"Attralus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"AT-02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Attralus \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Attralus \/ Undisclosed"},{"orgOrder":0,"company":"Attralus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"AT-02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Attralus \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Attralus \/ Undisclosed"},{"orgOrder":0,"company":"Attralus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"AT-02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Attralus \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Attralus \/ Undisclosed"},{"orgOrder":0,"company":"Attralus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"AT-02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Attralus \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Attralus \/ Undisclosed"},{"orgOrder":0,"company":"Attralus","sponsor":"Venbio Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Series A Financing","leadProduct":"AT-02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Attralus","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Infusion","sponsorNew":"Attralus \/ Venbio Partners","highestDevelopmentStatusID":"6","companyTruncated":"Attralus \/ Venbio Partners"},{"orgOrder":0,"company":"Attralus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"AT-02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Attralus \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Attralus \/ Undisclosed"},{"orgOrder":0,"company":"Attralus","sponsor":"University of Tennessee Graduate School of Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-peptide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"AT-04","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Attralus \/ University of Tennessee Graduate School of Medicine","highestDevelopmentStatusID":"4","companyTruncated":"Attralus \/ University of Tennessee Graduate School of Medicine"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATA-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Atamyo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"ATA-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Atamyo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Atamyo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"The Dion Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Partnership","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ The Dion Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ The Dion Foundation"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Bpifrance","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Financing","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Bpifrance","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Bpifrance"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Bain Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Series C Financing","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14999999999999999,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Bain Capital","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Bain Capital"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Red de Terapia Celular","sponsor":"Spanish National Health System | Hospital Universitario Virgen de la Arrixaca | Fundacion para la Formacion e Investigacion Sanitarias | Public Health Service, Murcia | Instituto Murciano de Investigaci\u00f3n Biosanitaria Virgen de la Arrixaca","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell & Gene Therapy","year":"2014","type":"Inapplicable","leadProduct":"Autologous Bone Marrow Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Red de Terapia Celular","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Red de Terapia Celular \/ Spanish National Health System | Hospital Universitario Virgen de la Arrixaca | Fundacion para la Formacion e Investigacion Sanitarias | Public Health Service, Murcia | Instituto Murciano de Investigaci\u00f3n Biosanitaria Virgen de la Arrixaca","highestDevelopmentStatusID":"6","companyTruncated":"Red de Terapia Celular \/ Spanish National Health System | Hospital Universitario Virgen de la Arrixaca | Fundacion para la Formacion e Investigacion Sanitarias | Public Health Service, Murcia | Instituto Murciano de Investigaci\u00f3n Biosanitaria Virgen de la Arrixaca"},{"orgOrder":0,"company":"University of Manchester","sponsor":"Orchard Therapeutics | CTI Clinical Trial and Consulting | University College, London | Great Ormond Street Hospital for Children NHS Foundation Trust | Manchester University NHS FT","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"Autologous CD34+ Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"University of Manchester","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Manchester \/ Orchard Therapeutics | CTI Clinical Trial and Consulting | University College, London | Great Ormond Street Hospital for Children NHS Foundation Trust | Manchester University NHS FT","highestDevelopmentStatusID":"7","companyTruncated":"University of Manchester \/ Orchard Therapeutics | CTI Clinical Trial and Consulting | University College, London | Great Ormond Street Hospital for Children NHS Foundation Trust | Manchester University NHS FT"},{"orgOrder":0,"company":"University of Manchester","sponsor":"CTI Clinical Trial and Consulting | Great Ormond Street Hospital for Children NHS Foundation Trust | Manchester University NHS FT | LifeArc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Autologous CD34+ Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"University of Manchester","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"University of Manchester \/ CTI Clinical Trial and Consulting | Great Ormond Street Hospital for Children NHS Foundation Trust | Manchester University NHS FT | LifeArc","highestDevelopmentStatusID":"7","companyTruncated":"University of Manchester \/ CTI Clinical Trial and Consulting | Great Ormond Street Hospital for Children NHS Foundation Trust | Manchester University NHS FT | LifeArc"},{"orgOrder":0,"company":"Immusoft Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Autologous Plasmablast","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Immusoft Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Immusoft Corporation \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Immusoft Corporation \/ Undisclosed"},{"orgOrder":0,"company":"AavantiBio","sponsor":"Solid Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Merger","leadProduct":"AVB-202","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AavantiBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AavantiBio \/ Solid Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"AavantiBio \/ Solid Biosciences"},{"orgOrder":0,"company":"AavantiBio","sponsor":"Solid Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Merger","leadProduct":"AVB-202","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AavantiBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AavantiBio \/ Solid Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"AavantiBio \/ Solid Biosciences"},{"orgOrder":0,"company":"AlveoGene","sponsor":"Oxford Science Enterprises","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Financing","leadProduct":"AVG-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AlveoGene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"AlveoGene \/ Oxford Science Enterprises","highestDevelopmentStatusID":"4","companyTruncated":"AlveoGene \/ Oxford Science Enterprises"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AVR\u2011RD\u201104","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-06","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Allievex","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"AX250","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Allievex","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Allievex \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Allievex \/ Undisclosed"},{"orgOrder":0,"company":"Allievex","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"AX250","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Allievex","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Allievex \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Allievex \/ Undisclosed"},{"orgOrder":0,"company":"Allievex","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"AX250","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Allievex","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Allievex \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Allievex \/ Undisclosed"},{"orgOrder":0,"company":"Axcella Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Dietary Supplement","year":"2019","type":"Inapplicable","leadProduct":"AXA4010","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Axcella Health","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Axcella Health \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Axcella Health \/ Undisclosed"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"Azli","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Gilead Sciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Gilead Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Baxalta Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"BAX 802","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Baxalta Pharmaceutical | Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"BAX 888","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical | Takeda Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical | Takeda Pharmaceutical"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"bb1111","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Shanghai Jiaotong University School of Medicine","sponsor":"Shanghai Mianyi Biopharmaceutical Co., Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"BBM-D101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shanghai Jiaotong University School of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Shanghai Jiaotong University School of Medicine \/ Shanghai Mianyi Biopharmaceutical Co., Ltd.","highestDevelopmentStatusID":"6","companyTruncated":"Shanghai Jiaotong University School of Medicine \/ Shanghai Mianyi Biopharmaceutical Co., Ltd."},{"orgOrder":0,"company":"Belief BioMed","sponsor":"Shanghai Mianyi Biopharmaceutical Co., Ltd. | Shanghai Xinzhi BioMed Co., Ltd","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"BBM-D101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Belief BioMed","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Belief BioMed \/ Shanghai Mianyi Biopharmaceutical Co., Ltd. | Shanghai Xinzhi BioMed Co., Ltd","highestDevelopmentStatusID":"7","companyTruncated":"Belief BioMed \/ Shanghai Mianyi Biopharmaceutical Co., Ltd. | Shanghai Xinzhi BioMed Co., Ltd"},{"orgOrder":0,"company":"Belief BioMed","sponsor":"Shanghai MYGT Biopharmaceutical LLC","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"BBM-H803","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Belief BioMed","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Belief BioMed \/ Shanghai MYGT Biopharmaceutical LLC","highestDevelopmentStatusID":"7","companyTruncated":"Belief BioMed \/ Shanghai MYGT Biopharmaceutical LLC"},{"orgOrder":0,"company":"Belief BioMed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BBM-H803","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Belief BioMed","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Belief BioMed \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Belief BioMed \/ Undisclosed"},{"orgOrder":0,"company":"Belief BioMed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"BBM-H901","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belief BioMed","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Belief BioMed \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belief BioMed \/ Undisclosed"},{"orgOrder":0,"company":"Belief BioMed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"BBM-H901","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Belief BioMed","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Belief BioMed \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Belief BioMed \/ Undisclosed"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BE-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Be Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Be Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BE-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Be Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Be Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"BE-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Be Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Be Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Arch Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Financing","leadProduct":"BE-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Be Biopharma","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"Be Biopharma \/ Arch Ventures","highestDevelopmentStatusID":"7","companyTruncated":"Be Biopharma \/ Arch Ventures"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Nextech","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Series C Financing","leadProduct":"BE-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Be Biopharma","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Infusion","sponsorNew":"Be Biopharma \/ Nextech","highestDevelopmentStatusID":"7","companyTruncated":"Be Biopharma \/ Nextech"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BE-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Be Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Be Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"BEAM-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BEAM-101","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"BEAM-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"BEAM-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"BEAM-302","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Western Sydney Local Health District","sponsor":"Westmead Institute for Medical Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Beetroot","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Western Sydney Local Health District","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Western Sydney Local Health District \/ Westmead Institute for Medical Research","highestDevelopmentStatusID":"1","companyTruncated":"Western Sydney Local Health District \/ Westmead Institute for Medical Research"},{"orgOrder":0,"company":"Oxford Biomedica","sponsor":"BlackfinBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Acquisition","leadProduct":"BFB-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oxford Biomedica","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oxford Biomedica \/ BlackfinBio","highestDevelopmentStatusID":"4","companyTruncated":"Oxford Biomedica \/ BlackfinBio"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"BI3720931","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"BI3720931","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"BMN 349","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Inapplicable","leadProduct":"BMN 390","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"BMN165","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Bioray Laboratories","sponsor":"Nanfang Hospital, Southern Medical University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BRL-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Bioray Laboratories","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bioray Laboratories \/ Nanfang Hospital, Southern Medical University","highestDevelopmentStatusID":"1","companyTruncated":"Bioray Laboratories \/ Nanfang Hospital, Southern Medical University"},{"orgOrder":0,"company":"Bioray Laboratories","sponsor":"First Affiliated Hospital of Guangxi Medical University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BRL-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Bioray Laboratories","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bioray Laboratories \/ First Affiliated Hospital of Guangxi Medical University","highestDevelopmentStatusID":"1","companyTruncated":"Bioray Laboratories \/ First Affiliated Hospital of Guangxi Medical University"},{"orgOrder":0,"company":"Bioray Laboratories","sponsor":"Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BRL-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Bioray Laboratories","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bioray Laboratories \/ Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College","highestDevelopmentStatusID":"1","companyTruncated":"Bioray Laboratories \/ Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College"},{"orgOrder":0,"company":"Bionic Sight","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BS01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bionic Sight","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bionic Sight \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bionic Sight \/ Undisclosed"},{"orgOrder":0,"company":"Bionic Sight","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"BS01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bionic Sight","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bionic Sight \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bionic Sight \/ Undisclosed"},{"orgOrder":0,"company":"Bionic Sight","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"BS01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bionic Sight","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bionic Sight \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bionic Sight \/ Undisclosed"},{"orgOrder":0,"company":"Bionic Sight","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BS01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bionic Sight","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bionic Sight \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bionic Sight \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"C1-esterase Inhibitor","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Caelum Biosciences","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Bortezomib","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Caelum Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Caelum Biosciences \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Caelum Biosciences \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Caelum Biosciences","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Bortezomib","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Caelum Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Caelum Biosciences \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Caelum Biosciences \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Caelum Biosciences","sponsor":"AstraZeneca","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Acquisition","leadProduct":"Bortezomib","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Caelum Biosciences","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.5,"dosageForm":"Infusion","sponsorNew":"Caelum Biosciences \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Caelum Biosciences \/ AstraZeneca"},{"orgOrder":0,"company":"Fortress Biotech","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Bortezomib","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fortress Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Fortress Biotech \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Fortress Biotech \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Caelum Biosciences","sponsor":"AstraZeneca","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Acquisition","leadProduct":"Bortezomib","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Caelum Biosciences","amount2":0.5,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.5,"dosageForm":"Infusion","sponsorNew":"Caelum Biosciences \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Caelum Biosciences \/ AstraZeneca"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"CAEL-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"CAEL-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"CAEL-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Abigail Wexner Research Institute","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"Calpain 3","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Abigail Wexner Research Institute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Abigail Wexner Research Institute \/ Sarepta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Abigail Wexner Research Institute \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Maastricht University Medical Center","sponsor":"DSM Food Specialties","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Canola Protein","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Maastricht University Medical Center","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Maastricht University Medical Center \/ DSM Food Specialties","highestDevelopmentStatusID":"1","companyTruncated":"Maastricht University Medical Center \/ DSM Food Specialties"},{"orgOrder":0,"company":"Children's Hospital of Fudan University","sponsor":"Hedu Biotechnology","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"CBT102-A","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Children's Hospital of Fudan University","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Children's Hospital of Fudan University \/ Hedu Biotechnology","highestDevelopmentStatusID":"1","companyTruncated":"Children's Hospital of Fudan University \/ Hedu Biotechnology"},{"orgOrder":0,"company":"National Institute of Allergy and Infectious Diseases","sponsor":"The University of Pittsburgh School of Medicine | PPD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"CD3-negative Allogeneic BMT Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"National Institute of Allergy and Infectious Diseases","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Institute of Allergy and Infectious Diseases \/ The University of Pittsburgh School of Medicine | PPD","highestDevelopmentStatusID":"7","companyTruncated":"National Institute of Allergy and Infectious Diseases \/ The University of Pittsburgh School of Medicine | PPD"},{"orgOrder":0,"company":"Codexis","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"CDX-6316","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Codexis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Codexis \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Codexis \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Celiprolol Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Acer Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Acer Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Celiprolol Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Acer Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Acer Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Celiprolol Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Acer Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Acer Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Celiprolol Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Acer Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Acer Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Arla Foods","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"CGMP Protein Substitute","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Arla Foods","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Arla Foods"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"CHF6333","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chiesi Group \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Chiesi Group \/ Undisclosed"},{"orgOrder":0,"company":"Cytokinetics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"CK-089","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Cytokinetics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cytokinetics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Cytokinetics \/ Undisclosed"},{"orgOrder":0,"company":"Chong Kun Dang Pharm","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"CKD-504","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Chong Kun Dang Pharm","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chong Kun Dang Pharm \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Chong Kun Dang Pharm \/ Undisclosed"},{"orgOrder":0,"company":"Cellatoz Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"CLZ-2002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Cellatoz Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Cellatoz Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Cellatoz Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Cystetic Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"CM001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Cystetic Medicines","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"Cystetic Medicines \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Cystetic Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Connoya Biomedical Technology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"CM336","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Connoya Biomedical Technology","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Connoya Biomedical Technology \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Connoya Biomedical Technology \/ Undisclosed"},{"orgOrder":0,"company":"Institute of Hematology & Blood Diseases Hospital, China","sponsor":"Connoya Biomedical Technology","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"CM-336","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Institute of Hematology & Blood Diseases Hospital, China","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Institute of Hematology & Blood Diseases Hospital, China \/ Keymed Biosciences","highestDevelopmentStatusID":"1","companyTruncated":"Institute of Hematology & Blood Diseases Hospital, China \/ Keymed Biosciences"},{"orgOrder":0,"company":"CAMP4 Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"CMP-CPS-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"CAMP4 Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CAMP4 Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"CAMP4 Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"CAMP4 Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"CMP-CPS-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"CAMP4 Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CAMP4 Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"CAMP4 Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"CAMP4 Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"CMP-CPS-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"CAMP4 Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CAMP4 Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"CAMP4 Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"CAMP4 Therapeutics","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Public Offering","leadProduct":"CMP-CPS-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"CAMP4 Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"CAMP4 Therapeutics \/ J.P. Morgan","highestDevelopmentStatusID":"6","companyTruncated":"CAMP4 Therapeutics \/ J.P. Morgan"},{"orgOrder":0,"company":"Hangzhou Zede Pharma-Tech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"CN128","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Hangzhou Zede Pharma-Tech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Hangzhou Zede Pharma-Tech \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Hangzhou Zede Pharma-Tech \/ Undisclosed"},{"orgOrder":0,"company":"Hangzhou Zede Pharma-Tech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"CN128","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Hangzhou Zede Pharma-Tech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Hangzhou Zede Pharma-Tech \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Hangzhou Zede Pharma-Tech \/ Undisclosed"},{"orgOrder":0,"company":"Hangzhou Zede Pharma-Tech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"CN128","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Hangzhou Zede Pharma-Tech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Hangzhou Zede Pharma-Tech \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Hangzhou Zede Pharma-Tech \/ Undisclosed"},{"orgOrder":0,"company":"Hangzhou Zede Pharma-Tech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"CN128","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Hangzhou Zede Pharma-Tech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Hangzhou Zede Pharma-Tech \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Hangzhou Zede Pharma-Tech \/ Undisclosed"},{"orgOrder":0,"company":"Gamida Cell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Plant Extract\/Herbal","year":"2015","type":"Inapplicable","leadProduct":"Cordin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Gamida Cell","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gamida Cell \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Gamida Cell \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Cotoretigene Toliparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"RSPR Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"CRD007","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"RSPR Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"RSPR Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"RSPR Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Cardurion Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"CRD-4730","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cardurion Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cardurion Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cardurion Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"CorrectSequence Therapeutics","sponsor":"First Affiliated Hospital of Guangxi Medical University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"CS-206","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"CorrectSequence Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"CorrectSequence Therapeutics \/ First Affiliated Hospital of Guangxi Medical University","highestDevelopmentStatusID":"6","companyTruncated":"CorrectSequence Therapeutics \/ First Affiliated Hospital of Guangxi Medical University"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"CSL200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"CSL222","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"CSL689","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Connecta therapeutics","sponsor":"Hospital del Mar Research Institute (IMIM) | Adknoma | Ministry of Science and Innovation, Spain","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"CTH120","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Connecta therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Connecta therapeutics \/ Hospital del Mar Research Institute (IMIM) | Adknoma | Ministry of Science and Innovation, Spain","highestDevelopmentStatusID":"6","companyTruncated":"Connecta therapeutics \/ Hospital del Mar Research Institute (IMIM) | Adknoma | Ministry of Science and Innovation, Spain"},{"orgOrder":0,"company":"Avrobio","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Divestment","leadProduct":"CTNS-RD-04","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Avrobio \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Ottawa Hospital Research Institute","sponsor":"Octapharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"Cutaquig","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Ottawa Hospital Research Institute","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ottawa Hospital Research Institute \/ Octapharma","highestDevelopmentStatusID":"11","companyTruncated":"Ottawa Hospital Research Institute \/ Octapharma"},{"orgOrder":0,"company":"Recordati Rare Diseases","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"Cysteamine Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Recordati Rare Diseases","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Recordati Rare Diseases \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Recordati Rare Diseases \/ Undisclosed"},{"orgOrder":0,"company":"Recordati Rare Diseases","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2020","type":"Inapplicable","leadProduct":"Cysteamine Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Recordati Rare Diseases","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Recordati Rare Diseases \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Recordati Rare Diseases \/ Undisclosed"},{"orgOrder":0,"company":"Leadiant Biosciences","sponsor":"AllianceRx Walgreens","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2022","type":"Partnership","leadProduct":"Cysteamine Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Leadiant Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Leadiant Biosciences \/ AllianceRx Walgreens","highestDevelopmentStatusID":"15","companyTruncated":"Leadiant Biosciences \/ AllianceRx Walgreens"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Inapplicable","leadProduct":"Delpacibart Braxlosiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"Delpacibart Braxlosiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"Delpacibart Braxlosiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2024","type":"Inapplicable","leadProduct":"Delpacibart Etedesiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"TD Cowen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2024","type":"Public Offering","leadProduct":"Delpacibart Etedesiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.46000000000000002,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ TD Cowen","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ TD Cowen"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2024","type":"Inapplicable","leadProduct":"Delpacibart Etedesiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2024","type":"Public Offering","leadProduct":"Delpacibart Etedesiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Leerink Partners","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Leerink Partners"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2024","type":"Public Offering","leadProduct":"Delpacibart Etedesiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0.25,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Leerink Partners","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Leerink Partners"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"TD Cowen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2024","type":"Public Offering","leadProduct":"Delpacibart Etedesiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0.46000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.46000000000000002,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ TD Cowen","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ TD Cowen"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"TD Cowen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2024","type":"Public Offering","leadProduct":"Delpacibart Etedesiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0.40000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.46000000000000002,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ TD Cowen","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ TD Cowen"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2025","type":"Inapplicable","leadProduct":"Delpacibart Etedesiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"SCF Pharma","sponsor":"Centre de recherche du Centre hospitalier universitaire de Sherbrooke | SOLUTEX GC SL","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"DHA Monoglyceride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SCF Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SCF Pharma \/ Centre de recherche du Centre hospitalier universitaire de Sherbrooke | SOLUTEX GC SL","highestDevelopmentStatusID":"8","companyTruncated":"SCF Pharma \/ Centre de recherche du Centre hospitalier universitaire de Sherbrooke | SOLUTEX GC SL"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"DNL126","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"DNL126","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"PXE International","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"DS-1211b","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Daiichi Sankyo \/ PXE International","highestDevelopmentStatusID":"8","companyTruncated":"Daiichi Sankyo \/ PXE International"},{"orgOrder":0,"company":"Sumitomo Pharma America","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"DSP-3077","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sumitomo Pharma America","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sumitomo Pharma America \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sumitomo Pharma America \/ Undisclosed"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"DT-216P2","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Design Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Design Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"DT-216P2","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Design Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Design Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"DTX201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Bayer AG \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Dystrogen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Dystrophin Expressing Chimeric Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Dystrogen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Dystrogen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Dystrogen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dystrogen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Dystrophin Expressing Chimeric Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Dystrogen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dystrogen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Dystrogen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ExCellThera","sponsor":"IRICoR","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Cell & Gene Therapy","year":"2020","type":"Funding","leadProduct":"ECT-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ExCellThera","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ExCellThera \/ IRICoR","highestDevelopmentStatusID":"4","companyTruncated":"ExCellThera \/ IRICoR"},{"orgOrder":0,"company":"ExCellThera","sponsor":"California Institute for Regenerative Medicine (CIRM)","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ECT-001-CB","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ExCellThera","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ExCellThera \/ California Institute for Regenerative Medicine (CIRM)","highestDevelopmentStatusID":"6","companyTruncated":"ExCellThera \/ California Institute for Regenerative Medicine (CIRM)"},{"orgOrder":0,"company":"iECURE","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ECUR-506","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"iECURE \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"iECURE \/ Undisclosed"},{"orgOrder":0,"company":"iECURE","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ECUR-506","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"iECURE \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"iECURE \/ Undisclosed"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ECUR-506","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Precision BioSciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Precision BioSciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Precision BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"iECURE","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ECUR-506","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"iECURE \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"iECURE \/ Undisclosed"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"iECURE","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ECUR-506","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Precision BioSciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Precision BioSciences \/ iECURE","highestDevelopmentStatusID":"7","companyTruncated":"Precision BioSciences \/ iECURE"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"EDK060","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Margaret Ragni","sponsor":"Health Resources and Services Administration","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Eloctate ITI","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Margaret Ragni","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Margaret Ragni \/ Health Resources and Services Administration","highestDevelopmentStatusID":"10","companyTruncated":"Margaret Ragni \/ Health Resources and Services Administration"},{"orgOrder":0,"company":"Emendo Biotherapeutics","sponsor":"AnGes","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Acquisition","leadProduct":"EMD-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Emendo Biotherapeutics","amount2":0.25,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.25,"dosageForm":"Undisclosed","sponsorNew":"Emendo Biotherapeutics \/ AnGes","highestDevelopmentStatusID":"4","companyTruncated":"Emendo Biotherapeutics \/ AnGes"},{"orgOrder":0,"company":"ENCell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EN001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ENCell","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ENCell \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ENCell \/ Undisclosed"},{"orgOrder":0,"company":"ENCell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"EN001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ENCell","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ENCell \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ENCell \/ Undisclosed"},{"orgOrder":0,"company":"ENCell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EN001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ENCell","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ENCell \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ENCell \/ Undisclosed"},{"orgOrder":0,"company":"ENCell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EN001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ENCell","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ENCell \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ENCell \/ Undisclosed"},{"orgOrder":0,"company":"ENCell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"EN001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ENCell","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ENCell \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ENCell \/ Undisclosed"},{"orgOrder":0,"company":"Entos Pharmaceuticals","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Funding","leadProduct":"ENTLEP001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Entos Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entos Pharmaceuticals \/ California Institute for Regenerative Medicine","highestDevelopmentStatusID":"5","companyTruncated":"Entos Pharmaceuticals \/ California Institute for Regenerative Medicine"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Collaboration","leadProduct":"ENTR-701","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Entrada Therapeutics","amount2":0.73999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.73999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Entrada Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Entrada Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Agreement","leadProduct":"ENTR-701","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Entrada Therapeutics","amount2":0.73999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.73999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Entrada Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Entrada Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"ExoRNA Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"ER2001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ExoRNA Bioscience","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ExoRNA Bioscience \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ExoRNA Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Expansion Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"ERX-963","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Expansion Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Expansion Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Expansion Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Expression Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ET3","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Expression Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Expression Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Expression Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"ETX001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Enterprise Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Enterprise Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"E-therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ETX-148","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"E-therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"E-therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"E-therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Forest Laboratories","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"EUR-1008","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Forest Laboratories","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Delayed Release","sponsorNew":"Forest Laboratories \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Forest Laboratories \/ Undisclosed"},{"orgOrder":0,"company":"Forest Laboratories","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"EUR-1008","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Forest Laboratories","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Delayed Release","sponsorNew":"Forest Laboratories \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Forest Laboratories \/ Undisclosed"},{"orgOrder":0,"company":"EV\u014cQ Nano","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"EVQ-218","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"EV\u014cQ Nano","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"EV\u014cQ Nano \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"EV\u014cQ Nano \/ Undisclosed"},{"orgOrder":0,"company":"Jiayin Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EXG001-307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jiayin Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Jiayin Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jiayin Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Jiayin Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"EXG001-307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jiayin Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Jiayin Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jiayin Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Jiayin Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EXG001-307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Jiayin Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Jiayin Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Jiayin Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Exegenesis Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"EXG001-307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Exegenesis Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Exegenesis Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Exegenesis Bio \/ Undisclosed"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"EXG34217","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Elixirgen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"EXG34217","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Elixirgen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Flatley Discovery Lab","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"FDL169","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Flatley Discovery Lab","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Flatley Discovery Lab \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Flatley Discovery Lab \/ Undisclosed"},{"orgOrder":0,"company":"Flatley Discovery Lab","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"FDL169","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Flatley Discovery Lab","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Flatley Discovery Lab \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Flatley Discovery Lab \/ Undisclosed"},{"orgOrder":0,"company":"Flatley Discovery Lab","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"FDL169","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Flatley Discovery Lab","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Flatley Discovery Lab \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Flatley Discovery Lab \/ Undisclosed"},{"orgOrder":0,"company":"Flatley Discovery Lab","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"FDL169","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Flatley Discovery Lab","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Flatley Discovery Lab \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Flatley Discovery Lab \/ Undisclosed"},{"orgOrder":0,"company":"Flatley Discovery Lab","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"FDL169","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Flatley Discovery Lab","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Flatley Discovery Lab \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Flatley Discovery Lab \/ Undisclosed"},{"orgOrder":0,"company":"Flatley Discovery Lab","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"FDL169","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Flatley Discovery Lab","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Flatley Discovery Lab \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Flatley Discovery Lab \/ Undisclosed"},{"orgOrder":0,"company":"Flatley Discovery Lab","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"FDL169","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Flatley Discovery Lab","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Flatley Discovery Lab \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Flatley Discovery Lab \/ Undisclosed"},{"orgOrder":0,"company":"Flatley Discovery Lab","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"FDL176","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Flatley Discovery Lab","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Flatley Discovery Lab \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Flatley Discovery Lab \/ Undisclosed"},{"orgOrder":0,"company":"Flatley Discovery Lab","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"FDL176","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Flatley Discovery Lab","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Flatley Discovery Lab \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Flatley Discovery Lab \/ Undisclosed"},{"orgOrder":0,"company":"Fibrocor Therapeutics","sponsor":"McQuade Center","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Collaboration","leadProduct":"FIB918","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Fibrocor Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Fibrocor Therapeutics \/ McQuade Center","highestDevelopmentStatusID":"5","companyTruncated":"Fibrocor Therapeutics \/ McQuade Center"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Acquisition","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Syncona Limited","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Syncona Limited"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Acquisition","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Syncona Limited","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Syncona Limited"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"FORCE-M23D","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Fordadistrogene","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"University of British Columbia","sponsor":"Vitaflo International, Ltd","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Free Amino Acid","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of British Columbia","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of British Columbia \/ Vitaflo International, Ltd","highestDevelopmentStatusID":"1","companyTruncated":"University of British Columbia \/ Vitaflo International, Ltd"},{"orgOrder":0,"company":"Jiangsu Gensciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"FRSW107","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jiangsu Gensciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jiangsu Gensciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Jiangsu Gensciences \/ Undisclosed"},{"orgOrder":0,"company":"Jiangsu Gensciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"FRSW117","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jiangsu Gensciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jiangsu Gensciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Jiangsu Gensciences \/ Undisclosed"},{"orgOrder":0,"company":"Jiangsu Gensciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"FRSW117","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jiangsu Gensciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jiangsu Gensciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Jiangsu Gensciences \/ Undisclosed"},{"orgOrder":0,"company":"Frontera Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"FT-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Frontera Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Frontera Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Frontera Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Frontera Therapeutics","sponsor":"Gene Therapy Research Center, Shariati Hospital, Tehran University of Medical Sciences, Iran | Peking Union Medical College Hospital | Henan Provincial People's Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FT-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Frontera Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Frontera Therapeutics \/ Gene Therapy Research Center, Shariati Hospital, Tehran University of Medical Sciences, Iran | Peking Union Medical College Hospital | Henan Provincial People's Hospital","highestDevelopmentStatusID":"7","companyTruncated":"Frontera Therapeutics \/ Gene Therapy Research Center, Shariati Hospital, Tehran University of Medical Sciences, Iran | Peking Union Medical College Hospital | Henan Provincial People's Hospital"},{"orgOrder":0,"company":"Frontera Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FT-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Frontera Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Frontera Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Frontera Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Children's Hospital Los Angeles","sponsor":"Grifols International","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"FVIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Children's Hospital Los Angeles","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Children's Hospital Los Angeles \/ Grifols International","highestDevelopmentStatusID":"11","companyTruncated":"Children's Hospital Los Angeles \/ Grifols International"},{"orgOrder":0,"company":"City of Hope","sponsor":"Charta Foundation | Grifols International | CSL Behring | Biotest","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"FVIII Concentrates","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"City of Hope","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"City of Hope \/ Charta Foundation | Grifols International | CSL Behring | Biotest","highestDevelopmentStatusID":"1","companyTruncated":"City of Hope \/ Charta Foundation | Grifols International | CSL Behring | Biotest"},{"orgOrder":0,"company":"University of California, Los Angeles","sponsor":"Boston Children's Hospital | National Heart, Lung, and Blood Institute | Genethon | California Institute for Regenerative Medicine (CIRM)","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"G1XCGD","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"University of California, Los Angeles","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"University of California, Los Angeles \/ Boston Children's Hospital | National Heart, Lung, and Blood Institute | Genethon | California Institute for Regenerative Medicine (CIRM)","highestDevelopmentStatusID":"7","companyTruncated":"University of California, Los Angeles \/ Boston Children's Hospital | National Heart, Lung, and Blood Institute | Genethon | California Institute for Regenerative Medicine (CIRM)"},{"orgOrder":0,"company":"Solve GNE","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Financing","leadProduct":"GA-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Solve GNE","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Solve GNE \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Solve GNE \/ Undisclosed"},{"orgOrder":0,"company":"Genosera","sponsor":"Solve GNE","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Agreement","leadProduct":"GA-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Genosera","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genosera \/ Solve GNE","highestDevelopmentStatusID":"4","companyTruncated":"Genosera \/ Solve GNE"},{"orgOrder":0,"company":"Children's University Hospital, Ireland","sponsor":"Health Research Board, Ireland | Medical Research Charities Group Ireland | University College Dublin","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Carbohydrate","year":"2014","type":"Inapplicable","leadProduct":"Galactose","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Children's University Hospital, Ireland","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Children's University Hospital, Ireland \/ Health Research Board, Ireland | Medical Research Charities Group Ireland | University College Dublin","highestDevelopmentStatusID":"1","companyTruncated":"Children's University Hospital, Ireland \/ Health Research Board, Ireland | Medical Research Charities Group Ireland | University College Dublin"},{"orgOrder":0,"company":"GeneCradle Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"GC101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GeneCradle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneCradle Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GeneCradle Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GeneCradle Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"GC101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GeneCradle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneCradle Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GeneCradle Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GeneCradle Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"GC101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GeneCradle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneCradle Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GeneCradle Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GeneCradle Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"GC101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GeneCradle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GeneCradle Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GeneCradle Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"GC1119","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Green Cross Lab Cell \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Green Cross Lab Cell \/ Undisclosed"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"GC1123","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GC Biopharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"GC Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"GC1130A","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GC Biopharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"GC Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Genecombio Ltd.","sponsor":"The Children's Hospital of Zhejiang University School of Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"GCB-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genecombio Ltd.","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genecombio Ltd. \/ The Children's Hospital of Zhejiang University School of Medicine","highestDevelopmentStatusID":"1","companyTruncated":"Genecombio Ltd. \/ The Children's Hospital of Zhejiang University School of Medicine"},{"orgOrder":0,"company":"Leiden University Medical Center","sponsor":"ZonMw | European Commission","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Gene Therapy","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Leiden University Medical Center","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Leiden University Medical Center \/ ZonMw | European Commission","highestDevelopmentStatusID":"1","companyTruncated":"Leiden University Medical Center \/ ZonMw | European Commission"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pill","sponsorNew":"Alkeus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alkeus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Bain Capital Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Series B Financing","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14999999999999999,"dosageForm":"Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Bain Capital Life Sciences","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Bain Capital Life Sciences"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"IPS Heart","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"GIVI-MPC","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"IPS Heart","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IPS Heart \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"IPS Heart \/ Undisclosed"},{"orgOrder":0,"company":"IPS Heart","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"GIVI-MPC","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"IPS Heart","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IPS Heart \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"IPS Heart \/ Undisclosed"},{"orgOrder":0,"company":"GLWL Research","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"GLWL-01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"GLWL Research","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"GLWL Research \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"GLWL Research \/ Undisclosed"},{"orgOrder":0,"company":"Nutricia Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Peptide, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"Glycomacropeptide-Based Protein Substitute","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Nutricia Inc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nutricia Inc \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Nutricia Inc \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"University College London Hospitals | University of Florida | Great Ormond Street Hospital for Children NHS Foundation Trust | University Medical Center Groningen | H\u00f4pital Necker-Enfants Malades","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"Glycosade","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vitaflo International, Ltd \/ University College London Hospitals | University of Florida | Great Ormond Street Hospital for Children NHS Foundation Trust | University Medical Center Groningen | H\u00f4pital Necker-Enfants Malades","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ University College London Hospitals | University of Florida | Great Ormond Street Hospital for Children NHS Foundation Trust | University Medical Center Groningen | H\u00f4pital Necker-Enfants Malades"},{"orgOrder":0,"company":"GlycosBio, Inc.","sponsor":"Baylor College of Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Dietary Supplement","year":"2021","type":"Inapplicable","leadProduct":"Glycosbio Nutritional Supplement","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"GlycosBio, Inc.","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GlycosBio, Inc. \/ Baylor College of Medicine","highestDevelopmentStatusID":"1","companyTruncated":"GlycosBio, Inc. \/ Baylor College of Medicine"},{"orgOrder":0,"company":"Ajinomoto Company","sponsor":"Birmingham Children's Hospital | Great Ormond Street Hospital for Children NHS Foundation Trust","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Glytactin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Ajinomoto Company","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ajinomoto Company \/ Birmingham Children's Hospital | Great Ormond Street Hospital for Children NHS Foundation Trust","highestDevelopmentStatusID":"1","companyTruncated":"Ajinomoto Company \/ Birmingham Children's Hospital | Great Ormond Street Hospital for Children NHS Foundation Trust"},{"orgOrder":0,"company":"Nutricia Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Dietary Supplement","year":"2025","type":"Inapplicable","leadProduct":"GMP Intervention Product","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Nutricia Inc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nutricia Inc \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Nutricia Inc \/ Undisclosed"},{"orgOrder":0,"company":"Dr. Sch\u00e4r AG \/ SPA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Dietary Supplement","year":"2020","type":"Inapplicable","leadProduct":"GMPOWER","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dr. Sch\u00e4r AG \/ SPA","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dr. Sch\u00e4r AG \/ SPA \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Dr. Sch\u00e4r AG \/ SPA \/ Undisclosed"},{"orgOrder":0,"company":"Generium","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"GNR-038","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Generium \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Generium \/ Undisclosed"},{"orgOrder":0,"company":"Generium","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"GNR-038","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Generium \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Generium \/ Undisclosed"},{"orgOrder":0,"company":"Generium","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"GNR-055","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Generium \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Generium \/ Undisclosed"},{"orgOrder":0,"company":"Generium","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"GNR-055","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Generium \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Generium \/ Undisclosed"},{"orgOrder":0,"company":"Genethon","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Collaboration","leadProduct":"GNT0004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genethon \/ Sarepta Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Genethon","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"GNT0004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genethon \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Undisclosed"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"GS030","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GenSight Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GenSight Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GenSight Biologics \/ Undisclosed"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"GS030","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GenSight Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GenSight Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GenSight Biologics \/ Undisclosed"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"GS030-DP","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GenSight Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GenSight Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GenSight Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Grace Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"GS-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grace Science \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Grace Science \/ Undisclosed"},{"orgOrder":0,"company":"Grace Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"GS-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grace Science \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Grace Science \/ Undisclosed"},{"orgOrder":0,"company":"Grace Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"GS-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grace Science \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Grace Science \/ Undisclosed"},{"orgOrder":0,"company":"Andelyn Biosciences","sponsor":"Grace Science","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Partnership","leadProduct":"GS-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Andelyn Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Andelyn Biosciences \/ Grace Science","highestDevelopmentStatusID":"7","companyTruncated":"Andelyn Biosciences \/ Grace Science"},{"orgOrder":0,"company":"Grace Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"GS-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grace Science \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Grace Science \/ Undisclosed"},{"orgOrder":0,"company":"Gritgen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"GS1191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Gritgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Gritgen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Gritgen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Gritgen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"GS1191-0445","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Gritgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Gritgen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Gritgen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"GSK3039294","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"GSK3039294","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"GSK3439171A","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"GSK356278","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"GSK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"GSK356278","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GSK \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"GSK \/ Undisclosed"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"GT-00513","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gain Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Mitsubishi UFJ Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell & Gene Therapy","year":"2020","type":"Collaboration","leadProduct":"GtCCR4","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Daiichi Sankyo \/ Mitsubishi UFJ Capital","highestDevelopmentStatusID":"1","companyTruncated":"Daiichi Sankyo \/ Mitsubishi UFJ Capital"},{"orgOrder":0,"company":"hC Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"HCB-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"hC Bioscience","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"hC Bioscience \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"hC Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"University of Virginia","sponsor":"Xemed LLC","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Helium-3","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Virginia","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Virginia \/ Xemed LLC","highestDevelopmentStatusID":"1","companyTruncated":"University of Virginia \/ Xemed LLC"},{"orgOrder":0,"company":"Talaris Therapeutics","sponsor":"Duke University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2011","type":"Inapplicable","leadProduct":"Hematopoietic Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Talaris Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Talaris Therapeutics \/ Duke University","highestDevelopmentStatusID":"7","companyTruncated":"Talaris Therapeutics \/ Duke University"},{"orgOrder":0,"company":"HuidaGene Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"HG004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"HuidaGene Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"HuidaGene Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"HuidaGene Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"HuidaGene Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"HG005","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"HuidaGene Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"HuidaGene Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"HuidaGene Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"HuidaGene Therapeutics","sponsor":"Peking University First Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"HG204","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"HuidaGene Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"HuidaGene Therapeutics \/ Peking University First Hospital","highestDevelopmentStatusID":"1","companyTruncated":"HuidaGene Therapeutics \/ Peking University First Hospital"},{"orgOrder":0,"company":"HuidaGene Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"HG302","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"HuidaGene Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"HuidaGene Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"HuidaGene Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"HGB-206","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"HGB-206","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"HGB-206","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Cytonet","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"Hhlivc","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cytonet","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cytonet \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cytonet \/ Undisclosed"},{"orgOrder":0,"company":"HANDOK","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"HL2351","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"HANDOK","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"HANDOK \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"HANDOK \/ Undisclosed"},{"orgOrder":0,"company":"Healx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"HLX-0201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Healx","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Healx \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Healx \/ Undisclosed"},{"orgOrder":0,"company":"Healx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"HLX-1502","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Healx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Healx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Healx \/ Undisclosed"},{"orgOrder":0,"company":"Healx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"HLX-1502","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Healx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Healx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Healx \/ Undisclosed"},{"orgOrder":0,"company":"Healx","sponsor":"R42 Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2024","type":"Series C Financing","leadProduct":"HLX-1502","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Healx","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Healx \/ R42 Group","highestDevelopmentStatusID":"8","companyTruncated":"Healx \/ R42 Group"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-203","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"HMI-203","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"ReNeuron","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"hRPC Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReNeuron","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ReNeuron \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReNeuron \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Human Coagulation Factor VIII Complex","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Human Coagulation Factor VIII Complex","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Human Coagulation Factor VIII Complex","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Human Coagulation Factor VIII Complex","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Human Immunoglobulin with Recombinant Hyaluronidase","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Baxalta Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2017","type":"Inapplicable","leadProduct":"Human Immunoglobulin with Recombinant Hyaluronidase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Baxalta Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2017","type":"Inapplicable","leadProduct":"Human Immunoglobulin with Recombinant Hyaluronidase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical"},{"orgOrder":0,"company":"jCyte","sponsor":"California Institute for Regenerative Medicine (CIRM)","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"Human Retinal Progenitor Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"jCyte","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"jCyte \/ California Institute for Regenerative Medicine (CIRM)","highestDevelopmentStatusID":"8","companyTruncated":"jCyte \/ California Institute for Regenerative Medicine (CIRM)"},{"orgOrder":0,"company":"jCyte","sponsor":"California Institute for Regenerative Medicine (CIRM)","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Human Retinal Progenitor Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"jCyte","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"jCyte \/ California Institute for Regenerative Medicine (CIRM)","highestDevelopmentStatusID":"8","companyTruncated":"jCyte \/ California Institute for Regenerative Medicine (CIRM)"},{"orgOrder":0,"company":"jCyte","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Human Retinal Progenitor Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"jCyte","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"jCyte \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"jCyte \/ Undisclosed"},{"orgOrder":0,"company":"jCyte","sponsor":"California Institute for Regenerative Medicine (CIRM)","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2014","type":"Inapplicable","leadProduct":"Human Retinal Progenitor Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"jCyte","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"jCyte \/ California Institute for Regenerative Medicine (CIRM)","highestDevelopmentStatusID":"8","companyTruncated":"jCyte \/ California Institute for Regenerative Medicine (CIRM)"},{"orgOrder":0,"company":"jCyte","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Allogeneic Human Retinal Progenitor Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"jCyte","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"jCyte \/ California Institute for Regenerative Medicine","highestDevelopmentStatusID":"8","companyTruncated":"jCyte \/ California Institute for Regenerative Medicine"},{"orgOrder":0,"company":"jCyte","sponsor":"Santen Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Licensing Agreement","leadProduct":"Allogeneic Human Retinal Progenitor Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"jCyte","amount2":0.25,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.25,"dosageForm":"Injection","sponsorNew":"jCyte \/ Santen Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"jCyte \/ Santen Pharmaceutical"},{"orgOrder":0,"company":"jCyte","sponsor":"Center for Breakthrough Medicines","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Partnership","leadProduct":"Allogeneic Human Retinal Progenitor Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"jCyte","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"jCyte \/ Center for Breakthrough Medicines","highestDevelopmentStatusID":"8","companyTruncated":"jCyte \/ Center for Breakthrough Medicines"},{"orgOrder":0,"company":"jCyte","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Allogeneic Human Retinal Progenitor Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"jCyte","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"jCyte \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"jCyte \/ Undisclosed"},{"orgOrder":0,"company":"jCyte","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Allogeneic Human Retinal Progenitor Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"jCyte","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"jCyte \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"jCyte \/ Undisclosed"},{"orgOrder":0,"company":"jCyte","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Allogeneic Human Retinal Progenitor Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"jCyte","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"jCyte \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"jCyte \/ Undisclosed"},{"orgOrder":0,"company":"ReNeuron","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2015","type":"Inapplicable","leadProduct":"Human Retinal Progenitor Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReNeuron","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"ReNeuron \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReNeuron \/ Undisclosed"},{"orgOrder":0,"company":"MED Institute Inc.","sponsor":"Signature Biologics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Human Umbilical Cord Mesenchymal Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"MED Institute Inc.","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MED Institute Inc. \/ Signature Biologics","highestDevelopmentStatusID":"1","companyTruncated":"MED Institute Inc. \/ Signature Biologics"},{"orgOrder":0,"company":"Kamada","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"Human, Alpha-1 Antitrypsin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Kamada","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Kamada \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Kamada \/ Undisclosed"},{"orgOrder":0,"company":"Attralus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"I124-AT01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Attralus \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Attralus \/ Undisclosed"},{"orgOrder":0,"company":"Rumi Scientific","sponsor":"X-Chem","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Partnership","leadProduct":"iBRD9","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Rumi Scientific","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rumi Scientific \/ X-Chem","highestDevelopmentStatusID":"3","companyTruncated":"Rumi Scientific \/ X-Chem"},{"orgOrder":0,"company":"Immusoft Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Iduronicrin Genleukocel-T","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Immusoft Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Immusoft Corporation \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Immusoft Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Immusoft Corporation","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Funding","leadProduct":"Iduronicrin Genleukocel-T","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Immusoft Corporation","amount2":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"Immusoft Corporation \/ California Institute for Regenerative Medicine","highestDevelopmentStatusID":"6","companyTruncated":"Immusoft Corporation \/ California Institute for Regenerative Medicine"},{"orgOrder":0,"company":"Immusoft Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Iduronicrin Genleukocel-T","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Immusoft Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Immusoft Corporation \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Immusoft Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Immusoft Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Iduronicrin Genleukocel-T","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Immusoft Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Immusoft Corporation \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Immusoft Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Intergalactic Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"IG-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intergalactic Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Intergalactic Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Intergalactic Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"IgPro","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"University of South Florida","sponsor":"Baxter Healthcare Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Immunoglobulins||Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of South Florida","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of South Florida \/ Baxter Healthcare Corporation","highestDevelopmentStatusID":"8","companyTruncated":"University of South Florida \/ Baxter Healthcare Corporation"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"University of South Florida","sponsor":"Baxter Healthcare Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of South Florida","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"University of South Florida \/ Baxter Healthcare Corporation","highestDevelopmentStatusID":"8","companyTruncated":"University of South Florida \/ Baxter Healthcare Corporation"},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"ADMA Biologics","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ADMA Biologics \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"ADMA Biologics \/ Undisclosed"},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Human Immunoglobulin","moa":"Fc receptor||Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ADMA Biologics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ADMA Biologics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"ADMA Biologics \/ Undisclosed"},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Human Immunoglobulin","moa":"Fc receptor||Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ADMA Biologics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ADMA Biologics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"ADMA Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Atlantic Research Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Green Cross Lab Cell \/ Atlantic Research Group","highestDevelopmentStatusID":"10","companyTruncated":"Green Cross Lab Cell \/ Atlantic Research Group"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"On Pharma","sponsor":"Hospital do Coracao","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"On Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"On Pharma \/ Hospital do Coracao","highestDevelopmentStatusID":"10","companyTruncated":"On Pharma \/ Hospital do Coracao"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Therapure Biomanufacturing","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Therapure Biomanufacturing","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Therapure Biomanufacturing \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Therapure Biomanufacturing \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Immune Globulin G","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Pre-Filled","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Immune Globulin Intravenous, Human","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"GC Biopharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"GC Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Immune Globulin Intravenous, Human","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"GC Biopharma \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"GC Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"KabaFusion","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Protein","year":"2024","type":"Partnership","leadProduct":"Immune Globulin Intravenous, Human","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"GC Biopharma \/ KabaFusion","highestDevelopmentStatusID":"15","companyTruncated":"GC Biopharma \/ KabaFusion"},{"orgOrder":0,"company":"Biotest","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Immune Globulin Intravenous, Human","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biotest","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biotest \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Biotest \/ Undisclosed"},{"orgOrder":0,"company":"Soleo Health","sponsor":"GC Biopharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Partnership","leadProduct":"Immune Globulin Intravenous, Human","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Soleo Health","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Soleo Health \/ GC Biopharma","highestDevelopmentStatusID":"15","companyTruncated":"Soleo Health \/ GC Biopharma"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Immune Globulin, Human","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"CSI Pharmacy","sponsor":"Bio Products Laboratory","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Agreement","leadProduct":"Human Immunoglobulin","moa":"Fc receptor||Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSI Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSI Pharmacy \/ Bio Products Laboratory","highestDevelopmentStatusID":"15","companyTruncated":"CSI Pharmacy \/ Bio Products Laboratory"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Immunoglobulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Immunoglobulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Green Cross Lab Cell \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Green Cross Lab Cell \/ Undisclosed"},{"orgOrder":0,"company":"Prothya Biosolutions","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Antibody, Unconjugated","year":"2013","type":"Inapplicable","leadProduct":"Immunoglobulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prothya Biosolutions","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prothya Biosolutions \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prothya Biosolutions \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2014","type":"Inapplicable","leadProduct":"Immunoglobulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Immunoglobulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Virchow Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Immunoglobulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Virchow Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Virchow Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Virchow Group \/ Undisclosed"},{"orgOrder":0,"company":"Incyte Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"INCB059872","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Incyte Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Incyte Corporation \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Incyte Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Evotec","sponsor":"Centogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2020","type":"Expanded Collaboration","leadProduct":"Induced Pluripotent Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Evotec","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Evotec \/ Centogene","highestDevelopmentStatusID":"2","companyTruncated":"Evotec \/ Centogene"},{"orgOrder":0,"company":"Generation Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"iqDNA","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Generation Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Generation Bio \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Generation Bio \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"ITU512","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"InnoVec Biotherapeutics","sponsor":"Peking Union Medical College Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"IVB102","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"InnoVec Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"InnoVec Biotherapeutics \/ Peking Union Medical College Hospital","highestDevelopmentStatusID":"6","companyTruncated":"InnoVec Biotherapeutics \/ Peking Union Medical College Hospital"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Series B Financing","leadProduct":"JAG101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jaguar Gene Therapy","amount2":0.14000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Jaguar Gene Therapy \/ Eli Lilly","highestDevelopmentStatusID":"4","companyTruncated":"Jaguar Gene Therapy \/ Eli Lilly"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"JAG101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jaguar Gene Therapy","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jaguar Gene Therapy \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Jaguar Gene Therapy \/ Undisclosed"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"JAG101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jaguar Gene Therapy","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jaguar Gene Therapy \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Jaguar Gene Therapy \/ Undisclosed"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"JAG201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jaguar Gene Therapy","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jaguar Gene Therapy \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jaguar Gene Therapy \/ Undisclosed"},{"orgOrder":0,"company":"Jemincare","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"JMKX003142","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Jemincare","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jemincare \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Jemincare \/ Undisclosed"},{"orgOrder":0,"company":"Jemincare","sponsor":"Zhejiang Hangyu Pharmaceutical Co., Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JMKX003142","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Jemincare","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jemincare \/ Zhejiang Hangyu Pharmaceutical Co., Ltd.","highestDevelopmentStatusID":"8","companyTruncated":"Jemincare \/ Zhejiang Hangyu Pharmaceutical Co., Ltd."},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JR-446","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Medipal Holdings Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JR-446","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Medipal Holdings Corporation","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Medipal Holdings Corporation"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Medipal Holdings Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JR-446","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Medipal Holdings Corporation","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Medipal Holdings Corporation"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"JR-446","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Kaleido Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"KB195","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Kaleido Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kaleido Biosciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Kaleido Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"KB408","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Krystal Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"KB408","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Krystal Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"KB408","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Krystal Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"KB408","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Krystal Biotech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Kedrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Kedrion IVIG","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kedrion","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kedrion \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kedrion \/ Undisclosed"},{"orgOrder":0,"company":"Kedrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Kedrion IVIG","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kedrion","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kedrion \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kedrion \/ Undisclosed"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"KK8398","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kyowa Kirin \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kyowa Kirin \/ Undisclosed"},{"orgOrder":0,"company":"Alphamab Oncology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"KN057","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alphamab Oncology","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alphamab Oncology \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alphamab Oncology \/ Undisclosed"},{"orgOrder":0,"company":"Alphamab Oncology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"KN057","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alphamab Oncology","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alphamab Oncology \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alphamab Oncology \/ Undisclosed"},{"orgOrder":0,"company":"Alphamab Oncology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"KN057","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alphamab Oncology","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alphamab Oncology \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alphamab Oncology \/ Undisclosed"},{"orgOrder":0,"company":"Alphamab Oncology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"KN057","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alphamab Oncology","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alphamab Oncology \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Alphamab Oncology \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"WuXi Biologics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2020","type":"Acquisition","leadProduct":"Koate DVI","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ WuXi Biologics","highestDevelopmentStatusID":"15","companyTruncated":"Bayer AG \/ WuXi Biologics"},{"orgOrder":0,"company":"Kate Therapeutics","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Acquisition","leadProduct":"KT809","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Kate Therapeutics","amount2":1.1000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":1.1000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Kate Therapeutics \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"4","companyTruncated":"Kate Therapeutics \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Expression Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Lentivirus-Hematopoietic Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Expression Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Expression Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Expression Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orpha Labs","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Carbohydrate","year":"2017","type":"Inapplicable","leadProduct":"L-Fucose","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orpha Labs","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orpha Labs \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orpha Labs \/ Undisclosed"},{"orgOrder":0,"company":"AskBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LION-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Undisclosed"},{"orgOrder":0,"company":"Dr. Sch\u00e4r AG \/ SPA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Dietary Supplement","year":"2020","type":"Inapplicable","leadProduct":"Lipano","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dr. Sch\u00e4r AG \/ SPA","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dr. Sch\u00e4r AG \/ SPA \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Dr. Sch\u00e4r AG \/ SPA \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Lipid Nanoparticles","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nutricia Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Low Calorie Protein Substitute","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Nutricia Inc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Nutricia Inc \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Nutricia Inc \/ Undisclosed"},{"orgOrder":0,"company":"Luye Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"LPM3770164","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Luye Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Sustained Release","sponsorNew":"Luye Pharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Luye Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Luye Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"LPM3770164","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Luye Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Sustained Release","sponsorNew":"Luye Pharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Luye Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Shanghai General Hospital","sponsor":"Innostellar Biotherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"LX103","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Shanghai General Hospital","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Shanghai General Hospital \/ Innostellar Biotherapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Shanghai General Hospital \/ Innostellar Biotherapeutics"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"LY3884961","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prevail Therapeutics \/ Eli Lilly","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Eli Lilly"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"LY3884961","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Prevail Therapeutics \/ Eli Lilly","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Eli Lilly"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Bpifrance","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Financing","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Bpifrance","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Bpifrance"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Axplora","sponsor":"Lysogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2020","type":"Collaboration","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Axplora","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Axplora \/ Lysogene","highestDevelopmentStatusID":"6","companyTruncated":"Axplora \/ Lysogene"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"M6P Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"M041","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"M6P Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"M6P Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"M6P Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"M6P Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"M041","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"M6P Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"M6P Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"M6P Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Hunterian Medicine","sponsor":"Inscripta","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Licensing Agreement","leadProduct":"MAD7 Nuclease","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Hunterian Medicine","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hunterian Medicine \/ Inscripta","highestDevelopmentStatusID":"1","companyTruncated":"Hunterian Medicine \/ Inscripta"},{"orgOrder":0,"company":"Sirion Biotech","sponsor":"Mustang Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2020","type":"Licensing Agreement","leadProduct":"MB-207","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sirion Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sirion Biotech \/ Mustang Bio","highestDevelopmentStatusID":"7","companyTruncated":"Sirion Biotech \/ Mustang Bio"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Busulfan","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-207","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-207","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Medibiofarma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"MBF-015","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Medibiofarma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Medibiofarma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Medibiofarma \/ Undisclosed"},{"orgOrder":0,"company":"Matrix Biomed","sponsor":"University of Texas Health Science Center at Houston","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"MBM-01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Matrix Biomed","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Matrix Biomed \/ University of Texas Health Science Center at Houston","highestDevelopmentStatusID":"8","companyTruncated":"Matrix Biomed \/ University of Texas Health Science Center at Houston"},{"orgOrder":0,"company":"MitoChem Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"MC16","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"MitoChem Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MitoChem Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"MitoChem Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Genevant Sciences","sponsor":"2Seventy Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Cell & Gene Therapy","year":"2022","type":"Licensing Agreement","leadProduct":"MegaTAL mRNA","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genevant Sciences","amount2":0.040000000000000001,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Genevant Sciences \/ 2Seventy Bio","highestDevelopmentStatusID":"1","companyTruncated":"Genevant Sciences \/ 2Seventy Bio"},{"orgOrder":0,"company":"2Seventy Bio","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Collaboration","leadProduct":"MegaTAL mRNA","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"2Seventy Bio","amount2":0.040000000000000001,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Undisclosed","sponsorNew":"2Seventy Bio \/ Novo Nordisk","highestDevelopmentStatusID":"1","companyTruncated":"2Seventy Bio \/ Novo Nordisk"},{"orgOrder":0,"company":"Elpida Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Melpida Gene Therapy","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Elpida Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Elpida Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Elpida Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Metagenomi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"MGX-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ Undisclosed"},{"orgOrder":0,"company":"SR-Tiget","sponsor":"Genespire","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2020","type":"Collaboration","leadProduct":"Microrna-Regulated Lentiviral Vectors","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"SR-Tiget","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SR-Tiget \/ Genespire","highestDevelopmentStatusID":"1","companyTruncated":"SR-Tiget \/ Genespire"},{"orgOrder":0,"company":"MMJ International Holding","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"MMJ-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"MMJ International Holding","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MMJ International Holding \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"MMJ International Holding \/ Undisclosed"},{"orgOrder":0,"company":"Minovia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"MNV-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Minovia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Minovia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Minovia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"OPKO Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"MOD-5014","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"OPKO Health","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"OPKO Health \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"OPKO Health \/ Undisclosed"},{"orgOrder":0,"company":"OPKO Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"MOD-5014","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"OPKO Health","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"OPKO Health \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"OPKO Health \/ Undisclosed"},{"orgOrder":0,"company":"Nestle Health Sciences SA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Dietary Supplement","year":"2016","type":"Inapplicable","leadProduct":"Modified Lipid Formulation","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Nestle Health Sciences SA","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Nestle Health Sciences SA \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Nestle Health Sciences SA \/ Undisclosed"},{"orgOrder":0,"company":"Dr. Sch\u00e4r AG \/ SPA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Dietary Supplement","year":"2020","type":"Inapplicable","leadProduct":"Motion","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dr. Sch\u00e4r AG \/ SPA","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dr. Sch\u00e4r AG \/ SPA \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Dr. Sch\u00e4r AG \/ SPA \/ Undisclosed"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vaccine","year":"2022","type":"Inapplicable","leadProduct":"mRNA-3139","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Moderna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Moderna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"mRNA-3210","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Moderna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"MRT5201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"AzurRx SAS","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"MS1819-SD","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AzurRx SAS","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AzurRx SAS \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AzurRx SAS \/ Undisclosed"},{"orgOrder":0,"company":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine","sponsor":"Mabwell","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"MWAV201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine \/ Mabwell","highestDevelopmentStatusID":"1","companyTruncated":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine \/ Mabwell"},{"orgOrder":0,"company":"Myogem Health Company, S.L.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Dietary Supplement","year":"2020","type":"Inapplicable","leadProduct":"Myodm","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Myogem Health Company, S.L.","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Myogem Health Company, S.L. \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Myogem Health Company, S.L. \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"NBIP-01435","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"NextCure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"NC605","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NextCure","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NextCure \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"NextCure \/ Undisclosed"},{"orgOrder":0,"company":"Erempharma","sponsor":"Hospices Civils de Lyon | University of Lyon","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"Nebcinal Tobi","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Erempharma","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Erempharma \/ Hospices Civils de Lyon | University of Lyon","highestDevelopmentStatusID":"1","companyTruncated":"Erempharma \/ Hospices Civils de Lyon | University of Lyon"},{"orgOrder":0,"company":"AgeX Therapeutics","sponsor":"University of California San Diego","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Collaboration","leadProduct":"Neural Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"AgeX Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AgeX Therapeutics \/ University of California San Diego","highestDevelopmentStatusID":"1","companyTruncated":"AgeX Therapeutics \/ University of California San Diego"},{"orgOrder":0,"company":"NGGT Suzhou Biotechnology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NGGT002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"NGGT Suzhou Biotechnology","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NGGT Suzhou Biotechnology \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"NGGT Suzhou Biotechnology \/ Undisclosed"},{"orgOrder":0,"company":"Next Generation Gene Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NGGT002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Next Generation Gene Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Next Generation Gene Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Next Generation Gene Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"NGGT","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NGGT002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"NGGT","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NGGT \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"NGGT \/ Undisclosed"},{"orgOrder":0,"company":"Neurogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"NGN-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"NNC0165-1562","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"NNC0442-0344","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Neuren Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Neuren Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Neuren Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Neuren Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Tahitian Noni International, Inc.","sponsor":"University of Illinois, Chicago","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Plant Extract\/Herbal","year":"2012","type":"Inapplicable","leadProduct":"Noni Juice","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tahitian Noni International, Inc.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tahitian Noni International, Inc. \/ University of Illinois, Chicago","highestDevelopmentStatusID":"8","companyTruncated":"Tahitian Noni International, Inc. \/ University of Illinois, Chicago"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Quince Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Acquisition","leadProduct":"NOV004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Quince Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Quince Therapeutics \/ Quince Therapeutics","highestDevelopmentStatusID":"5","companyTruncated":"Quince Therapeutics \/ Quince Therapeutics"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Quince Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Acquisition","leadProduct":"NOV004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Quince Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Quince Therapeutics \/ Quince Therapeutics","highestDevelopmentStatusID":"5","companyTruncated":"Quince Therapeutics \/ Quince Therapeutics"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"NOV004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Quince Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Quince Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Quince Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"NOV004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Quince Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Quince Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Quince Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"NOV004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Quince Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Quince Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Quince Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"NS-050","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NS Pharma \/ Nippon Shinyaku","highestDevelopmentStatusID":"7","companyTruncated":"NS Pharma \/ Nippon Shinyaku"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NS-050\/NCNP-03","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NS-050\/NCNP-03","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"National Center of Neurology and Psychiatry, Japan","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"NS-089","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"National Center of Neurology and Psychiatry, Japan","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Center of Neurology and Psychiatry, Japan \/ Nippon Shinyaku","highestDevelopmentStatusID":"7","companyTruncated":"National Center of Neurology and Psychiatry, Japan \/ Nippon Shinyaku"},{"orgOrder":0,"company":"Nippon Shinyaku","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"NS-089","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nippon Shinyaku","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nippon Shinyaku \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Nippon Shinyaku \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"NS-089","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NS Pharma \/ Nippon Shinyaku","highestDevelopmentStatusID":"8","companyTruncated":"NS Pharma \/ Nippon Shinyaku"},{"orgOrder":0,"company":"NeuBase Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"NT-0231.F","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NeuBase Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"NeuBase Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"NeuBase Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"NeuBase Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"NT-0231.F","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NeuBase Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"NeuBase Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"NeuBase Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NTLA-3001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"NTLA-3001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Nucresiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alnylam Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Alnylam Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Kamau Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Nula-Cel","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Kamau Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kamau Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Kamau Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"LGD","sponsor":"CEN Biotech | Assistance Publique Hopitaux De Marseille | Etablissement Fran\u00e7ais du Sang","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Dietary Supplement","year":"2023","type":"Inapplicable","leadProduct":"NUV001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"LGD","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"LGD \/ CEN Biotech | Assistance Publique Hopitaux De Marseille | Etablissement Fran\u00e7ais du Sang","highestDevelopmentStatusID":"1","companyTruncated":"LGD \/ CEN Biotech | Assistance Publique Hopitaux De Marseille | Etablissement Fran\u00e7ais du Sang"},{"orgOrder":0,"company":"LGD","sponsor":"Prorelix Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Dietary Supplement","year":"2023","type":"Inapplicable","leadProduct":"NUV001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"LGD","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"LGD \/ Prorelix Research","highestDevelopmentStatusID":"1","companyTruncated":"LGD \/ Prorelix Research"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"N-sulphoglucosamine sulphohydrolase (SGSH)||Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Lysogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Termination","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sarepta Therapeutics \/ Lysogene","highestDevelopmentStatusID":"9","companyTruncated":"Sarepta Therapeutics \/ Lysogene"},{"orgOrder":0,"company":"BDD Pharma","sponsor":"AlgiPharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"OligoG CF-5\/20","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"BDD Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"BDD Pharma \/ AlgiPharma","highestDevelopmentStatusID":"8","companyTruncated":"BDD Pharma \/ AlgiPharma"},{"orgOrder":0,"company":"Emendo Biotherapeutics","sponsor":"AnGes","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Series B Financing","leadProduct":"OMNI Nuclease","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Emendo Biotherapeutics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Emendo Biotherapeutics \/ AnGes","highestDevelopmentStatusID":"1","companyTruncated":"Emendo Biotherapeutics \/ AnGes"},{"orgOrder":0,"company":"Omeros","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"OMS643762","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Omeros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Omeros \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Omeros \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec-Xioi","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Fujifilm Cellular Dynamics","sponsor":"BlueRock Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Licensing Agreement","leadProduct":"OpCT-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Fujifilm Cellular Dynamics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Fujifilm Cellular Dynamics \/ BlueRock Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Fujifilm Cellular Dynamics \/ BlueRock Therapeutics"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Foundation Fighting Blindness","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Funding","leadProduct":"OPGx-RHO","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Opus Genetics \/ Foundation Fighting Blindness","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ Foundation Fighting Blindness"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"Optivate Human Coagulation Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"Optivate Human Coagulation Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"Optivate Human Coagulation Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"Optivate Human Coagulation Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"Optivate Human Coagulation Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Orpha Labs","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"ORL-1M - D-mannose","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orpha Labs","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orpha Labs \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orpha Labs \/ Undisclosed"},{"orgOrder":0,"company":"Orpha Labs","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"Orl-D-Galactose","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orpha Labs","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orpha Labs \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orpha Labs \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTQ923","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Memorial Sloan Kettering Cancer Center","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTQ923","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Memorial Sloan Kettering Cancer Center","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Memorial Sloan Kettering Cancer Center \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Memorial Sloan Kettering Cancer Center \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTQ923","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Collaboration","leadProduct":"PBGENE-HbE","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Precision BioSciences","amount2":1.48,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.48,"dosageForm":"Undisclosed","sponsorNew":"Precision BioSciences \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"2","companyTruncated":"Precision BioSciences \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Prana Biotechnology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"PBT2","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Prana Biotechnology","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prana Biotechnology \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Prana Biotechnology \/ Undisclosed"},{"orgOrder":0,"company":"Celularity","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"PDA-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Celularity","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Celularity \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Celularity \/ Undisclosed"},{"orgOrder":0,"company":"Ascension Healthcare Development","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Pegylated Liposome","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ascension Healthcare Development","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ascension Healthcare Development \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ascension Healthcare Development \/ Undisclosed"},{"orgOrder":0,"company":"Poseida Therapeutics, Inc","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Collaboration","leadProduct":"P-FVIII-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Poseida Therapeutics, Inc","amount2":3.6000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":3.6000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Poseida Therapeutics, Inc \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Poseida Therapeutics, Inc \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Nutricia Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Phenylalanine-Free Protein","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Nutricia Inc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nutricia Inc \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Nutricia Inc \/ Undisclosed"},{"orgOrder":0,"company":"GALEN LTD","sponsor":"Birmingham Women's and Children's NHS Foundation Trust","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2024","type":"Inapplicable","leadProduct":"PKU Easy Microtabs Plus","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"GALEN LTD","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"GALEN LTD \/ Birmingham Women's and Children's NHS Foundation Trust","highestDevelopmentStatusID":"1","companyTruncated":"GALEN LTD \/ Birmingham Women's and Children's NHS Foundation Trust"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Birmingham Women's and Children's NHS Foundation Trust","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2017","type":"Inapplicable","leadProduct":"PKU Explore","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Birmingham Women's and Children's NHS Foundation Trust","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Birmingham Women's and Children's NHS Foundation Trust"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2021","type":"Inapplicable","leadProduct":"PKU Explore","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2021","type":"Inapplicable","leadProduct":"PKU Express Plus","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Undisclosed"},{"orgOrder":0,"company":"APR Applied Pharma Research","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"PKU Golike","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"APR Applied Pharma Research","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"APR Applied Pharma Research \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"APR Applied Pharma Research \/ Undisclosed"},{"orgOrder":0,"company":"APR Applied Pharma Research","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"PKU Golike","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"APR Applied Pharma Research","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"APR Applied Pharma Research \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"APR Applied Pharma Research \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Oregon Health and Science University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2018","type":"Inapplicable","leadProduct":"PKU Sphere","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Oregon Health and Science University","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Oregon Health and Science University"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Kreiskliniken Reutlingen | Johannes Gutenberg University Mainz","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2018","type":"Inapplicable","leadProduct":"PKU Sphere","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Kreiskliniken Reutlingen | Johannes Gutenberg University Mainz","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Kreiskliniken Reutlingen | Johannes Gutenberg University Mainz"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2020","type":"Inapplicable","leadProduct":"PKU Sphere","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2021","type":"Inapplicable","leadProduct":"PKU Sphere","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2017","type":"Inapplicable","leadProduct":"PKU Start","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Birmingham Women's and Children's NHS Foundation Trust","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2023","type":"Inapplicable","leadProduct":"PKU UP","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vitaflo International, Ltd \/ Birmingham Women's and Children's NHS Foundation Trust","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Birmingham Women's and Children's NHS Foundation Trust"},{"orgOrder":0,"company":"Nutricia Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"PKU\/TYR GMP Protein","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Nutricia Inc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nutricia Inc \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Nutricia Inc \/ Undisclosed"},{"orgOrder":0,"company":"Fondazione Angelo Bianchi Bonomi","sponsor":"Sintesi Research Srl","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"Plasma Derived Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Fondazione Angelo Bianchi Bonomi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Fondazione Angelo Bianchi Bonomi \/ Sintesi Research Srl","highestDevelopmentStatusID":"11","companyTruncated":"Fondazione Angelo Bianchi Bonomi \/ Sintesi Research Srl"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"PM359","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prime Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prime Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Prime Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"PM359","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prime Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prime Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Prime Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"PM359","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prime Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prime Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Prime Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"PM359","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prime Medicine","amount2":0.13,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Infusion","sponsorNew":"Prime Medicine \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Prime Medicine \/ J.P. Morgan"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"PM359","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prime Medicine","amount2":0.16,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Infusion","sponsorNew":"Prime Medicine \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Prime Medicine \/ J.P. Morgan"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"HLS Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2020","type":"Acquisition","leadProduct":"Porcine recombinant Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Takeda Pharmaceutical","amount2":0.050000000000000003,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Takeda Pharmaceutical \/ HLS Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Takeda Pharmaceutical \/ HLS Therapeutics"},{"orgOrder":0,"company":"Portal Therapeutics","sponsor":"Celerion","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"PORT-77","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Portal Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Portal Therapeutics \/ Celerion","highestDevelopmentStatusID":"8","companyTruncated":"Portal Therapeutics \/ Celerion"},{"orgOrder":0,"company":"Papillon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"PPL-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Papillon Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Papillon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Papillon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PRG Science & Technology Co., Ltd.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Progerinin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PRG Science & Technology Co., Ltd.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PRG Science & Technology Co., Ltd. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"PRG Science & Technology Co., Ltd. \/ Undisclosed"},{"orgOrder":0,"company":"PRG Science & Technology Co., Ltd.","sponsor":"Amarex Clinical Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Progerinin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PRG Science & Technology Co., Ltd.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PRG Science & Technology Co., Ltd. \/ Amarex Clinical Research","highestDevelopmentStatusID":"8","companyTruncated":"PRG Science & Technology Co., Ltd. \/ Amarex Clinical Research"},{"orgOrder":0,"company":"PRG S&Tech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Progerinin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"PRG S&Tech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PRG S&Tech \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"PRG S&Tech \/ Undisclosed"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"PTX-022","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palvella Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Palvella Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"PTX-022","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palvella Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Palvella Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"PTX-022","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palvella Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Palvella Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Pulmatrix","sponsor":"Celerion","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"PUR118","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pulmatrix","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pulmatrix \/ Celerion","highestDevelopmentStatusID":"6","companyTruncated":"Pulmatrix \/ Celerion"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"PYC-003","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Qu Biologics Inc","sponsor":"The National Research Council of Canada Industrial Research Assistance Program","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Vaccine","year":"2022","type":"Inapplicable","leadProduct":"Qbkpn SSI","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Qu Biologics Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Qu Biologics Inc \/ The National Research Council of Canada Industrial Research Assistance Program","highestDevelopmentStatusID":"8","companyTruncated":"Qu Biologics Inc \/ The National Research Council of Canada Industrial Research Assistance Program"},{"orgOrder":0,"company":"Qu Biologics Inc","sponsor":"National Research Council of Canada Industrial Research Assistance Program","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Vaccine","year":"2022","type":"Funding","leadProduct":"QBKPN SSI","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Qu Biologics Inc","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Injection","sponsorNew":"Qu Biologics Inc \/ National Research Council of Canada Industrial Research Assistance Program","highestDevelopmentStatusID":"8","companyTruncated":"Qu Biologics Inc \/ National Research Council of Canada Industrial Research Assistance Program"},{"orgOrder":0,"company":"River 3 Renal Corp.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"R3R01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"River 3 Renal Corp.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"River 3 Renal Corp. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"River 3 Renal Corp. \/ Undisclosed"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2015","type":"Inapplicable","leadProduct":"rAAV2tYF-CB-hRS1","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"TeamedOn","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"rAAV2tYF-CB-hRS1","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ TeamedOn","highestDevelopmentStatusID":"1","companyTruncated":"Agtc \/ TeamedOn"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"rAAV2tYF-GRK1-hRPGRco","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"rAAVrh8-HexA\/HexB","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sio Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Sio Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Ractigen Therapeutics","sponsor":"SDIC CS Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Oligonucleotide","year":"2022","type":"Series A Financing","leadProduct":"RAG-06","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Ractigen Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Ractigen Therapeutics \/ SDIC CS Capital","highestDevelopmentStatusID":"5","companyTruncated":"Ractigen Therapeutics \/ SDIC CS Capital"},{"orgOrder":0,"company":"Ractigen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"RAG-18","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ractigen Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ractigen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Ractigen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ractigen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"RAG-18","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ractigen Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ractigen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Ractigen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"4BIO Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Financing","leadProduct":"Ray-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ray Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Injection","sponsorNew":"Ray Therapeutics \/ 4BIO Capital","highestDevelopmentStatusID":"4","companyTruncated":"Ray Therapeutics \/ 4BIO Capital"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"Forge Biologics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Partnership","leadProduct":"Ray-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ray Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ray Therapeutics \/ Forge Biologics","highestDevelopmentStatusID":"4","companyTruncated":"Ray Therapeutics \/ Forge Biologics"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Funding","leadProduct":"Ray-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ray Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ray Therapeutics \/ California Institute for Regenerative Medicine","highestDevelopmentStatusID":"4","companyTruncated":"Ray Therapeutics \/ California Institute for Regenerative Medicine"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"RCT2100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"RCT2100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"RCT2100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"RCT2100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"St. James's Hospital","sponsor":"Baxter Healthcare Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Recombinant Antihemophilic Factor","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"St. James's Hospital","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"St. James's Hospital \/ Baxter Healthcare Corporation","highestDevelopmentStatusID":"11","companyTruncated":"St. James's Hospital \/ Baxter Healthcare Corporation"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Recombinant Antihemophilic Factor","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Recombinant Factor Ix-Albumin Fusion Protein","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Recombinant Factor Ix-Albumin Fusion Protein","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Recombinant Factor Ix-Albumin Fusion Protein","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Recombinant Factor VIIa BI","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Green Cross Lab Cell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Recombinant Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Green Cross Lab Cell","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Green Cross Lab Cell \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Green Cross Lab Cell \/ Undisclosed"},{"orgOrder":0,"company":"Chia Tai Tianqing Pharmaceutical Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"Recombinant Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chia Tai Tianqing Pharmaceutical Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Recombinant Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Recombinant Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Recombinant Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Recombinant Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Recombinant Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Recombinant Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein-drug Conjugate","year":"2010","type":"Inapplicable","leadProduct":"Recombinant Factor VIII Fc","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Recombinant Fusion Protein Linking Coagulation Factor Ix","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Greenovation Biotech","sponsor":"FGK Clinical Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Enzyme","year":"2016","type":"Inapplicable","leadProduct":"Recombinant Human Alpha-Galactosidase A","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Greenovation Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Greenovation Biotech \/ FGK Clinical Research","highestDevelopmentStatusID":"6","companyTruncated":"Greenovation Biotech \/ FGK Clinical Research"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2013","type":"Inapplicable","leadProduct":"Recombinant Human Coagulation Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Recombinant Human Coagulation Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2013","type":"Inapplicable","leadProduct":"Recombinant Human Coagulation Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"GenScript ProBio","sponsor":"Neoletix","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Collaboration","leadProduct":"Recombinant Human Coagulation Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"GenScript ProBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GenScript ProBio \/ Neoletix","highestDevelopmentStatusID":"4","companyTruncated":"GenScript ProBio \/ Neoletix"},{"orgOrder":0,"company":"Chia Tai Tianqing Pharmaceutical Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Recombinant Human Coagulation FVIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chia Tai Tianqing Pharmaceutical Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed"},{"orgOrder":0,"company":"GeneScience Pharmaceuticals Co Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Recombinant Human FSH-CTP Fusion Protein","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GeneScience Pharmaceuticals Co Ltd","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneScience Pharmaceuticals Co Ltd \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"GeneScience Pharmaceuticals Co Ltd \/ Undisclosed"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Recombinant Human Heparan N-Sulfatase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GC Biopharma \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"GC Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Novel Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Recombinant Human Heparan N-Sulfatase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GC Biopharma \/ Novel Pharma","highestDevelopmentStatusID":"6","companyTruncated":"GC Biopharma \/ Novel Pharma"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Novel Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Recombinant Human Heparan N-Sulfatase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GC Biopharma \/ Novel Pharma","highestDevelopmentStatusID":"6","companyTruncated":"GC Biopharma \/ Novel Pharma"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Novel Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Recombinant Human Heparan N-Sulfatase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GC Biopharma \/ Novel Pharma","highestDevelopmentStatusID":"6","companyTruncated":"GC Biopharma \/ Novel Pharma"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2014","type":"Inapplicable","leadProduct":"Recombinant Human Heparan N-Sulfatase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Recombinant Human Heparan N-Sulfatase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"Recombinant Human HNS","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Recombinant, Pegylated Antihemophilic Factor","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Erytech Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Red Blood Cell-Encapsulated Arginine Deiminase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Erytech Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Erytech Pharma \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Erytech Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Virica Biotech","sponsor":"Innovation, Science and Economic Development Canada","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Cell & Gene Therapy","year":"2022","type":"Collaboration","leadProduct":"Re-engineered AAV-LPL","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Virica Biotech","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Virica Biotech \/ Innovation, Science and Economic Development Canada","highestDevelopmentStatusID":"1","companyTruncated":"Virica Biotech \/ Innovation, Science and Economic Development Canada"},{"orgOrder":0,"company":"Austin Institute for Clinical Research","sponsor":"The HydraFacial Company","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"ReGen-GF","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Austin Institute for Clinical Research","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Serum","sponsorNew":"Austin Institute for Clinical Research \/ The HydraFacial Company","highestDevelopmentStatusID":"11","companyTruncated":"Austin Institute for Clinical Research \/ The HydraFacial Company"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Intellia Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"REGV131","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regeneron Pharmaceuticals \/ Intellia Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Regeneron Pharmaceuticals \/ Intellia Therapeutics"},{"orgOrder":0,"company":"Repair Biotechnologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"REP-0003","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Repair Biotechnologies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Repair Biotechnologies \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Repair Biotechnologies \/ Undisclosed"},{"orgOrder":0,"company":"Repair Biotechnologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"REP-0003","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Repair Biotechnologies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Repair Biotechnologies \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Repair Biotechnologies \/ Undisclosed"},{"orgOrder":0,"company":"Cornell University","sponsor":"reVision Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Licensing Agreement","leadProduct":"REV-0100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Cornell University","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cornell University \/ reVision Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Cornell University \/ reVision Therapeutics"},{"orgOrder":0,"company":"reVision Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"REV-0100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"reVision Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"reVision Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"reVision Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Bellicum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"Rivogenlecleucel","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Bellicum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bellicum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Bellicum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"923rd Hospital of Joint Logistics Support Force of PLA","sponsor":"Reforgene Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"RM-004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"923rd Hospital of Joint Logistics Support Force of PLA","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"923rd Hospital of Joint Logistics Support Force of PLA \/ Reforgene Medicine","highestDevelopmentStatusID":"6","companyTruncated":"923rd Hospital of Joint Logistics Support Force of PLA \/ Reforgene Medicine"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"RO5186582","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"RO6885247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"RO7204239","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"RO7204239","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"Respirion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"RSP-1502","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Respirion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Respirion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Respirion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"RTx-015","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Ray Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ray Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Ray Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Adverum Biotechnologies","sponsor":"Ray Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Licensing Agreement","leadProduct":"AAV.7m8","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Adverum Biotechnologies","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Adverum Biotechnologies \/ Ray Therapeutics","highestDevelopmentStatusID":"5","companyTruncated":"Adverum Biotechnologies \/ Ray Therapeutics"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Funding","leadProduct":"RTx-015","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Ray Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Injection","sponsorNew":"Ray Therapeutics \/ California Institute for Regenerative Medicine","highestDevelopmentStatusID":"6","companyTruncated":"Ray Therapeutics \/ California Institute for Regenerative Medicine"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"Forge Biologics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Collaboration","leadProduct":"RTx-015","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Ray Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ray Therapeutics \/ Forge Biologics","highestDevelopmentStatusID":"6","companyTruncated":"Ray Therapeutics \/ Forge Biologics"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"Novo Holding","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Series A Financing","leadProduct":"RTx-015","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Ray Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"Ray Therapeutics \/ Novo Holding","highestDevelopmentStatusID":"6","companyTruncated":"Ray Therapeutics \/ Novo Holding"},{"orgOrder":0,"company":"Rubius Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"RTX-134","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Rubius Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rubius Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Rubius Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Rubius Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"RTX-134","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Rubius Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rubius Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Rubius Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"SolAeroMed Inc.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"S-1226","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SolAeroMed Inc.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"SolAeroMed Inc. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SolAeroMed Inc. \/ Undisclosed"},{"orgOrder":0,"company":"Sarcomatrix","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Funding","leadProduct":"S-969","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Sarcomatrix","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sarcomatrix \/ National Institutes of Health","highestDevelopmentStatusID":"5","companyTruncated":"Sarcomatrix \/ National Institutes of Health"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"SAF-301","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"SAF-301","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Salanersen","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"University of Virginia","sponsor":"CSL Behring | Jeffrey Modell Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vaccine","year":"2019","type":"Inapplicable","leadProduct":"Salmonella Typhi Polysaccharide Vaccine","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"University of Virginia","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Virginia \/ CSL Behring | Jeffrey Modell Foundation","highestDevelopmentStatusID":"6","companyTruncated":"University of Virginia \/ CSL Behring | Jeffrey Modell Foundation"},{"orgOrder":0,"company":"Dr. Reddy's Laboratories","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Dr. Reddy\\'s Laboratories","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Solution","sponsorNew":"Dr. Reddy\\'s Laboratories \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Dr. Reddy\\'s Laboratories \/ Undisclosed"},{"orgOrder":0,"company":"Dr. Reddy's Laboratories","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Dr. Reddy\\'s Laboratories","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Solution","sponsorNew":"Dr. Reddy\\'s Laboratories \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Dr. Reddy\\'s Laboratories \/ Undisclosed"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Relief Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Relief Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"SAR439459","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Solution","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SAR444836","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Medicinova","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SAR444836","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Medicinova","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Medicinova \/ Sanofi","highestDevelopmentStatusID":"7","companyTruncated":"Medicinova \/ Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"SAR446268","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"SAR447537","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"SAR447537","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Bloom Burton Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Public Offering","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Bloom Burton Securities","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Bloom Burton Securities"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SB-007","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SpliceBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SpliceBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SpliceBio \/ Undisclosed"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SB-007","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SpliceBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SpliceBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SpliceBio \/ Undisclosed"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"SB-007","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SpliceBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SpliceBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SpliceBio \/ Undisclosed"},{"orgOrder":0,"company":"Micelle BioPharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"SC411","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Micelle BioPharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Micelle BioPharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Micelle BioPharma \/ Undisclosed"},{"orgOrder":0,"company":"Invenux","sponsor":"SUNY Downstate Medical Center","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"SCD-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Invenux","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Invenux \/ SUNY Downstate Medical Center","highestDevelopmentStatusID":"6","companyTruncated":"Invenux \/ SUNY Downstate Medical Center"},{"orgOrder":0,"company":"Siena Biotech S.p.A.","sponsor":"Seventh Framework Programme (FP7) | European Huntington's Disease Network","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"SEN0014196","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Siena Biotech S.p.A.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Immediate Release","sponsorNew":"Siena Biotech S.p.A. \/ Seventh Framework Programme (FP7) | European Huntington's Disease Network","highestDevelopmentStatusID":"8","companyTruncated":"Siena Biotech S.p.A. \/ Seventh Framework Programme (FP7) | European Huntington's Disease Network"},{"orgOrder":0,"company":"Siena Biotech S.p.A.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"SEN0014196","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Siena Biotech S.p.A.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Siena Biotech S.p.A. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Siena Biotech S.p.A. \/ Undisclosed"},{"orgOrder":0,"company":"Siena Biotech S.p.A.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"SEN0014196","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Siena Biotech S.p.A.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Immediate Release","sponsorNew":"Siena Biotech S.p.A. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Siena Biotech S.p.A. \/ Undisclosed"},{"orgOrder":0,"company":"Siena Biotech S.p.A.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"SEN0014196","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Siena Biotech S.p.A.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Siena Biotech S.p.A. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Siena Biotech S.p.A. \/ Undisclosed"},{"orgOrder":0,"company":"Somagenetix","sponsor":"Vi Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2024","type":"Series A Financing","leadProduct":"SGX-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Somagenetix","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"Somagenetix \/ Vi Partners","highestDevelopmentStatusID":"4","companyTruncated":"Somagenetix \/ Vi Partners"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"SHP648","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Sigilon Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"SIG-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eli Lilly \/ Sigilon Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Eli Lilly \/ Sigilon Therapeutics"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Public Offering","leadProduct":"SIG-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sigilon Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Sigilon Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Sigilon Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Canada Pension Plan Investment Board","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Series B Financing","leadProduct":"SIG-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sigilon Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Sigilon Therapeutics \/ Canada Pension Plan Investment Board","highestDevelopmentStatusID":"7","companyTruncated":"Sigilon Therapeutics \/ Canada Pension Plan Investment Board"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"SIG-005","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sigilon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sigilon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sigilon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"SIG-005","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sigilon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sigilon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sigilon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"SIG-005","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sigilon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sigilon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sigilon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SIG-005","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sigilon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sigilon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sigilon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"SK Plasma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"SKP-0141","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"SK Plasma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SK Plasma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"SK Plasma \/ Undisclosed"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"SKY-0515","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Skyhawk Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Skyhawk Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SKY-0515","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Skyhawk Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Skyhawk Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SKY-0515","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Skyhawk Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Skyhawk Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SKY-0515","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Skyhawk Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Skyhawk Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"OmniaBio","sponsor":"Somite Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Collaboration","leadProduct":"SMT-M01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"OmniaBio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"OmniaBio \/ Somite Therapeutics","highestDevelopmentStatusID":"5","companyTruncated":"OmniaBio \/ Somite Therapeutics"},{"orgOrder":0,"company":"McQuade Center for Strategic Research and Development","sponsor":"Sentinel Oncology","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Collaboration","leadProduct":"SOL784","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"McQuade Center for Strategic Research and Development","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"McQuade Center for Strategic Research and Development \/ Sentinel Oncology","highestDevelopmentStatusID":"4","companyTruncated":"McQuade Center for Strategic Research and Development \/ Sentinel Oncology"},{"orgOrder":0,"company":"Spirovant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SP-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spirovant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Spirovant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spirovant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"SPK-10001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Spark Therapeutics, Inc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spark Therapeutics, Inc \/ Undisclosed"},{"orgOrder":0,"company":"SpliSense","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"SPL84","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SpliSense","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"SpliSense \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SpliSense \/ Undisclosed"},{"orgOrder":0,"company":"SpliSense","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"SPL84","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SpliSense","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"SpliSense \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SpliSense \/ Undisclosed"},{"orgOrder":0,"company":"SpliSense","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Oligonucleotide","year":"2024","type":"Funding","leadProduct":"SPL84","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SpliSense","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Solution for Nebulizer","sponsorNew":"SpliSense \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"8","companyTruncated":"SpliSense \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"SpliSense","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"SPL84","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SpliSense","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"SpliSense \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SpliSense \/ Undisclosed"},{"orgOrder":0,"company":"SpliSense","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"SPL84","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SpliSense","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"SpliSense \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SpliSense \/ Undisclosed"},{"orgOrder":0,"company":"SpliSense","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"SPL84","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SpliSense","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"SpliSense \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SpliSense \/ Undisclosed"},{"orgOrder":0,"company":"SpliSense","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"SPL84","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SpliSense","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"SpliSense \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SpliSense \/ Undisclosed"},{"orgOrder":0,"company":"Spyryx Biosciences, Inc.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"SPX-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spyryx Biosciences, Inc.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Spyryx Biosciences, Inc. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spyryx Biosciences, Inc. \/ Undisclosed"},{"orgOrder":0,"company":"Spyryx Biosciences, Inc.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"SPX-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spyryx Biosciences, Inc.","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Spyryx Biosciences, Inc. \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Spyryx Biosciences, Inc. \/ Undisclosed"},{"orgOrder":0,"company":"Sqy Therapeutics","sponsor":"Biotrial","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"SQY51","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sqy Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sqy Therapeutics \/ Biotrial","highestDevelopmentStatusID":"7","companyTruncated":"Sqy Therapeutics \/ Biotrial"},{"orgOrder":0,"company":"Equilibra Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"SR604","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Equilibra Bioscience","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Equilibra Bioscience \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Equilibra Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Medera","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"SRD-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Medera","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Medera \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Medera \/ Undisclosed"},{"orgOrder":0,"company":"Jiangsu Gensciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"SS109","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jiangsu Gensciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jiangsu Gensciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jiangsu Gensciences \/ Undisclosed"},{"orgOrder":0,"company":"Jiangsu Gensciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"SS109","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jiangsu Gensciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jiangsu Gensciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jiangsu Gensciences \/ Undisclosed"},{"orgOrder":0,"company":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","sponsor":"Beijing Novikang Medical Technology","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"STSP-0601","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Staidson (Beijing) Biopharmaceuticals Co., Ltd \/ Beijing Novikang Medical Technology","highestDevelopmentStatusID":"8","companyTruncated":"Staidson (Beijing) Biopharmaceuticals Co., Ltd \/ Beijing Novikang Medical Technology"},{"orgOrder":0,"company":"Jiangsu BioJeTay Biotechnology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"STSP-0601","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Jiangsu BioJeTay Biotechnology","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Jiangsu BioJeTay Biotechnology \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Jiangsu BioJeTay Biotechnology \/ Undisclosed"},{"orgOrder":0,"company":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"STSP-0601","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Staidson (Beijing) Biopharmaceuticals Co., Ltd \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Staidson (Beijing) Biopharmaceuticals Co., Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"STSP-0601","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Staidson (Beijing) Biopharmaceuticals Co., Ltd \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Staidson (Beijing) Biopharmaceuticals Co., Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"STSP-0601","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Staidson (Beijing) Biopharmaceuticals Co., Ltd","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Staidson (Beijing) Biopharmaceuticals Co., Ltd \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Staidson (Beijing) Biopharmaceuticals Co., Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Bio Products Laboratory","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"Subgam","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Bio Products Laboratory","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bio Products Laboratory \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Bio Products Laboratory \/ Undisclosed"},{"orgOrder":0,"company":"Laboratorios Ordesa","sponsor":"Quantum Experimental | Peruvian Clinical Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"Symbiotic Formula","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Laboratorios Ordesa","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Laboratorios Ordesa \/ Quantum Experimental | Peruvian Clinical Research","highestDevelopmentStatusID":"1","companyTruncated":"Laboratorios Ordesa \/ Quantum Experimental | Peruvian Clinical Research"},{"orgOrder":0,"company":"Synlogic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"SYNB1618","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Synlogic \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Synlogic \/ Undisclosed"},{"orgOrder":0,"company":"Synlogic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"SYNB1618","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Synlogic \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Synlogic \/ Undisclosed"},{"orgOrder":0,"company":"Synlogic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"SYNB1618","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Synlogic \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Synlogic \/ Undisclosed"},{"orgOrder":0,"company":"Nutricia Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Dietary Supplement","year":"2017","type":"Inapplicable","leadProduct":"Synergy","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Nutricia Inc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Nutricia Inc \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Nutricia Inc \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"TAK-577","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"TAK-625","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"TAK-672","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"TAK-771","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"TAK-771","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Takeda Pharmaceutical | Baxalta Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"TAK-881","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Takeda Pharmaceutical | Baxalta Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Takeda Pharmaceutical | Baxalta Pharmaceutical"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"TAK-881","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"TAK-881","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Taiga Biotechnologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"TBX-1400","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Taiga Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Taiga Biotechnologies \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Taiga Biotechnologies \/ Undisclosed"},{"orgOrder":0,"company":"Sanford Burnham Prebys Medical Discovery Institute","sponsor":"Soft Bones","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Funding","leadProduct":"Tissue Non-Specific Alkaline Phosphatase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Sanford Burnham Prebys Medical Discovery Institute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanford Burnham Prebys Medical Discovery Institute \/ Soft Bones","highestDevelopmentStatusID":"4","companyTruncated":"Sanford Burnham Prebys Medical Discovery Institute \/ Soft Bones"},{"orgOrder":0,"company":"Chia Tai Tianqing Pharmaceutical Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"TQ05105","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Chia Tai Tianqing Pharmaceutical Group","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed"},{"orgOrder":0,"company":"Chia Tai Tianqing Pharmaceutical Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"TQ-B3234","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Chia Tai Tianqing Pharmaceutical Group","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed"},{"orgOrder":0,"company":"Chia Tai Tianqing Pharmaceutical Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"TQG203","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Chia Tai Tianqing Pharmaceutical Group","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Chia Tai Tianqing Pharmaceutical Group \/ Undisclosed"},{"orgOrder":0,"company":"Corporacion Parc Tauli","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Tridocosahexaenoine","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Corporacion Parc Tauli","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Corporacion Parc Tauli \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Corporacion Parc Tauli \/ Undisclosed"},{"orgOrder":0,"company":"PRG Science & Technology Co., Ltd.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Trineumin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PRG Science & Technology Co., Ltd.","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PRG Science & Technology Co., Ltd. \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PRG Science & Technology Co., Ltd. \/ Undisclosed"},{"orgOrder":0,"company":"UT Southwestern Medical Center","sponsor":"Taysha Gene Therapies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Expanded Collaboration","leadProduct":"TSHA-112","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"UT Southwestern Medical Center","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"UT Southwestern Medical Center \/ Taysha Gene Therapies","highestDevelopmentStatusID":"2","companyTruncated":"UT Southwestern Medical Center \/ Taysha Gene Therapies"},{"orgOrder":0,"company":"Triplet Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"TTX-3360","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Triplet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Triplet Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Triplet Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Tiumbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"TU7710","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tiumbio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tiumbio \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Tiumbio \/ Undisclosed"},{"orgOrder":0,"company":"Tiumbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"TU7710","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tiumbio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tiumbio \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Tiumbio \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2019","type":"Inapplicable","leadProduct":"TYR sphere","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2021","type":"Inapplicable","leadProduct":"TYR sphere","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Applied Science & Performance Institute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Plant Extract\/Herbal","year":"2025","type":"Inapplicable","leadProduct":"CAS 501-94-0","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Applied Science & Performance Institute","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oil","sponsorNew":"Applied Science & Performance Institute \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Applied Science & Performance Institute \/ Undisclosed"},{"orgOrder":0,"company":"Restem","sponsor":"SOLVE FSHD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Umbilical Cord Lining-Derived Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Restem","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Restem \/ SOLVE FSHD","highestDevelopmentStatusID":"6","companyTruncated":"Restem \/ SOLVE FSHD"},{"orgOrder":0,"company":"Sclnow Biotechnology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"Umbilical Cord Mesenchymal Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sclnow Biotechnology","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sclnow Biotechnology \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sclnow Biotechnology \/ Undisclosed"},{"orgOrder":0,"company":"PT Prodia StemCell Indonesia","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDONESIA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Umbilical Cord Mesenchymal Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"PT Prodia StemCell Indonesia","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PT Prodia StemCell Indonesia \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"PT Prodia StemCell Indonesia \/ Undisclosed"},{"orgOrder":0,"company":"PT Prodia StemCell Indonesia","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDONESIA","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Umbilical Cord Mesenchymal Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"PT Prodia StemCell Indonesia","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PT Prodia StemCell Indonesia \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"PT Prodia StemCell Indonesia \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Velmanase Alpha","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Chiesi Group \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Chiesi Group \/ Undisclosed"},{"orgOrder":0,"company":"ViGeneron","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2021","type":"Collaboration","leadProduct":"VG801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ViGeneron","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ViGeneron \/ Biogen","highestDevelopmentStatusID":"4","companyTruncated":"ViGeneron \/ Biogen"},{"orgOrder":0,"company":"ViGeneron","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"VG801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"ViGeneron","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ViGeneron \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"ViGeneron \/ Undisclosed"},{"orgOrder":0,"company":"ViGeneron","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2022","type":"Collaboration","leadProduct":"VG801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ViGeneron","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ViGeneron \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"ViGeneron \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"WuXi Biologics","sponsor":"ViGeneron","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2020","type":"Partnership","leadProduct":"VG901","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"WuXi Biologics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"WuXi Biologics \/ ViGeneron","highestDevelopmentStatusID":"5","companyTruncated":"WuXi Biologics \/ ViGeneron"},{"orgOrder":0,"company":"ViGeneron","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"VG901","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ViGeneron","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ViGeneron \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ViGeneron \/ Undisclosed"},{"orgOrder":0,"company":"ViGeneron","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"VG901","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ViGeneron","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ViGeneron \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ViGeneron \/ Undisclosed"},{"orgOrder":0,"company":"ViGeneron","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"VG901","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"ViGeneron","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ViGeneron \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ViGeneron \/ Undisclosed"},{"orgOrder":0,"company":"Vega Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"VGA039","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vega Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Vega Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vega Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vega Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"VGA039","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vega Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Vega Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vega Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Star Therapeutics","sponsor":"Sofinnova Investments","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Series C Financing","leadProduct":"VGA039","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Star Therapeutics","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Injection","sponsorNew":"Star Therapeutics \/ Sofinnova Investments","highestDevelopmentStatusID":"10","companyTruncated":"Star Therapeutics \/ Sofinnova Investments"},{"orgOrder":0,"company":"Vega Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"VGA039","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vega Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Vega Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vega Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Star Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"VGA039","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Star Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Star Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Star Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vega Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"VGA039","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vega Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vega Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Vega Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine","sponsor":"Shanghai Vitalgen BioPharma Co., Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"VGN-R08b","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine \/ Shanghai Vitalgen BioPharma Co., Ltd.","highestDevelopmentStatusID":"6","companyTruncated":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine \/ Shanghai Vitalgen BioPharma Co., Ltd."},{"orgOrder":0,"company":"Shanghai Jiaotong University School of Medicine","sponsor":"Shanghai Vitalgen BioPharma Co., Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"VGN-R09b","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shanghai Jiaotong University School of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Shanghai Jiaotong University School of Medicine \/ Shanghai Vitalgen BioPharma Co., Ltd.","highestDevelopmentStatusID":"6","companyTruncated":"Shanghai Jiaotong University School of Medicine \/ Shanghai Vitalgen BioPharma Co., Ltd."},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"VMCO-I","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"vMCO-I","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"CAS 830-96-6","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"Vital Therapeutics & Formulations Pvt Ltd","sponsor":"Cambrian Biopharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Cell & Gene Therapy","year":"2021","type":"Series A Financing","leadProduct":"VTA-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Vital Therapeutics & Formulations Pvt Ltd","amount2":0.029999999999999999,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Vital Therapeutics & Formulations Pvt Ltd \/ Cambrian Biopharma","highestDevelopmentStatusID":"5","companyTruncated":"Vital Therapeutics & Formulations Pvt Ltd \/ Cambrian Biopharma"},{"orgOrder":0,"company":"Vita Therapeutics","sponsor":"Cambrian Biopharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Series B Financing","leadProduct":"VTA-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Vita Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Vita Therapeutics \/ Cambrian Biopharma","highestDevelopmentStatusID":"5","companyTruncated":"Vita Therapeutics \/ Cambrian Biopharma"},{"orgOrder":0,"company":"Vita Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"VTA-110","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vita Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vita Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Vita Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Mirum Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Collaboration","leadProduct":"VTX-803","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ Mirum Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ Mirum Pharmaceuticals"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"VTX-803","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"VX-407","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"VX-407","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"VX-407","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"VX-407","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"VX-828","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"VX-828","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Neurocrine Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Collaboration","leadProduct":"VY-FXN01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Voyager Therapeutics, Inc","amount2":1.8700000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":1.8700000000000001,"dosageForm":"Injection","sponsorNew":"Voyager Therapeutics, Inc \/ Neurocrine Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"Voyager Therapeutics, Inc \/ Neurocrine Biosciences"},{"orgOrder":0,"company":"Adaptive Phage Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2023","type":"Inapplicable","leadProduct":"WRAIR_EPa11","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Adaptive Phage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Adaptive Phage Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Adaptive Phage Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"National Institutes of Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2022","type":"Inapplicable","leadProduct":"WRAIR_EPa11","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"National Institutes of Health","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"National Institutes of Health \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"National Institutes of Health \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Public Offering","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ J.P. Morgan"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Public Offering","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ J.P. Morgan"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Genethon","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"X Vivo Gene Therapy","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Genethon \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Genethon \/ Undisclosed"},{"orgOrder":0,"company":"Genethon","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"X Vivo Gene Therapy","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Genethon \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Genethon \/ Undisclosed"},{"orgOrder":0,"company":"Indiana University","sponsor":"Global Health Uganda | Makerere University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Zinc Sulphate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Indiana University","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Indiana University \/ Global Health Uganda | Makerere University","highestDevelopmentStatusID":"9","companyTruncated":"Indiana University \/ Global Health Uganda | Makerere University"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Almirall","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Licensing Agreement","leadProduct":"ZKN-013","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Eloxx Pharmaceuticals","amount2":0.46999999999999997,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.46999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Eloxx Pharmaceuticals \/ Almirall","highestDevelopmentStatusID":"5","companyTruncated":"Eloxx Pharmaceuticals \/ Almirall"},{"orgOrder":0,"company":"Zhongmou Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"ZM-01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Zhongmou Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zhongmou Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Zhongmou Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Zhongmou Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"ZM-02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Zhongmou Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zhongmou Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Zhongmou Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Chigenovo","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"ZVS203e","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Chigenovo","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chigenovo \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Chigenovo \/ Undisclosed"},{"orgOrder":0,"company":"Shenzhen Hemogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"\u0392-Globin Restored Autologous Hematopoietic Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shenzhen Hemogen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Shenzhen Hemogen \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Shenzhen Hemogen \/ Undisclosed"},{"orgOrder":0,"company":"Shenzhen Hemogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"\u0392-Globin Restored Autologous Hematopoietic Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shenzhen Hemogen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Shenzhen Hemogen \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Shenzhen Hemogen \/ Undisclosed"},{"orgOrder":0,"company":"Shenzhen Hemogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"\u0392-Globin Restored Autologous Hematopoietic Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shenzhen Hemogen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Shenzhen Hemogen \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Shenzhen Hemogen \/ Undisclosed"},{"orgOrder":0,"company":"APR Applied Pharma Research","sponsor":"Relief Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Acquisition","leadProduct":"Amino Acids Mixture","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"APR Applied Pharma Research","amount2":0.070000000000000007,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Granules, Controlled Release","sponsorNew":"APR Applied Pharma Research \/ Relief Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"APR Applied Pharma Research \/ Relief Therapeutics"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Amino Acids Mixture","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules, Controlled Release","sponsorNew":"Relief Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Relief Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"University Hospital, Lille","sponsor":"SHIRE PLC","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"IgHy10","moa":"Unknown","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"University Hospital, Lille","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University Hospital, Lille \/ SHIRE PLC","highestDevelopmentStatusID":"11","companyTruncated":"University Hospital, Lille \/ SHIRE PLC"},{"orgOrder":0,"company":"Wellstat Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Triacetyluridine","moa":"Uridine","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Wellstat Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Wellstat Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Wellstat Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Ultevursen","moa":"usherin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Laboratoires Thea","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Laboratoires Thea \/ Sepul Bio","highestDevelopmentStatusID":"9","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Ultevursen","moa":"usherin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Laboratoires Thea","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Laboratoires Thea \/ Sepul Bio","highestDevelopmentStatusID":"9","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"QR-421a","moa":"Usherin messenger RNA (USH2A mRNA)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Laboratoires Thea","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Laboratoires Thea \/ Sepul Bio","highestDevelopmentStatusID":"9","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"QR-421a","moa":"Usherin messenger RNA (USH2A mRNA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ProQR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"QR-421a","moa":"Usherin messenger RNA (USH2A mRNA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ProQR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Mission Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"MTX652","moa":"USP-30","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Mission Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mission Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Mission Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Summit Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Ezutromid","moa":"Utrophin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Summit Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Summit Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Summit Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Summit Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Ezutromid","moa":"Utrophin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Summit Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Summit Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Summit Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Summit Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Ezutromid","moa":"Utrophin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Summit Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Summit Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Summit Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"University of Alabama at Birmingham","sponsor":"Genentech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2013","type":"Inapplicable","leadProduct":"Bevacizumab","moa":"Vascular endothelial growth factor A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of Alabama at Birmingham","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"University of Alabama at Birmingham \/ Genentech","highestDevelopmentStatusID":"8","companyTruncated":"University of Alabama at Birmingham \/ Genentech"},{"orgOrder":0,"company":"University of California, San Francisco","sponsor":"Merck & Co","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Bevacizumab","moa":"Vascular endothelial growth factor A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of California, San Francisco","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of California, San Francisco \/ Merck & Co","highestDevelopmentStatusID":"8","companyTruncated":"University of California, San Francisco \/ Merck & Co"},{"orgOrder":0,"company":"University of California, San Diego","sponsor":"Genentech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2011","type":"Inapplicable","leadProduct":"Ranibizumab","moa":"Vascular endothelial growth factor A","graph1":"Genetic Disease","graph2":"Phase I","graph3":"University of California, San Diego","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"University of California, San Diego \/ Genentech","highestDevelopmentStatusID":"6","companyTruncated":"University of California, San Diego \/ Genentech"},{"orgOrder":0,"company":"NYU Langone Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Axitinib","moa":"Vascular endothelial growth factor receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NYU Langone Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NYU Langone Health \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"NYU Langone Health \/ Undisclosed"},{"orgOrder":0,"company":"Gabrail Cancer Center Research","sponsor":"Children's Healthcare of Atlanta | National Center for Complementary and Integrative Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Amino Acid","year":"2015","type":"Inapplicable","leadProduct":"L-Arginine","moa":"Vasopressin receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Gabrail Cancer Center Research","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gabrail Cancer Center Research \/ Children's Healthcare of Atlanta | National Center for Complementary and Integrative Health","highestDevelopmentStatusID":"7","companyTruncated":"Gabrail Cancer Center Research \/ Children's Healthcare of Atlanta | National Center for Complementary and Integrative Health"},{"orgOrder":0,"company":"The Hospital for Sick Children","sponsor":"Canadian Hemophilia Society | Unity Health Toronto | Nationwide Children\u2019s Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Desmopressin Acetate","moa":"Vasopressin receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"The Hospital for Sick Children","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"The Hospital for Sick Children \/ Canadian Hemophilia Society | Unity Health Toronto | Nationwide Children\u2019s Hospital","highestDevelopmentStatusID":"11","companyTruncated":"The Hospital for Sick Children \/ Canadian Hemophilia Society | Unity Health Toronto | Nationwide Children\u2019s Hospital"},{"orgOrder":0,"company":"Pontificia Universidad Catolica de Chile","sponsor":"Ferring Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHILE","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Desmopressin Acetate","moa":"Vasopressin receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pontificia Universidad Catolica de Chile","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pontificia Universidad Catolica de Chile \/ Ferring Pharmaceuticals","highestDevelopmentStatusID":"11","companyTruncated":"Pontificia Universidad Catolica de Chile \/ Ferring Pharmaceuticals"},{"orgOrder":0,"company":"Palladio Zannini Industrie","sponsor":"Samsara BioCapital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2020","type":"Series B Financing","leadProduct":"Lixivaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palladio Zannini Industrie","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Capsule","sponsorNew":"Palladio Zannini Industrie \/ Samsara BioCapital","highestDevelopmentStatusID":"10","companyTruncated":"Palladio Zannini Industrie \/ Samsara BioCapital"},{"orgOrder":0,"company":"Palladio Biosciences","sponsor":"Centessa Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Lixivaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palladio Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Palladio Biosciences \/ Centessa Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Palladio Biosciences \/ Centessa Pharmaceuticals"},{"orgOrder":0,"company":"Palladio Biosciences","sponsor":"Centessa Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Lixivaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palladio Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Palladio Biosciences \/ Centessa Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Palladio Biosciences \/ Centessa Pharmaceuticals"},{"orgOrder":0,"company":"Palladio Biosciences","sponsor":"Centessa Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Lixivaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palladio Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Palladio Biosciences \/ Centessa Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Palladio Biosciences \/ Centessa Pharmaceuticals"},{"orgOrder":0,"company":"Palladio Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Lixivaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palladio Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Palladio Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Palladio Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Lixivaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Centessa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Centessa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Centessa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Palladio Biosciences","sponsor":"Centessa Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Lixivaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palladio Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Palladio Biosciences \/ Centessa Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Palladio Biosciences \/ Centessa Pharmaceuticals"},{"orgOrder":0,"company":"Palladio Biosciences","sponsor":"Centessa Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Lixivaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palladio Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Palladio Biosciences \/ Centessa Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Palladio Biosciences \/ Centessa Pharmaceuticals"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"General Atlantic","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Series A Financing","leadProduct":"Lixivaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Centessa Pharmaceuticals","amount2":0.25,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.25,"dosageForm":"Capsule","sponsorNew":"Centessa Pharmaceuticals \/ General Atlantic","highestDevelopmentStatusID":"10","companyTruncated":"Centessa Pharmaceuticals \/ General Atlantic"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Lixivaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Centessa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Centessa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Centessa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Public Offering","leadProduct":"Lixivaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Centessa Pharmaceuticals","amount2":0.33000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.33000000000000002,"dosageForm":"Capsule","sponsorNew":"Centessa Pharmaceuticals \/ Morgan Stanley","highestDevelopmentStatusID":"10","companyTruncated":"Centessa Pharmaceuticals \/ Morgan Stanley"},{"orgOrder":0,"company":"Otsuka Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Tolvaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Otsuka Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Otsuka Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Otsuka Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Otsuka Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Tolvaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Otsuka Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Otsuka Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Otsuka Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Tolvaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Immediate Release","sponsorNew":"Lupin Ltd \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Lupin Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Korea Otsuka Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Tolvaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Korea Otsuka Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Korea Otsuka Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Korea Otsuka Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Otsuka Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Tolvaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Otsuka Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Otsuka Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Otsuka Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Otsuka Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Tolvaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Otsuka Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Otsuka Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Otsuka Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Otsuka Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Tolvaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Otsuka Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Otsuka Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Otsuka Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Otsuka Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Tolvaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Otsuka Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Immediate Release","sponsorNew":"Otsuka Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Otsuka Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Otsuka Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Tolvaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Otsuka Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Otsuka Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Otsuka Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Mario Negri Institute for Pharmacological Research","sponsor":"Otsuka Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Tolvaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mario Negri Institute for Pharmacological Research","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mario Negri Institute for Pharmacological Research \/ Otsuka Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Mario Negri Institute for Pharmacological Research \/ Otsuka Pharmaceutical"},{"orgOrder":0,"company":"Autifony Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"AUT00201","moa":"VGKCs","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Autifony Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Autifony Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Autifony Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Cyan Bio","sponsor":"Adge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2022","type":"Partnership","leadProduct":"Elocalcitol","moa":"Vitamin D receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cyan Bio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Cyan Bio \/ Adge Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Cyan Bio \/ Adge Pharmaceuticals"},{"orgOrder":0,"company":"CHU Sainte-Justine","sponsor":"Euro-Pharm","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2018","type":"Inapplicable","leadProduct":"Vitamin D","moa":"Vitamin D receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CHU Sainte-Justine","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CHU Sainte-Justine \/ Euro-Pharm","highestDevelopmentStatusID":"10","companyTruncated":"CHU Sainte-Justine \/ Euro-Pharm"},{"orgOrder":0,"company":"Wake Forest University Health Sciences","sponsor":"Children's Healthcare of Atlanta | Gabrail Cancer Center Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2011","type":"Inapplicable","leadProduct":"Vitamin D","moa":"Vitamin D receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Wake Forest University Health Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Wake Forest University Health Sciences \/ Children's Healthcare of Atlanta | Gabrail Cancer Center Research","highestDevelopmentStatusID":"8","companyTruncated":"Wake Forest University Health Sciences \/ Children's Healthcare of Atlanta | Gabrail Cancer Center Research"},{"orgOrder":0,"company":"Virginia Commonwealth University","sponsor":"CSL Behring","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2010","type":"Inapplicable","leadProduct":"Vitamin D","moa":"Vitamin D receptor","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Virginia Commonwealth University","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Virginia Commonwealth University \/ CSL Behring","highestDevelopmentStatusID":"1","companyTruncated":"Virginia Commonwealth University \/ CSL Behring"},{"orgOrder":0,"company":"University of Colorado, Denver","sponsor":"Cystic Fibrosis Foundation | Yasoo Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2013","type":"Inapplicable","leadProduct":"Aquadeks","moa":"Vitamin\/trace element","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of Colorado, Denver","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Softgel Capsule","sponsorNew":"University of Colorado, Denver \/ Cystic Fibrosis Foundation | Yasoo Health","highestDevelopmentStatusID":"8","companyTruncated":"University of Colorado, Denver \/ Cystic Fibrosis Foundation | Yasoo Health"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"HMB-002","moa":"VMF\/Factor VII level","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Hemab Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"HMB-002","moa":"VMF\/Factor VII level","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hemab Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"University of Minnesota","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Gabapentin","moa":"Voltage-gated calcium channel","graph1":"Genetic Disease","graph2":"Phase II","graph3":"University of Minnesota","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"University of Minnesota \/ Sanofi","highestDevelopmentStatusID":"8","companyTruncated":"University of Minnesota \/ Sanofi"},{"orgOrder":0,"company":"Catalyst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Amifampridine Phosphate","moa":"Voltage-gated potassium channel","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Catalyst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Amifampridine Phosphate","moa":"Voltage-gated potassium channel","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Catalyst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Catalyst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Catalyst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Amifampridine Phosphate","moa":"Voltage-gated potassium channel","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Catalyst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Catalyst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Catalyst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Catalyst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Amifampridine Phosphate","moa":"Voltage-gated potassium channel","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catalyst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Catalyst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Catalyst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"University of Florida","sponsor":"Acorda Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Fampridine","moa":"Voltage-gated potassium channel","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Florida","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Florida \/ Acorda Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"University of Florida \/ Acorda Therapeutics"},{"orgOrder":0,"company":"Solaxa","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Fampridine","moa":"Voltage-gated potassium channel","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Solaxa","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Orally Disintegrating","sponsorNew":"Solaxa \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Solaxa \/ Undisclosed"},{"orgOrder":0,"company":"Unity Health Toronto","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Von Willebrand Factor","moa":"von Willebrand factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Unity Health Toronto","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Unity Health Toronto \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Unity Health Toronto \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Vonicog Alfa","moa":"von Willebrand factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Vonicog Alfa","moa":"von Willebrand factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2014","type":"Inapplicable","leadProduct":"Vonicog Alfa","moa":"von Willebrand factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"Vonicog Alfa","moa":"von Willebrand factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"Baxalta Pharmaceutical \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Vonicog Alfa","moa":"von Willebrand factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Voncento","moa":"von Willebrand factor | Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Immunoforge","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Pemziviptadil","moa":"VPAC2 receptor","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Immunoforge","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Immunoforge \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Immunoforge \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Sanofi","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Sanofi","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Sanofi"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Sanofi","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"||VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Swedish Orphan Biovitrum AB \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Baxalta Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2016","type":"Inapplicable","leadProduct":"Von Willebrand Factor, Recombinant","moa":"VWF","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Takeda Pharmaceutical \/ Baxalta Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Baxalta Pharmaceutical"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Von Willebrand Factor, Recombinant","moa":"VWF","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Baxalta Pharmaceutical \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Sapience Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Irinotecan","moa":"||Wnt\/beta-catenin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sapience Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sapience Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sapience Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Alliance Global Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Public Offering","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Alliance Global Partners","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Alliance Global Partners"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Altasciences Company Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Altasciences Company Inc","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Altasciences Company Inc"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AGTC-501","moa":"X-linked retinitis pigmentosa GTPase regulator (RPGR)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AGTC-501","moa":"X-linked retinitis pigmentosa GTPase regulator (RPGR)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Agtc \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"BIIB112","moa":"X-linked retinitis pigmentosa GTPase regulator (RPGR)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Radiolabeled Compound","year":"2012","type":"Inapplicable","leadProduct":"14-C AT1001","moa":"Zonulin","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Hitachi Global Life Solutions","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Agreement","leadProduct":"EXG-34217","moa":"ZSCAN4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Hitachi Global Life Solutions","highestDevelopmentStatusID":"6","companyTruncated":"Elixirgen Therapeutics \/ Hitachi Global Life Solutions"},{"orgOrder":0,"company":"Orphalan","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Trientine Tetrahydrochloride","moa":"#N\/A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orphalan","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Orphalan \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Orphalan \/ Undisclosed"},{"orgOrder":0,"company":"Orphalan","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Trientine Tetrahydrochloride","moa":"#N\/A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orphalan","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Orphalan \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Orphalan \/ Undisclosed"},{"orgOrder":0,"company":"Orphalan","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Trientine Tetrahydrochloride","moa":"#N\/A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orphalan","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Orphalan \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Orphalan \/ Undisclosed"},{"orgOrder":0,"company":"Orphalan","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Trientine Tetrahydrochloride","moa":"#N\/A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orphalan","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Orphalan \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Orphalan \/ Undisclosed"},{"orgOrder":0,"company":"Orphalan","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Trientine Tetrahydrochloride","moa":"#N\/A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orphalan","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Orphalan \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Orphalan \/ Undisclosed"},{"orgOrder":0,"company":"Tottori University","sponsor":"GC Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Tottori University","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tottori University \/ GC Pharma","highestDevelopmentStatusID":"2","companyTruncated":"Tottori University \/ GC Pharma"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CANbridge Pharmaceuticals \/ UMass Chan Medical School","highestDevelopmentStatusID":"4","companyTruncated":"CANbridge Pharmaceuticals \/ UMass Chan Medical School"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CANbridge Pharmaceuticals \/ UMass Chan Medical School","highestDevelopmentStatusID":"4","companyTruncated":"CANbridge Pharmaceuticals \/ UMass Chan Medical School"},{"orgOrder":0,"company":"GERO.AI","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SINGAPORE","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"GERO.AI","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GERO.AI \/ Pfizer Inc","highestDevelopmentStatusID":"2","companyTruncated":"GERO.AI \/ Pfizer Inc"},{"orgOrder":0,"company":"EdiGene","sponsor":"University of Wisconsin","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"EdiGene","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"EdiGene \/ University of Wisconsin","highestDevelopmentStatusID":"2","companyTruncated":"EdiGene \/ University of Wisconsin"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CANbridge Pharmaceuticals \/ UMass Chan Medical School","highestDevelopmentStatusID":"4","companyTruncated":"CANbridge Pharmaceuticals \/ UMass Chan Medical School"},{"orgOrder":0,"company":"PeptiDream","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"PeptiDream","amount2":2.2000000000000002,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":2.2000000000000002,"dosageForm":"Undisclosed","sponsorNew":"PeptiDream \/ Alnylam Pharmaceuticals","highestDevelopmentStatusID":"3","companyTruncated":"PeptiDream \/ Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"University Hospital, Lille","sponsor":"Imagine Institute | Octapharma | CSL Behring | LFB Group | Air Liquide Group | The Binding Site Ltd","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University Hospital, Lille","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University Hospital, Lille \/ Imagine Institute | Octapharma | CSL Behring | LFB Group | Air Liquide Group | The Binding Site Ltd","highestDevelopmentStatusID":"1","companyTruncated":"University Hospital, Lille \/ Imagine Institute | Octapharma | CSL Behring | LFB Group | Air Liquide Group | The Binding Site Ltd"},{"orgOrder":0,"company":"Assistance Publique \u2013 H\u00f4pitaux de Paris","sponsor":"Vaincre la Mucoviscidose","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Assistance Publique \u2013 H\u00f4pitaux de Paris","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Vaincre la Mucoviscidose","highestDevelopmentStatusID":"1","companyTruncated":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Vaincre la Mucoviscidose"},{"orgOrder":0,"company":"Biotrial","sponsor":"European Union | Nantes University Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Biotrial","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biotrial \/ European Union | Nantes University Hospital","highestDevelopmentStatusID":"1","companyTruncated":"Biotrial \/ European Union | Nantes University Hospital"},{"orgOrder":0,"company":"University Hospital, Montpellier","sponsor":"Institut de Recherche en Infectiologie de Montpellier (CNRS) | A*STAR","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University Hospital, Montpellier","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University Hospital, Montpellier \/ Institut de Recherche en Infectiologie de Montpellier (CNRS) | A*STAR","highestDevelopmentStatusID":"1","companyTruncated":"University Hospital, Montpellier \/ Institut de Recherche en Infectiologie de Montpellier (CNRS) | A*STAR"},{"orgOrder":0,"company":"Centre Hospitalier de Saint-Denis","sponsor":"INSERM U955","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Centre Hospitalier de Saint-Denis","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Centre Hospitalier de Saint-Denis \/ INSERM U955","highestDevelopmentStatusID":"1","companyTruncated":"Centre Hospitalier de Saint-Denis \/ INSERM U955"},{"orgOrder":0,"company":"University of Primorska","sponsor":"Biostile d.o.o. | QFarm s.r.l.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SLOVENIA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Primorska","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Primorska \/ Biostile d.o.o. | QFarm s.r.l.","highestDevelopmentStatusID":"1","companyTruncated":"University of Primorska \/ Biostile d.o.o. | QFarm s.r.l."},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"OMass Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"OMass Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"OMass Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"GV","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2022","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"OMass Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Inhalation","sponsorNew":"OMass Therapeutics \/ GV","highestDevelopmentStatusID":"2","companyTruncated":"OMass Therapeutics \/ GV"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Laboratoires Thea","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Laboratoires Thea \/ Sepul Bio","highestDevelopmentStatusID":"8","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"Cellectis","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cellectis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Cellectis \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Cellectis \/ Undisclosed"},{"orgOrder":0,"company":"Genespire","sponsor":"Sofinnova Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genespire","amount2":0.02,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Injection","sponsorNew":"Genespire \/ Sofinnova Partners","highestDevelopmentStatusID":"1","companyTruncated":"Genespire \/ Sofinnova Partners"},{"orgOrder":0,"company":"ReNeuron","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"ReNeuron","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ReNeuron \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"ReNeuron \/ Undisclosed"},{"orgOrder":0,"company":"LoQus23 Therapeutics","sponsor":"Forbion","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2024","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"LoQus23 Therapeutics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Undisclosed","sponsorNew":"LoQus23 Therapeutics \/ Forbion","highestDevelopmentStatusID":"4","companyTruncated":"LoQus23 Therapeutics \/ Forbion"},{"orgOrder":0,"company":"Exicure","sponsor":"Ipsen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Exicure","amount2":1.02,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.02,"dosageForm":"Undisclosed","sponsorNew":"Exicure \/ Ipsen","highestDevelopmentStatusID":"2","companyTruncated":"Exicure \/ Ipsen"},{"orgOrder":0,"company":"Bit Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Bit Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bit Bio \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"Bit Bio \/ Undisclosed"},{"orgOrder":0,"company":"Elastin BioSciences","sponsor":"Longaevus Technologies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2023","type":"Demerger","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Elastin BioSciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Elastin BioSciences \/ Longaevus Technologies","highestDevelopmentStatusID":"4","companyTruncated":"Elastin BioSciences \/ Longaevus Technologies"},{"orgOrder":0,"company":"Caszyme","sponsor":"Integra Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"LITHUANIA","productType":"Undisclosed","year":"2024","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Caszyme","amount2":0.040000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Caszyme \/ Integra Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Caszyme \/ Integra Therapeutics"},{"orgOrder":0,"company":"Cellectis","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cellectis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cellectis \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Cellectis \/ Undisclosed"},{"orgOrder":0,"company":"Vipergen","sponsor":"Casma Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Undisclosed","year":"2020","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Vipergen","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vipergen \/ Casma Therapeutics","highestDevelopmentStatusID":"3","companyTruncated":"Vipergen \/ Casma Therapeutics"},{"orgOrder":0,"company":"Bit Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Bit Bio","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bit Bio \/ Undisclosed","highestDevelopmentStatusID":"3","companyTruncated":"Bit Bio \/ Undisclosed"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"ProQR Therapeutics","amount2":1.3,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.3,"dosageForm":"Undisclosed","sponsorNew":"ProQR Therapeutics \/ Eli Lilly","highestDevelopmentStatusID":"2","companyTruncated":"ProQR Therapeutics \/ Eli Lilly"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Yarrow Biotechnology","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ProQR Therapeutics \/ Yarrow Biotechnology","highestDevelopmentStatusID":"2","companyTruncated":"ProQR Therapeutics \/ Yarrow Biotechnology"},{"orgOrder":0,"company":"CYTOO","sponsor":"Astellas Gene Therapies","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"CYTOO","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CYTOO \/ Astellas Gene Therapies","highestDevelopmentStatusID":"1","companyTruncated":"CYTOO \/ Astellas Gene Therapies"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"CMT Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oryzon Genomics \/ CMT Research Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Oryzon Genomics \/ CMT Research Foundation"},{"orgOrder":0,"company":"Addex Therapeutics","sponsor":"Charcot Marie Tooth Association","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Addex Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Addex Therapeutics \/ Charcot Marie Tooth Association","highestDevelopmentStatusID":"4","companyTruncated":"Addex Therapeutics \/ Charcot Marie Tooth Association"},{"orgOrder":0,"company":"Anima Medical Centre","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Anima Medical Centre","amount2":2.4199999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":2.4199999999999999,"dosageForm":"Undisclosed","sponsorNew":"Anima Medical Centre \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Anima Medical Centre \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"VectorY","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"VectorY","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"VectorY \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"VectorY \/ Undisclosed"},{"orgOrder":0,"company":"Harness Therapeutics","sponsor":"Epidarex Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Harness Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"Harness Therapeutics \/ Epidarex Capital","highestDevelopmentStatusID":"4","companyTruncated":"Harness Therapeutics \/ Epidarex Capital"},{"orgOrder":0,"company":"Bit Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Bit Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bit Bio \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"Bit Bio \/ Undisclosed"},{"orgOrder":0,"company":"LoQus23 Therapeutics","sponsor":"Dementia Discovery Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"LoQus23 Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"LoQus23 Therapeutics \/ Dementia Discovery Fund","highestDevelopmentStatusID":"4","companyTruncated":"LoQus23 Therapeutics \/ Dementia Discovery Fund"},{"orgOrder":0,"company":"VectorY","sponsor":"Wageningen University","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"VectorY","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"VectorY \/ Wageningen University","highestDevelopmentStatusID":"4","companyTruncated":"VectorY \/ Wageningen University"},{"orgOrder":0,"company":"VECT-HORUS","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2021","type":"Series D Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"VECT-HORUS","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"VECT-HORUS \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"VECT-HORUS \/ Undisclosed"},{"orgOrder":0,"company":"Silence Therapeutics","sponsor":"Hansoh Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Silence Therapeutics","amount2":1.3200000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":1.3200000000000001,"dosageForm":"Undisclosed","sponsorNew":"Silence Therapeutics \/ Hansoh Pharma","highestDevelopmentStatusID":"4","companyTruncated":"Silence Therapeutics \/ Hansoh Pharma"},{"orgOrder":0,"company":"SOM Biotech","sponsor":"University of Minnesota","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2022","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SOM Biotech","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SOM Biotech \/ University of Minnesota","highestDevelopmentStatusID":"4","companyTruncated":"SOM Biotech \/ University of Minnesota"},{"orgOrder":0,"company":"MitoRx Therapeutics","sponsor":"Wren Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"MitoRx Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"MitoRx Therapeutics \/ Wren Capital","highestDevelopmentStatusID":"4","companyTruncated":"MitoRx Therapeutics \/ Wren Capital"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Life Science Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vico Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Vico Therapeutics \/ Life Science Partners","highestDevelopmentStatusID":"4","companyTruncated":"Vico Therapeutics \/ Life Science Partners"},{"orgOrder":0,"company":"ViaNautis Bio","sponsor":"SomaServe","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2023","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"ViaNautis Bio","amount2":0.029999999999999999,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Undisclosed","sponsorNew":"ViaNautis Bio \/ SomaServe","highestDevelopmentStatusID":"3","companyTruncated":"ViaNautis Bio \/ SomaServe"},{"orgOrder":0,"company":"Scenic Biotech","sponsor":"Bristol Myers Squibb","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Scenic Biotech","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Scenic Biotech \/ Bristol Myers Squibb","highestDevelopmentStatusID":"3","companyTruncated":"Scenic Biotech \/ Bristol Myers Squibb"},{"orgOrder":0,"company":"Sanofi","sponsor":"Global Blood Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2021","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Sanofi","amount2":0.34999999999999998,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Global Blood Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Sanofi \/ Global Blood Therapeutics"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Bill & Melinda Gates Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Bill & Melinda Gates Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Bill & Melinda Gates Foundation"},{"orgOrder":0,"company":"Proteros","sponsor":"Adrestia Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Proteros","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proteros \/ Adrestia Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Proteros \/ Adrestia Therapeutics"},{"orgOrder":0,"company":"Scenic Biotech","sponsor":"Genentech","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Scenic Biotech","amount2":0.38,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0.38,"dosageForm":"Undisclosed","sponsorNew":"Scenic Biotech \/ Genentech","highestDevelopmentStatusID":"3","companyTruncated":"Scenic Biotech \/ Genentech"},{"orgOrder":0,"company":"Light Chain Bioscience","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Light Chain Bioscience","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Light Chain Bioscience \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"2","companyTruncated":"Light Chain Bioscience \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Genflow Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Genflow Biosciences","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genflow Biosciences \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"Genflow Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Sirion Biotech","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sirion Biotech","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sirion Biotech \/ Sanofi","highestDevelopmentStatusID":"1","companyTruncated":"Sirion Biotech \/ Sanofi"},{"orgOrder":0,"company":"Genespire","sponsor":"Sofinnova Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2020","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genespire","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genespire \/ Sofinnova Partners","highestDevelopmentStatusID":"1","companyTruncated":"Genespire \/ Sofinnova Partners"},{"orgOrder":0,"company":"Anjarium Biosciences","sponsor":"Abingworth","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Anjarium Biosciences","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Anjarium Biosciences \/ Abingworth","highestDevelopmentStatusID":"1","companyTruncated":"Anjarium Biosciences \/ Abingworth"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Ysios Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"SpliceBio","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"SpliceBio \/ Ysios Capital","highestDevelopmentStatusID":"1","companyTruncated":"SpliceBio \/ Ysios Capital"},{"orgOrder":0,"company":"Kamada","sponsor":"Syneos Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kamada","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Kamada \/ Syneos Health","highestDevelopmentStatusID":"10","companyTruncated":"Kamada \/ Syneos Health"},{"orgOrder":0,"company":"Quris","sponsor":"Welltech Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Undisclosed","year":"2022","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Quris","amount2":0.029999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Quris \/ Welltech Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Quris \/ Welltech Ventures"},{"orgOrder":0,"company":"Edity Therapeutics","sponsor":"Dr. Reddy's Laboratories","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Undisclosed","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Edity Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Edity Therapeutics \/ Dr. Reddy's Laboratories","highestDevelopmentStatusID":"4","companyTruncated":"Edity Therapeutics \/ Dr. Reddy's Laboratories"},{"orgOrder":0,"company":"SEAL Therapeutics","sponsor":"Santhera Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SEAL Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SEAL Therapeutics \/ Santhera Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"SEAL Therapeutics \/ Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Brigham and Women's Hospital","sponsor":"Baylor College of Medicine | Boston Children's Hospital | Broad Institute of MIT and Harvard | Dartmouth-Hitchcock Medical Center | Harvard Pilgrim Health Care | Howard University | HudsonAlpha Institute for Biotechnology | Massachusetts General Hospital ","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Brigham and Women's Hospital","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Brigham and Women's Hospital \/ Baylor College of Medicine | Boston Children's Hospital | Broad Institute of MIT and Harvard | Dartmouth-Hitchcock Medical Center | Harvard Pilgrim Health Care | Howard University | HudsonAlpha Institute for Biotechnology | Massachusetts General Hospital ","highestDevelopmentStatusID":"1","companyTruncated":"Brigham and Women's Hospital \/ Baylor College of Medicine | Boston Children's Hospital | Broad Institute of MIT and Harvard | Dartmouth-Hitchcock Medical Center | Harvard Pilgrim Health Care | Howard University | HudsonAlpha Institute for Biotechnology | Massachusetts General Hospital "},{"orgOrder":0,"company":"Kriya Therapeutics","sponsor":"Kriya Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Kriya Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kriya Therapeutics \/ Kriya Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Kriya Therapeutics \/ Kriya Therapeutics"},{"orgOrder":0,"company":"University of Massachusetts Medical School","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Massachusetts Medical School","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Massachusetts Medical School \/ CANbridge Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"University of Massachusetts Medical School \/ CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"University of Massachusetts","sponsor":"ASC Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"University of Massachusetts","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Massachusetts \/ ASC Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"University of Massachusetts \/ ASC Therapeutics"},{"orgOrder":0,"company":"University of Massachusetts Medical School","sponsor":"Foundation To Fight H-Abc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"University of Massachusetts Medical School","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Massachusetts Medical School \/ Foundation To Fight H-Abc","highestDevelopmentStatusID":"2","companyTruncated":"University of Massachusetts Medical School \/ Foundation To Fight H-Abc"},{"orgOrder":0,"company":"Harvard Stem Cell Institute","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Harvard Stem Cell Institute","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Harvard Stem Cell Institute \/ Sarepta Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Harvard Stem Cell Institute \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"UMass Chan Medical School","sponsor":"QurAlis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"UMass Chan Medical School","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"UMass Chan Medical School \/ QurAlis","highestDevelopmentStatusID":"2","companyTruncated":"UMass Chan Medical School \/ QurAlis"},{"orgOrder":0,"company":"Wake Forest Institute for Regenerative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Wake Forest Institute for Regenerative Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Wake Forest Institute for Regenerative Medicine \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Wake Forest Institute for Regenerative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Nationwide Children\u2019s Hospital","sponsor":"SOLVE FSHD","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Nationwide Children\u2019s Hospital","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nationwide Children\u2019s Hospital \/ SOLVE FSHD","highestDevelopmentStatusID":"4","companyTruncated":"Nationwide Children\u2019s Hospital \/ SOLVE FSHD"},{"orgOrder":0,"company":"Oregon State University","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oregon State University","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Oregon State University \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Oregon State University \/ Undisclosed"},{"orgOrder":0,"company":"Genelex Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genelex Corporation","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genelex Corporation \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Genelex Corporation \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"MPM Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Aerosol","sponsorNew":"ReCode Therapeutics \/ MPM Capital","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ MPM Capital"},{"orgOrder":0,"company":"XOMA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"XOMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"XOMA \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"XOMA \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Leiden University Medical Centre","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Leiden University Medical Centre","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Leiden University Medical Centre"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arcturus Therapeutics \/ Guggenheim Securities","highestDevelopmentStatusID":"1","companyTruncated":"Arcturus Therapeutics \/ Guggenheim Securities"},{"orgOrder":0,"company":"Nova Mentis Life Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Nova Mentis Life Science","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Nova Mentis Life Science \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Nova Mentis Life Science \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Generation Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Generation Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Generation Bio \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Generation Bio \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Tessera Therapeutics","sponsor":"Bill & Melinda Gates Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Tessera Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Injection","sponsorNew":"Tessera Therapeutics \/ Bill & Melinda Gates Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Tessera Therapeutics \/ Bill & Melinda Gates Foundation"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Guide Therapeutics","sponsor":"Beam Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Guide Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Guide Therapeutics \/ Beam Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Guide Therapeutics \/ Beam Therapeutics"},{"orgOrder":0,"company":"Aldevron","sponsor":"AavantiBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Aldevron","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aldevron \/ AavantiBio","highestDevelopmentStatusID":"4","companyTruncated":"Aldevron \/ AavantiBio"},{"orgOrder":0,"company":"Anagram Therapeutics","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Anagram Therapeutics","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Tablet","sponsorNew":"Anagram Therapeutics \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Anagram Therapeutics \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pfizer Inc \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"NeuExcell therapeutics","sponsor":"Spark Therapeutics, Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"NeuExcell therapeutics","amount2":0.19,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.19,"dosageForm":"Undisclosed","sponsorNew":"NeuExcell therapeutics \/ Spark Therapeutics, Inc","highestDevelopmentStatusID":"5","companyTruncated":"NeuExcell therapeutics \/ Spark Therapeutics, Inc"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Voyager Therapeutics, Inc","amount2":1.3,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":1.3,"dosageForm":"Undisclosed","sponsorNew":"Voyager Therapeutics, Inc \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"4","companyTruncated":"Voyager Therapeutics, Inc \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Voyager Therapeutics, Inc","amount2":0.63,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.63,"dosageForm":"Undisclosed","sponsorNew":"Voyager Therapeutics, Inc \/ Pfizer Inc","highestDevelopmentStatusID":"4","companyTruncated":"Voyager Therapeutics, Inc \/ Pfizer Inc"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"SR One Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"ADARx Pharmaceuticals","amount2":0.080000000000000002,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"ADARx Pharmaceuticals \/ SR One Capital Management","highestDevelopmentStatusID":"5","companyTruncated":"ADARx Pharmaceuticals \/ SR One Capital Management"},{"orgOrder":0,"company":"DTx Pharma","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"DTx Pharma","amount2":0.10000000000000001,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Undisclosed","sponsorNew":"DTx Pharma \/ RA Capital Management","highestDevelopmentStatusID":"5","companyTruncated":"DTx Pharma \/ RA Capital Management"},{"orgOrder":0,"company":"Ascidian Therapeutics","sponsor":"Apple Tree Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Ascidian Therapeutics","amount2":0.040000000000000001,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Ascidian Therapeutics \/ Apple Tree Partners","highestDevelopmentStatusID":"5","companyTruncated":"Ascidian Therapeutics \/ Apple Tree Partners"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Actio Biosciences","sponsor":"Canaan Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Actio Biosciences","amount2":0.059999999999999998,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Actio Biosciences \/ Canaan Partners","highestDevelopmentStatusID":"4","companyTruncated":"Actio Biosciences \/ Canaan Partners"},{"orgOrder":0,"company":"Sail Biomedicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Sail Biomedicines","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sail Biomedicines \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Sail Biomedicines \/ Undisclosed"},{"orgOrder":0,"company":"Resilience","sponsor":"AavantiBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Resilience","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Resilience \/ AavantiBio","highestDevelopmentStatusID":"4","companyTruncated":"Resilience \/ AavantiBio"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gain Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"MaxCyte","sponsor":"Be Biopharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"MaxCyte","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MaxCyte \/ Be Biopharma","highestDevelopmentStatusID":"4","companyTruncated":"MaxCyte \/ Be Biopharma"},{"orgOrder":0,"company":"Metagenomi","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0.089999999999999997,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ J.P. Morgan","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ J.P. Morgan"},{"orgOrder":0,"company":"Metagenomi","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0.089999999999999997,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ J.P. Morgan","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ J.P. Morgan"},{"orgOrder":0,"company":"Metagenomi","sponsor":"Novo Holding","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0.28000000000000003,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.28000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ Novo Holding","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ Novo Holding"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intellia Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Intellia Therapeutics \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Intellia Therapeutics \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Chameleon Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Chameleon Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chameleon Biosciences \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Chameleon Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Be Biopharma \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Be Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"SalioGen Therapeutics","sponsor":"GordonMD Global","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SalioGen Therapeutics","amount2":0.12,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Undisclosed","sponsorNew":"SalioGen Therapeutics \/ GordonMD Global","highestDevelopmentStatusID":"4","companyTruncated":"SalioGen Therapeutics \/ GordonMD Global"},{"orgOrder":0,"company":"Casma Therapeutics","sponsor":"The Column Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Casma Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Casma Therapeutics \/ The Column Group","highestDevelopmentStatusID":"4","companyTruncated":"Casma Therapeutics \/ The Column Group"},{"orgOrder":0,"company":"Code Bio","sponsor":"Northpond Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Code Bio","amount2":0.080000000000000002,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Code Bio \/ Northpond Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Code Bio \/ Northpond Ventures"},{"orgOrder":0,"company":"Capsida","sponsor":"Kate Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Capsida","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Capsida \/ Kate Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Capsida \/ Kate Therapeutics"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Vida Ventures LLC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Dyne Therapeutics","amount2":0.12,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Undisclosed","sponsorNew":"Dyne Therapeutics \/ Vida Ventures LLC","highestDevelopmentStatusID":"4","companyTruncated":"Dyne Therapeutics \/ Vida Ventures LLC"},{"orgOrder":0,"company":"Ashvattha Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ashvattha Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ashvattha Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Ashvattha Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Stoke Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Stoke Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Stoke Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Massachusetts Eye And Ear","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Massachusetts Eye And Ear","highestDevelopmentStatusID":"4","companyTruncated":"Biogen \/ Massachusetts Eye And Ear"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Cure AP-4","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Charles River Laboratories, Inc \/ Cure AP-4","highestDevelopmentStatusID":"4","companyTruncated":"Charles River Laboratories, Inc \/ Cure AP-4"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Acadia Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Stoke Therapeutics","amount2":0.96999999999999997,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.96999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Stoke Therapeutics \/ Acadia Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Stoke Therapeutics \/ Acadia Pharmaceuticals"},{"orgOrder":0,"company":"Diagonal Therapeutics","sponsor":"BVF Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Diagonal Therapeutics","amount2":0.13,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"Undisclosed","sponsorNew":"Diagonal Therapeutics \/ BVF Partners","highestDevelopmentStatusID":"4","companyTruncated":"Diagonal Therapeutics \/ BVF Partners"},{"orgOrder":0,"company":"Frequency Therapeutics","sponsor":"Korro Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Merger","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Frequency Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Frequency Therapeutics \/ Korro Bio","highestDevelopmentStatusID":"3","companyTruncated":"Frequency Therapeutics \/ Korro Bio"},{"orgOrder":0,"company":"Amber Bio","sponsor":"Playground Global","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Amber Bio","amount2":0.029999999999999999,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Amber Bio \/ Playground Global","highestDevelopmentStatusID":"3","companyTruncated":"Amber Bio \/ Playground Global"},{"orgOrder":0,"company":"Metagenomi","sponsor":"Moderna Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Metagenomi","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ Moderna Therapeutics","highestDevelopmentStatusID":"3","companyTruncated":"Metagenomi \/ Moderna Therapeutics"},{"orgOrder":0,"company":"Arbor Pharmaceuticals","sponsor":"EdiGene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Arbor Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Arbor Pharmaceuticals \/ EdiGene","highestDevelopmentStatusID":"3","companyTruncated":"Arbor Pharmaceuticals \/ EdiGene"},{"orgOrder":0,"company":"MaxCyte","sponsor":"Prime Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"MaxCyte","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MaxCyte \/ Prime Medicine","highestDevelopmentStatusID":"4","companyTruncated":"MaxCyte \/ Prime Medicine"},{"orgOrder":0,"company":"Integral Molecular","sponsor":"Optimeos Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Integral Molecular","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Integral Molecular \/ Optimeos Life Sciences","highestDevelopmentStatusID":"3","companyTruncated":"Integral Molecular \/ Optimeos Life Sciences"},{"orgOrder":0,"company":"Genetic Leap","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Genetic Leap","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genetic Leap \/ Eli Lilly","highestDevelopmentStatusID":"3","companyTruncated":"Genetic Leap \/ Eli Lilly"},{"orgOrder":0,"company":"Alltrna","sponsor":"Flagship Pioneering","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Alltrna","amount2":0.11,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"Alltrna \/ Flagship Pioneering","highestDevelopmentStatusID":"3","companyTruncated":"Alltrna \/ Flagship Pioneering"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Eventide Asset Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Korro Bio","amount2":0.12,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Undisclosed","sponsorNew":"Korro Bio \/ Eventide Asset Management","highestDevelopmentStatusID":"2","companyTruncated":"Korro Bio \/ Eventide Asset Management"},{"orgOrder":0,"company":"CAMP4 Therapeutics","sponsor":"Fulcrum Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"CAMP4 Therapeutics","amount2":0.070000000000000007,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Undisclosed","sponsorNew":"CAMP4 Therapeutics \/ Fulcrum Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"CAMP4 Therapeutics \/ Fulcrum Therapeutics"},{"orgOrder":0,"company":"Gensaic","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Gensaic","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gensaic \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Gensaic \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Flagship Pioneering","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Flagship Pioneering","amount2":0.11,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"Flagship Pioneering \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Flagship Pioneering \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Scribe Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Expanded Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Scribe Therapeutics","amount2":1.24,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.24,"dosageForm":"Undisclosed","sponsorNew":"Scribe Therapeutics \/ Sanofi","highestDevelopmentStatusID":"2","companyTruncated":"Scribe Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"Dyno Therapeutics","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Dyno Therapeutics","amount2":1.6200000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.6200000000000001,"dosageForm":"Undisclosed","sponsorNew":"Dyno Therapeutics \/ Astellas Pharma","highestDevelopmentStatusID":"2","companyTruncated":"Dyno Therapeutics \/ Astellas Pharma"},{"orgOrder":0,"company":"Sanegene Bio","sponsor":"Orbit Discovery","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Sanegene Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanegene Bio \/ Orbit Discovery","highestDevelopmentStatusID":"2","companyTruncated":"Sanegene Bio \/ Orbit Discovery"},{"orgOrder":0,"company":"Genevant Sciences","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Genevant Sciences","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genevant Sciences \/ Novo Nordisk","highestDevelopmentStatusID":"2","companyTruncated":"Genevant Sciences \/ Novo Nordisk"},{"orgOrder":0,"company":"Metrion Biosciences","sponsor":"KCNC1 Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Metrion Biosciences","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Metrion Biosciences \/ KCNC1 Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Metrion Biosciences \/ KCNC1 Foundation"},{"orgOrder":0,"company":"Kate Therapeutics","sponsor":"Westlake Village BioPartners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Kate Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Kate Therapeutics \/ Westlake Village BioPartners","highestDevelopmentStatusID":"2","companyTruncated":"Kate Therapeutics \/ Westlake Village BioPartners"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Genevant Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Korro Bio \/ Genevant Sciences","highestDevelopmentStatusID":"2","companyTruncated":"Korro Bio \/ Genevant Sciences"},{"orgOrder":0,"company":"National Institutes of Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"National Institutes of Health","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Institutes of Health \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"National Institutes of Health \/ Undisclosed"},{"orgOrder":0,"company":"Codexis","sponsor":"Crosswalk Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Codexis","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Codexis \/ Crosswalk Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Codexis \/ Crosswalk Therapeutics"},{"orgOrder":0,"company":"AavantiBio","sponsor":"University of Florida","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"AavantiBio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AavantiBio \/ University of Florida","highestDevelopmentStatusID":"1","companyTruncated":"AavantiBio \/ University of Florida"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Personalis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Personalis","highestDevelopmentStatusID":"1","companyTruncated":"Sarepta Therapeutics \/ Personalis"},{"orgOrder":0,"company":"2Seventy Bio","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"2Seventy Bio","amount2":0.040000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Undisclosed","sponsorNew":"2Seventy Bio \/ Novo Nordisk","highestDevelopmentStatusID":"4","companyTruncated":"2Seventy Bio \/ Novo Nordisk"},{"orgOrder":0,"company":"Duchenne research","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Duchenne research","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Duchenne research \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Duchenne research \/ Undisclosed"},{"orgOrder":0,"company":"MaxCyte","sponsor":"Curamys","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"MaxCyte","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MaxCyte \/ Curamys","highestDevelopmentStatusID":"1","companyTruncated":"MaxCyte \/ Curamys"},{"orgOrder":0,"company":"PepGen","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"PepGen","amount2":0.050000000000000003,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"PepGen \/ RA Capital Management","highestDevelopmentStatusID":"1","companyTruncated":"PepGen \/ RA Capital Management"},{"orgOrder":0,"company":"Dewpoint Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dewpoint Therapeutics","amount2":0.23999999999999999,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Dewpoint Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"1","companyTruncated":"Dewpoint Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"Andelyn Biosciences","sponsor":"Armatus Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Andelyn Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Andelyn Biosciences \/ Armatus Bio","highestDevelopmentStatusID":"4","companyTruncated":"Andelyn Biosciences \/ Armatus Bio"},{"orgOrder":0,"company":"Rumi Scientific","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rumi Scientific","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rumi Scientific \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Rumi Scientific \/ Undisclosed"},{"orgOrder":0,"company":"ReviR Therapeutics","sponsor":"CMT Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"ReviR Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ReviR Therapeutics \/ CMT Research Foundation","highestDevelopmentStatusID":"3","companyTruncated":"ReviR Therapeutics \/ CMT Research Foundation"},{"orgOrder":0,"company":"ReviR Therapeutics","sponsor":"Lapam Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"ReviR Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ReviR Therapeutics \/ Lapam Capital","highestDevelopmentStatusID":"3","companyTruncated":"ReviR Therapeutics \/ Lapam Capital"},{"orgOrder":0,"company":"Nanite","sponsor":"CMT Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Nanite","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nanite \/ CMT Research Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Nanite \/ CMT Research Foundation"},{"orgOrder":0,"company":"Aitia","sponsor":"UCB Pharma S.A","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Aitia","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aitia \/ UCB Pharma S.A","highestDevelopmentStatusID":"2","companyTruncated":"Aitia \/ UCB Pharma S.A"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Neurochase","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Spark Therapeutics, Inc \/ Neurochase","highestDevelopmentStatusID":"2","companyTruncated":"Spark Therapeutics, Inc \/ Neurochase"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Versant Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Graphite Bio","amount2":0.050000000000000003,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Graphite Bio \/ Versant Ventures","highestDevelopmentStatusID":"5","companyTruncated":"Graphite Bio \/ Versant Ventures"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"RTW Investments","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series C Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Avidity Biosciences","amount2":0.10000000000000001,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Avidity Biosciences \/ RTW Investments","highestDevelopmentStatusID":"5","companyTruncated":"Avidity Biosciences \/ RTW Investments"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Prime Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Prime Medicine \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Prime Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Eurostars","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gain Therapeutics \/ Eurostars","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Eurostars"},{"orgOrder":0,"company":"Scribe Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Expanded Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Scribe Therapeutics","amount2":1.24,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":1.24,"dosageForm":"Undisclosed","sponsorNew":"Scribe Therapeutics \/ Sanofi","highestDevelopmentStatusID":"4","companyTruncated":"Scribe Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Undisclosed","sponsorNew":"ReCode Therapeutics \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Pfizer Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"ReCode Therapeutics \/ Pfizer Ventures","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Pfizer Ventures"},{"orgOrder":0,"company":"Nanite","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Nanite","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nanite \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Nanite \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Prime Medicine","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Undisclosed","sponsorNew":"Prime Medicine \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Prime Medicine \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Catalent Pharma Solutions","sponsor":"AavantiBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Catalent Pharma Solutions","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Catalent Pharma Solutions \/ AavantiBio","highestDevelopmentStatusID":"4","companyTruncated":"Catalent Pharma Solutions \/ AavantiBio"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gain Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Rectify Pharma","sponsor":"Atlas Venture","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rectify Pharma","amount2":0.10000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Rectify Pharma \/ Atlas Venture","highestDevelopmentStatusID":"4","companyTruncated":"Rectify Pharma \/ Atlas Venture"},{"orgOrder":0,"company":"Metagenomi","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0.089999999999999997,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ J.P. Morgan","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ J.P. Morgan"},{"orgOrder":0,"company":"2Seventy Bio","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Divestment","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"2Seventy Bio","amount2":0.040000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Undisclosed","sponsorNew":"2Seventy Bio \/ Novo Nordisk","highestDevelopmentStatusID":"4","companyTruncated":"2Seventy Bio \/ Novo Nordisk"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Termination","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intellia Therapeutics","amount2":0.41999999999999998,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.41999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Intellia Therapeutics \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Intellia Therapeutics \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Be Biopharma \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Be Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"ASC Therapeutics","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ASC Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ASC Therapeutics \/ UMass Chan Medical School","highestDevelopmentStatusID":"4","companyTruncated":"ASC Therapeutics \/ UMass Chan Medical School"},{"orgOrder":0,"company":"INmune Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"INmune Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"INmune Bio \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"INmune Bio \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Wellington Management Company","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Entrada Therapeutics","amount2":0.12,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Undisclosed","sponsorNew":"Entrada Therapeutics \/ Wellington Management Company","highestDevelopmentStatusID":"4","companyTruncated":"Entrada Therapeutics \/ Wellington Management Company"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Prevail Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Termination","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Precision BioSciences","amount2":0.56000000000000005,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.56000000000000005,"dosageForm":"Undisclosed","sponsorNew":"Precision BioSciences \/ Prevail Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Precision BioSciences \/ Prevail Therapeutics"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jnana Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Jnana Therapeutics \/ RA Capital Management","highestDevelopmentStatusID":"4","companyTruncated":"Jnana Therapeutics \/ RA Capital Management"},{"orgOrder":0,"company":"GondolaBio","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Demerger","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"GondolaBio","amount2":0.29999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.29999999999999999,"dosageForm":"Undisclosed","sponsorNew":"GondolaBio \/ BridgeBio Pharma","highestDevelopmentStatusID":"4","companyTruncated":"GondolaBio \/ BridgeBio Pharma"},{"orgOrder":0,"company":"IVERIC bio","sponsor":"Opus Genetics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"IVERIC bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IVERIC bio \/ Opus Genetics","highestDevelopmentStatusID":"4","companyTruncated":"IVERIC bio \/ Opus Genetics"},{"orgOrder":0,"company":"Oak Bay Biosciences","sponsor":"FrontFundr","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2021","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oak Bay Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oak Bay Biosciences \/ FrontFundr","highestDevelopmentStatusID":"4","companyTruncated":"Oak Bay Biosciences \/ FrontFundr"},{"orgOrder":0,"company":"Frequency Therapeutics","sponsor":"Korro Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Merger","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Frequency Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Frequency Therapeutics \/ Korro Bio","highestDevelopmentStatusID":"3","companyTruncated":"Frequency Therapeutics \/ Korro Bio"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"GSK","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Wave Life Sciences","amount2":3.3399999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":3.3399999999999999,"dosageForm":"Undisclosed","sponsorNew":"Wave Life Sciences \/ GSK","highestDevelopmentStatusID":"4","companyTruncated":"Wave Life Sciences \/ GSK"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"ReCode Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Intellia Therapeutics \/ ReCode Therapeutics","highestDevelopmentStatusID":"3","companyTruncated":"Intellia Therapeutics \/ ReCode Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"The University Of Colorado Anschutz Medical Campus","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BridgeBio Pharma \/ The University Of Colorado Anschutz Medical Campus","highestDevelopmentStatusID":"3","companyTruncated":"BridgeBio Pharma \/ The University Of Colorado Anschutz Medical Campus"},{"orgOrder":0,"company":"Evozyne","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Evozyne","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Evozyne \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"3","companyTruncated":"Evozyne \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Deep Genomics","sponsor":"SoftBank Vision Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2021","type":"Series C Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Deep Genomics","amount2":0.17999999999999999,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Deep Genomics \/ SoftBank Vision Fund","highestDevelopmentStatusID":"3","companyTruncated":"Deep Genomics \/ SoftBank Vision Fund"},{"orgOrder":0,"company":"Alloy Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Alloy Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alloy Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"3","companyTruncated":"Alloy Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Obsidian Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Obsidian Therapeutics","amount2":1.3,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":1.3,"dosageForm":"Undisclosed","sponsorNew":"Obsidian Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"3","companyTruncated":"Obsidian Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Ring Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Ring Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ring Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"3","companyTruncated":"Ring Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Neochromosome","sponsor":"University of Edinburgh","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Neochromosome","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neochromosome \/ University of Edinburgh","highestDevelopmentStatusID":"3","companyTruncated":"Neochromosome \/ University of Edinburgh"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"GondolaBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ GondolaBio","highestDevelopmentStatusID":"3","companyTruncated":"n-Lorem Foundation \/ GondolaBio"},{"orgOrder":0,"company":"ElevateBio","sponsor":"Amazon Web Services","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"ElevateBio","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ElevateBio \/ Amazon Web Services","highestDevelopmentStatusID":"3","companyTruncated":"ElevateBio \/ Amazon Web Services"},{"orgOrder":0,"company":"Enveda Bioscience","sponsor":"Lux Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Enveda Bioscience","amount2":0.050000000000000003,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Enveda Bioscience \/ Lux Capital","highestDevelopmentStatusID":"3","companyTruncated":"Enveda Bioscience \/ Lux Capital"},{"orgOrder":0,"company":"Rampart Bioscience","sponsor":"OrbiMed Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Rampart Bioscience","amount2":0.089999999999999997,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Rampart Bioscience \/ OrbiMed Advisors","highestDevelopmentStatusID":"3","companyTruncated":"Rampart Bioscience \/ OrbiMed Advisors"},{"orgOrder":0,"company":"Protego Biopharma","sponsor":"Lightspeed Venture Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Protego Biopharma","amount2":0.050000000000000003,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Protego Biopharma \/ Lightspeed Venture Partners","highestDevelopmentStatusID":"3","companyTruncated":"Protego Biopharma \/ Lightspeed Venture Partners"},{"orgOrder":0,"company":"Transcripta Bio","sponsor":"SOLVE FSHD","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"Transcripta Bio","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Transcripta Bio \/ SOLVE FSHD","highestDevelopmentStatusID":"3","companyTruncated":"Transcripta Bio \/ SOLVE FSHD"},{"orgOrder":0,"company":"Scribe Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Expanded Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Scribe Therapeutics","amount2":1.24,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":1.24,"dosageForm":"Undisclosed","sponsorNew":"Scribe Therapeutics \/ Sanofi","highestDevelopmentStatusID":"4","companyTruncated":"Scribe Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"Orna Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Orna Therapeutics","amount2":0.69999999999999996,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.69999999999999996,"dosageForm":"Undisclosed","sponsorNew":"Orna Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"Orna Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"MyoKardia","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Fulcrum Therapeutics","amount2":0.46999999999999997,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.46999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Fulcrum Therapeutics \/ MyoKardia","highestDevelopmentStatusID":"2","companyTruncated":"Fulcrum Therapeutics \/ MyoKardia"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Moderna Therapeutics","amount2":0.46000000000000002,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.46000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Moderna Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"Moderna Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Scriptr","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Scriptr","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Scriptr \/ CANbridge Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"Scriptr \/ CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Baylor College of Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BridgeBio Pharma \/ Baylor College of Medicine","highestDevelopmentStatusID":"3","companyTruncated":"BridgeBio Pharma \/ Baylor College of Medicine"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Renaissance Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Design Therapeutics","amount2":0.23000000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Design Therapeutics \/ Renaissance Capital","highestDevelopmentStatusID":"2","companyTruncated":"Design Therapeutics \/ Renaissance Capital"},{"orgOrder":0,"company":"Serotiny","sponsor":"Tessera Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Serotiny","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Serotiny \/ Tessera Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Serotiny \/ Tessera Therapeutics"},{"orgOrder":0,"company":"Ceptur Therapeutics","sponsor":"Venbio Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Ceptur Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Ceptur Therapeutics \/ Venbio Partners","highestDevelopmentStatusID":"2","companyTruncated":"Ceptur Therapeutics \/ Venbio Partners"},{"orgOrder":0,"company":"NeuBase Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"NeuBase Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NeuBase Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"NeuBase Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Shape Therapeutics","sponsor":"Decheng Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Shape Therapeutics","amount2":0.11,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"Shape Therapeutics \/ Decheng Capital","highestDevelopmentStatusID":"2","companyTruncated":"Shape Therapeutics \/ Decheng Capital"},{"orgOrder":0,"company":"PAQ Therapeutics","sponsor":"Sherpa Healthcare","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"PAQ Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Undisclosed","sponsorNew":"PAQ Therapeutics \/ Sherpa Healthcare","highestDevelopmentStatusID":"2","companyTruncated":"PAQ Therapeutics \/ Sherpa Healthcare"},{"orgOrder":0,"company":"Myosana Therapeutics","sponsor":"John Ballantyne","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Myosana Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"Myosana Therapeutics \/ John Ballantyne","highestDevelopmentStatusID":"2","companyTruncated":"Myosana Therapeutics \/ John Ballantyne"},{"orgOrder":0,"company":"Altay Therapeutics","sponsor":"SOLVE FSHD","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Altay Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Altay Therapeutics \/ SOLVE FSHD","highestDevelopmentStatusID":"2","companyTruncated":"Altay Therapeutics \/ SOLVE FSHD"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Genetic Cures for Kids","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Charles River Laboratories, Inc \/ Genetic Cures for Kids","highestDevelopmentStatusID":"4","companyTruncated":"Charles River Laboratories, Inc \/ Genetic Cures for Kids"},{"orgOrder":0,"company":"AavantiBio","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"AavantiBio","amount2":0.11,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"AavantiBio \/ Sarepta Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"AavantiBio \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Entos Pharmaceuticals","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Entos Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Entos Pharmaceuticals \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Entos Pharmaceuticals \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Mammoth Biosciences","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Mammoth Biosciences","amount2":0.68999999999999995,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.68999999999999995,"dosageForm":"Undisclosed","sponsorNew":"Mammoth Biosciences \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"Mammoth Biosciences \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"hC Bioscience","sponsor":"Arch Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"hC Bioscience","amount2":0.02,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Undisclosed","sponsorNew":"hC Bioscience \/ Arch Ventures","highestDevelopmentStatusID":"1","companyTruncated":"hC Bioscience \/ Arch Ventures"},{"orgOrder":0,"company":"Vigene Bioscience","sponsor":"ASC Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vigene Bioscience","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vigene Bioscience \/ ASC Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Vigene Bioscience \/ ASC Therapeutics"},{"orgOrder":0,"company":"Myosana Therapeutics","sponsor":"Cureduchenne Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Myosana Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Myosana Therapeutics \/ Cureduchenne Ventures","highestDevelopmentStatusID":"2","companyTruncated":"Myosana Therapeutics \/ Cureduchenne Ventures"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Affinia Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Affinia Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"Affinia Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Cureduchenne Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dyne Therapeutics \/ Cureduchenne Ventures","highestDevelopmentStatusID":"1","companyTruncated":"Dyne Therapeutics \/ Cureduchenne Ventures"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Precision BioSciences","amount2":0.56000000000000005,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.56000000000000005,"dosageForm":"Undisclosed","sponsorNew":"Precision BioSciences \/ Eli Lilly","highestDevelopmentStatusID":"4","companyTruncated":"Precision BioSciences \/ Eli Lilly"},{"orgOrder":0,"company":"Replay Bio","sponsor":"KKR","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Replay Bio","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Replay Bio \/ KKR","highestDevelopmentStatusID":"1","companyTruncated":"Replay Bio \/ KKR"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Funding","leadProduct":"Undisclosed","moa":"G542X","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Prime Medicine","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Undisclosed","sponsorNew":"Prime Medicine \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Prime Medicine \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Neucore Bio","sponsor":"National Science Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Neucore Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neucore Bio \/ National Science Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Neucore Bio \/ National Science Foundation"},{"orgOrder":0,"company":"Endure Bio","sponsor":"National Cancer Institute","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2025","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Endure Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Endure Bio \/ National Cancer Institute","highestDevelopmentStatusID":"4","companyTruncated":"Endure Bio \/ National Cancer Institute"},{"orgOrder":0,"company":"Neucore Bio","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Neucore Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neucore Bio \/ National Institutes of Health","highestDevelopmentStatusID":"4","companyTruncated":"Neucore Bio \/ National Institutes of Health"},{"orgOrder":0,"company":"Chris Goss","sponsor":"Cystic Fibrosis Foundation | CF Therapeutics Development Network Coordinating Center | Medical University of South Carolina | University of Washington","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Chris Goss","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chris Goss \/ Cystic Fibrosis Foundation | CF Therapeutics Development Network Coordinating Center | Medical University of South Carolina | University of Washington","highestDevelopmentStatusID":"11","companyTruncated":"Chris Goss \/ Cystic Fibrosis Foundation | CF Therapeutics Development Network Coordinating Center | Medical University of South Carolina | University of Washington"},{"orgOrder":0,"company":"Seattle Children\u2019s Research Institute","sponsor":"Moderna Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Seattle Children\u2019s Research Institute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Seattle Children\u2019s Research Institute \/ Moderna Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Seattle Children\u2019s Research Institute \/ Moderna Therapeutics"},{"orgOrder":0,"company":"LambdaVision","sponsor":"NASA","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"LambdaVision","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"LambdaVision \/ NASA","highestDevelopmentStatusID":"4","companyTruncated":"LambdaVision \/ NASA"},{"orgOrder":0,"company":"Ocugen","sponsor":"Kwangdong Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Licensing Agreement","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0.01,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Kwangdong Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Kwangdong Pharmaceutical"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Public Offering","leadProduct":"Delpacibart Zotadirsen","moa":"Dystrophin exon-44","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0.68999999999999995,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.68999999999999995,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Leerink Partners"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Multicare","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Italfarmaco S.p.A","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Italfarmaco S.p.A \/ Multicare","highestDevelopmentStatusID":"15","companyTruncated":"Italfarmaco S.p.A \/ Multicare"},{"orgOrder":0,"company":"Shape Therapeutics","sponsor":"VectorY","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Licensing Agreement","leadProduct":"VTx-003","moa":"\u03b1v\u03b21","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Shape Therapeutics","amount2":1.2,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.2,"dosageForm":"Undisclosed","sponsorNew":"Shape Therapeutics \/ VectorY","highestDevelopmentStatusID":"2","companyTruncated":"Shape Therapeutics \/ VectorY"},{"orgOrder":0,"company":"Ensoma","sponsor":"Gilead Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Financing","leadProduct":"EN-374","moa":"CYBB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ensoma","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Infusion","sponsorNew":"Ensoma \/ Gilead Sciences","highestDevelopmentStatusID":"7","companyTruncated":"Ensoma \/ Gilead Sciences"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Kinea Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Licensing Agreement","leadProduct":"KNA-155","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Solid Biosciences \/ Kinea Bio","highestDevelopmentStatusID":"4","companyTruncated":"Solid Biosciences \/ Kinea Bio"},{"orgOrder":0,"company":"Genfit","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"VS-01","moa":"Ammonia clearance","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Genfit","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genfit \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Genfit \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"NS-051\/NCNP-04","moa":"DMD exon 51","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"INV-347","moa":"CB1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Shattuck Labs","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"SL-325","moa":"DR3","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shattuck Labs","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Shattuck Labs \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Shattuck Labs \/ Undisclosed"},{"orgOrder":0,"company":"Versiti","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"F10","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Versiti","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Versiti \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Versiti \/ Undisclosed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Atumelnant","moa":"MC2R","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Crinetics Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Crinetics Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"VX-407","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Pharmbio Korea","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Levodropropizine","moa":"C-fiber sensory nerves","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pharmbio Korea","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pharmbio Korea \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Pharmbio Korea \/ Undisclosed"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"AAVB-039","moa":"ABCA4","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AAVantgarde Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"CHF6467","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chiesi Group \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Chiesi Group \/ Undisclosed"},{"orgOrder":0,"company":"QED Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"QED Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"QED Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"QED Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GeneCradle Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"GC310","moa":"ATP7B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GeneCradle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneCradle Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GeneCradle Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Laruparetigene Zosaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"CorrectSequence Therapeutics","sponsor":"The First Affiliated Hospital of Anhui Medical University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"CS-121","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"CorrectSequence Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CorrectSequence Therapeutics \/ The First Affiliated Hospital of Anhui Medical University","highestDevelopmentStatusID":"6","companyTruncated":"CorrectSequence Therapeutics \/ The First Affiliated Hospital of Anhui Medical University"},{"orgOrder":0,"company":"Alesta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"ALE1","moa":"PPi","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alesta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alesta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Alesta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SGT-212","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Jemincare","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Amiodarone Hydrochloride","moa":"HERG","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Jemincare","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Jemincare \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Jemincare \/ Undisclosed"},{"orgOrder":0,"company":"Adventia Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Dietary Supplement","year":"2025","type":"Inapplicable","leadProduct":"Eicosapentaenoic Acid|Docosahexaenoic Acid","moa":"Lipid|PPAR-gamma","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Adventia Pharma","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oil","sponsorNew":"Adventia Pharma \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Adventia Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Cagrilintide","moa":"GLP-1 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Crinecerfont","moa":"Corticotropin releasing factor receptor 1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Neurocrine Biosciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Neurocrine Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"DISC-3405","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Disc medicine \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Disc medicine \/ Undisclosed"},{"orgOrder":0,"company":"Shanghai Siponuoyin Biotechnology Co Ltd","sponsor":"Shanghai Children's Medical Center","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SPOT-mRNA03","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shanghai Siponuoyin Biotechnology Co Ltd","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Shanghai Siponuoyin Biotechnology Co Ltd \/ Shanghai Children's Medical Center","highestDevelopmentStatusID":"6","companyTruncated":"Shanghai Siponuoyin Biotechnology Co Ltd \/ Shanghai Children's Medical Center"},{"orgOrder":0,"company":"Beijing Inno Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"YN001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Beijing Inno Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Beijing Inno Medicine \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Beijing Inno Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid, Suspension","sponsorNew":"Applied Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Angelini Pharma","sponsor":"Cross Research S.A.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Benzydamine Hydrochloride","moa":"Cyclooxygenase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Angelini Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Angelini Pharma \/ Cross Research S.A.","highestDevelopmentStatusID":"6","companyTruncated":"Angelini Pharma \/ Cross Research S.A."},{"orgOrder":0,"company":"Kinea Bio","sponsor":"Jain Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Funding","leadProduct":"KNA-155","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Kinea Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kinea Bio \/ Jain Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Kinea Bio \/ Jain Foundation"},{"orgOrder":0,"company":"Erkim","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"TURKEY","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"Fosdenopterin","moa":"||Molybdenum cofactor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Erkim","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Erkim \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Erkim \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"Uniqure","sponsor":"Hercules Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Financing","leadProduct":"Antimony pentoxide","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Hercules Capital","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Hercules Capital"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Public Offering","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0.13,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Tablet, Film Coated","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Public Offering","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Leerink Partners"},{"orgOrder":0,"company":"PepGen","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Public Offering","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Leerink Partners"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Public Offering","leadProduct":"Sebetralstat","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KalVista Pharmaceuticals","amount2":0.11,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Tablet, Film Coated","sponsorNew":"KalVista Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KalVista Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Public Offering","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0.12,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Leerink Partners"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"R-Bridge Healthcare Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Steroid","year":"2025","type":"Financing","leadProduct":"Vamorolone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0.029999999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ R-Bridge Healthcare Fund","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ R-Bridge Healthcare Fund"},{"orgOrder":0,"company":"Uniqure","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"Antimony pentoxide","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Leerink Partners","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Leerink Partners"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Financing","leadProduct":"RCT2100","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"7","companyTruncated":"ReCode Therapeutics \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Star Therapeutics","sponsor":"Sanofi Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Series D Financing","leadProduct":"VGA039","moa":"PROS1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Star Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Star Therapeutics \/ Sanofi Ventures","highestDevelopmentStatusID":"10","companyTruncated":"Star Therapeutics \/ Sanofi Ventures"},{"orgOrder":0,"company":"Cure Rare","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"CRD-003","moa":"FKRP","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cure Rare","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cure Rare \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Cure Rare \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Evinacumab","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zydus Lifesciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Steroid","year":"2025","type":"Inapplicable","leadProduct":"Deflazacort","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Zydus Lifesciences","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Zydus Lifesciences \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Zydus Lifesciences \/ Undisclosed"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Private Placement","leadProduct":"Tralesinidase Alfa","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Infusion","sponsorNew":"Spruce Biosciences \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Leerink Partners"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Menagen Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2025","type":"Licensing Agreement","leadProduct":"Agalsidase Beta","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Menagen Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"JCR Pharmaceuticals \/ Menagen Pharmaceutical"},{"orgOrder":0,"company":"Ribomic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Umedaptanib Pegol","moa":"FGF2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ribomic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ribomic \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ribomic \/ Undisclosed"},{"orgOrder":0,"company":"Kedrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"IgG","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Kedrion","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kedrion \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Kedrion \/ Undisclosed"},{"orgOrder":0,"company":"Star Therapeutics","sponsor":"GordonMD Global Investments","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Series D Financing","leadProduct":"VGA039","moa":"PROS1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Star Therapeutics","amount2":0.13,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"Injection","sponsorNew":"Star Therapeutics \/ GordonMD Global Investments","highestDevelopmentStatusID":"10","companyTruncated":"Star Therapeutics \/ GordonMD Global Investments"},{"orgOrder":0,"company":"Harness Therapeutics","sponsor":"Ono Venture Investment","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2025","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Harness Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Harness Therapeutics \/ Ono Venture Investment","highestDevelopmentStatusID":"2","companyTruncated":"Harness Therapeutics \/ Ono Venture Investment"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Biomedica","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Steroid","year":"2025","type":"Collaboration","leadProduct":"Vamorolone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Santhera Pharmaceuticals \/ Biomedica","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Biomedica"},{"orgOrder":0,"company":"Tonix Pharmaceuticals Holding Corp","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2025","type":"Inapplicable","leadProduct":"Oxytocin","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tonix Pharmaceuticals Holding Corp","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"Tonix Pharmaceuticals Holding Corp \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Tonix Pharmaceuticals Holding Corp \/ Undisclosed"},{"orgOrder":0,"company":"Souffle Therapeutics","sponsor":"Souffle Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Souffle Therapeutics","amount2":0.20000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Souffle Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Souffle Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Karuna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Xanomeline","moa":"Muscarinic acetylcholine receptor M1 | Muscarinic acetylcholine receptor M4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Karuna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Karuna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Karuna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Navenibart","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"MRM-3379","moa":"Type 4 cyclic nucleotide phosphodiesterase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Anebulo Pharmaceuticals","sponsor":"National Institute on Drug Abuse","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2025","type":"Inapplicable","leadProduct":"Selonabant","moa":"CNR1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Anebulo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Anebulo Pharmaceuticals \/ National Institute on Drug Abuse","highestDevelopmentStatusID":"6","companyTruncated":"Anebulo Pharmaceuticals \/ National Institute on Drug Abuse"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"BAY 3401016","moa":"SEMA3A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Macupatide","moa":"GIPR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eli Lilly \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eli Lilly \/ Undisclosed"},{"orgOrder":0,"company":"The Emmes Company, LLC","sponsor":"Rachel G. Greenberg, MD, MB, MHS","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Guanfacine","moa":"Adrenergic receptor alpha-2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"The Emmes Company, LLC","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"The Emmes Company, LLC \/ Rachel G. Greenberg, MD, MB, MHS","highestDevelopmentStatusID":"8","companyTruncated":"The Emmes Company, LLC \/ Rachel G. Greenberg, MD, MB, MHS"},{"orgOrder":0,"company":"GSK","sponsor":"Lucas Otieno Tina, MD MSc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Lofexidine","moa":"Adrenergic receptor alpha-2","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GSK \/ Lucas Otieno Tina, MD MSc","highestDevelopmentStatusID":"1","companyTruncated":"GSK \/ Lucas Otieno Tina, MD MSc"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Scott R. Plotkin, MD, PhD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Brigatinib","moa":"ALK tyrosine kinase receptor | Epidermal growth factor receptor erbB1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Takeda Pharmaceutical \/ Scott R. Plotkin, MD, PhD","highestDevelopmentStatusID":"8","companyTruncated":"Takeda Pharmaceutical \/ Scott R. Plotkin, MD, PhD"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Michael Rafii, MD, PhD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Donanemab","moa":"Amyloid-beta A4 protein","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Eli Lilly \/ Michael Rafii, MD, PhD","highestDevelopmentStatusID":"11","companyTruncated":"Eli Lilly \/ Michael Rafii, MD, PhD"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Daniel Gaudet","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Evinacumab","moa":"Angiopoietin-related protein 3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Daniel Gaudet","highestDevelopmentStatusID":"15","companyTruncated":"Ultragenyx Pharmaceutical \/ Daniel Gaudet"},{"orgOrder":0,"company":"Genentech","sponsor":"Rajshekhar Chakraborty, MD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Venetoclax","moa":"Apoptosis regulator Bcl-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Genentech \/ Rajshekhar Chakraborty, MD","highestDevelopmentStatusID":"7","companyTruncated":"Genentech \/ Rajshekhar Chakraborty, MD"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Alfred Chung","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Venetoclax","moa":"Apoptosis regulator Bcl-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Alfred Chung","highestDevelopmentStatusID":"7","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Alfred Chung"},{"orgOrder":0,"company":"Paratek Pharmaceuticals","sponsor":"Paul Beringer","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2020","type":"Inapplicable","leadProduct":"Omadacycline","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Paratek Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Paratek Pharmaceuticals \/ Paul Beringer","highestDevelopmentStatusID":"11","companyTruncated":"Paratek Pharmaceuticals \/ Paul Beringer"},{"orgOrder":0,"company":"Merck & Co","sponsor":"Joseph Kuti","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Ceftolozane Sulfate","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Merck & Co","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Merck & Co \/ Joseph Kuti","highestDevelopmentStatusID":"11","companyTruncated":"Merck & Co \/ Joseph Kuti"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"GWT-TUD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ GWT-TUD","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ GWT-TUD"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Region Skane","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Trametinib","moa":"Dual specificity mitogen-activated protein kinase kinase; MEK1\/2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Region Skane","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Region Skane"},{"orgOrder":0,"company":"Audentes Therapeutics","sponsor":"Megan Waldrop","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"scAAV9.U7.ACCA","moa":"Dystrophin exon-51","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Audentes Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Audentes Therapeutics \/ Megan Waldrop","highestDevelopmentStatusID":"7","companyTruncated":"Audentes Therapeutics \/ Megan Waldrop"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Kevin Flanigan","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2019","type":"Inapplicable","leadProduct":"Casimersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Kevin Flanigan","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Kevin Flanigan"},{"orgOrder":0,"company":"Healx","sponsor":"Craig Erickson","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Gaboxadol","moa":"GABA-A receptor; agonist GABA site","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Healx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Healx \/ Craig Erickson","highestDevelopmentStatusID":"8","companyTruncated":"Healx \/ Craig Erickson"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Filip Krag Knop","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2021","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zealand Pharma \/ Filip Krag Knop","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Filip Krag Knop"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Ari Zimran","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2019","type":"Inapplicable","leadProduct":"Taliglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Ari Zimran","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Ari Zimran"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Jerry Vockley","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Acer Therapeutics \/ Jerry Vockley","highestDevelopmentStatusID":"8","companyTruncated":"Acer Therapeutics \/ Jerry Vockley"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Jerry Vockley","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zevra Therapeutics \/ Jerry Vockley","highestDevelopmentStatusID":"8","companyTruncated":"Zevra Therapeutics \/ Jerry Vockley"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Elizabeth Yang","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Global Blood Therapeutics \/ Elizabeth Yang","highestDevelopmentStatusID":"15","companyTruncated":"Global Blood Therapeutics \/ Elizabeth Yang"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Amy Tang","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Amy Tang","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Amy Tang"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"Ala'a Sharara","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Sofosbuvir","moa":"Hepatitis C virus NS5B RNA-dependent RNA polymerase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gilead Sciences \/ Ala'a Sharara","highestDevelopmentStatusID":"11","companyTruncated":"Gilead Sciences \/ Ala'a Sharara"},{"orgOrder":0,"company":"Secura Bio","sponsor":"Abdullah Kutlar","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Panobinostat lactate","moa":"Histone deacetylase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Secura Bio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Secura Bio \/ Abdullah Kutlar","highestDevelopmentStatusID":"6","companyTruncated":"Secura Bio \/ Abdullah Kutlar"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Lynda E Polgreen","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"Anakinra","moa":"Interleukin-1 receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Swedish Orphan Biovitrum AB \/ Lynda E Polgreen","highestDevelopmentStatusID":"7","companyTruncated":"Swedish Orphan Biovitrum AB \/ Lynda E Polgreen"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Michael Campos, MD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Alpha 1-Antitrypsin","moa":"Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Michael Campos, MD","highestDevelopmentStatusID":"8","companyTruncated":"CSL Behring \/ Michael Campos, MD"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Elizabeth Berry-Kravis","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Mavoglurant","moa":"Metabotropic glutamate receptor 5","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Elizabeth Berry-Kravis","highestDevelopmentStatusID":"9","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Elizabeth Berry-Kravis"},{"orgOrder":0,"company":"Attralus","sponsor":"Sharmila Dorbala","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Radiolabeled Compound","year":"2025","type":"Inapplicable","leadProduct":"124-I Evuzamitide","moa":"MUC1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Attralus \/ Sharmila Dorbala","highestDevelopmentStatusID":"10","companyTruncated":"Attralus \/ Sharmila Dorbala"},{"orgOrder":0,"company":"Dompe Farmaceutici","sponsor":"Ospedale San Raffaele","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Recombinant Human Nerve Growth Factor","moa":"Neurotrophic tyrosine kinase receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Dompe Farmaceutici","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Dompe Farmaceutici \/ Ospedale San Raffaele","highestDevelopmentStatusID":"8","companyTruncated":"Dompe Farmaceutici \/ Ospedale San Raffaele"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Linda Randolph","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Linda Randolph","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Linda Randolph"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"John Mark Sloan","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Idelalisib","moa":"PI3-kinase p110-delta subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gilead Sciences \/ John Mark Sloan","highestDevelopmentStatusID":"8","companyTruncated":"Gilead Sciences \/ John Mark Sloan"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Romain Lazor","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Nintedanib Esylate","moa":"Platelet-derived growth factor receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Romain Lazor","highestDevelopmentStatusID":"8","companyTruncated":"Boehringer Ingelheim GmbH \/ Romain Lazor"},{"orgOrder":0,"company":"Lingyi Biotech","sponsor":"He Huang","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"LY-M001","moa":"SARS-CoV-2 spike protein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lingyi Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lingyi Biotech \/ He Huang","highestDevelopmentStatusID":"7","companyTruncated":"Lingyi Biotech \/ He Huang"},{"orgOrder":0,"company":"H. Lundbeck AS","sponsor":"William Ondo, MD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Tetrabenazine","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"H. Lundbeck AS","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pill","sponsorNew":"H. Lundbeck AS \/ William Ondo, MD","highestDevelopmentStatusID":"11","companyTruncated":"H. Lundbeck AS \/ William Ondo, MD"},{"orgOrder":0,"company":"Genentech","sponsor":"Jesse Roman","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Pirfenidone","moa":"TNF","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Genentech \/ Jesse Roman","highestDevelopmentStatusID":"8","companyTruncated":"Genentech \/ Jesse Roman"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Adrian Lacy","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Adrian Lacy","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Adrian Lacy"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Jerry Vockley","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Jerry Vockley","highestDevelopmentStatusID":"8","companyTruncated":"Ultragenyx Pharmaceutical \/ Jerry Vockley"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Areeg El-Gharbawy","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Areeg El-Gharbawy","highestDevelopmentStatusID":"6","companyTruncated":"Ultragenyx Pharmaceutical \/ Areeg El-Gharbawy"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Jerry Vockley","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Jerry Vockley","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Jerry Vockley"},{"orgOrder":0,"company":"Incyte Corporation","sponsor":"Aaron Logan","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Ruxolitinib Phosphate","moa":"Tyrosine-protein kinase JAK1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Incyte Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Incyte Corporation \/ Aaron Logan","highestDevelopmentStatusID":"8","companyTruncated":"Incyte Corporation \/ Aaron Logan"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"David Williams","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Autologous CD34+ Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ David Williams","highestDevelopmentStatusID":"8","companyTruncated":"Bluebird Bio \/ David Williams"},{"orgOrder":0,"company":"Cerus Corporation","sponsor":"Marianne Yee","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Biotin Labeled Red Blood Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Cerus Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cerus Corporation \/ Marianne Yee","highestDevelopmentStatusID":"6","companyTruncated":"Cerus Corporation \/ Marianne Yee"},{"orgOrder":0,"company":"Catalyst Pharmaceuticals","sponsor":"Ricardo Maselli","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"Diaminopyridine","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Catalyst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Catalyst Pharmaceuticals \/ Ricardo Maselli","highestDevelopmentStatusID":"1","companyTruncated":"Catalyst Pharmaceuticals \/ Ricardo Maselli"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Kathryn Wagner","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"Domagrozumab","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Kathryn Wagner","highestDevelopmentStatusID":"7","companyTruncated":"Pfizer Inc \/ Kathryn Wagner"},{"orgOrder":0,"company":"Grifols International","sponsor":"Deepa Manwani","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Deepa Manwani","highestDevelopmentStatusID":"7","companyTruncated":"Grifols International \/ Deepa Manwani"},{"orgOrder":0,"company":"NuvOx Pharma","sponsor":"Amma Owusu-Ansah","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"NVX-508","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"NuvOx Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Emulsion","sponsorNew":"NuvOx Pharma \/ Amma Owusu-Ansah","highestDevelopmentStatusID":"6","companyTruncated":"NuvOx Pharma \/ Amma Owusu-Ansah"},{"orgOrder":0,"company":"SOLUTEX GC SL","sponsor":"Robin E. Miller","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"SCD-Omegatex","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SOLUTEX GC SL","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"SOLUTEX GC SL \/ Robin E. Miller","highestDevelopmentStatusID":"7","companyTruncated":"SOLUTEX GC SL \/ Robin E. Miller"},{"orgOrder":0,"company":"Savara","sponsor":"Schmidhofer, Mark, MD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Sodium Nitrite","moa":"Vasodilator","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Savara","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Savara \/ Schmidhofer, Mark, MD","highestDevelopmentStatusID":"7","companyTruncated":"Savara \/ Schmidhofer, Mark, MD"},{"orgOrder":0,"company":"Richmond Pharmacology","sponsor":"Dr Jorg Taubel","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Richmond Pharmacology","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Richmond Pharmacology \/ Dr Jorg Taubel","highestDevelopmentStatusID":"1","companyTruncated":"Richmond Pharmacology \/ Dr Jorg Taubel"},{"orgOrder":0,"company":"UgeneX Therapeutics","sponsor":"AviadoBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Licensing Agreement","leadProduct":"UGX-202","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"UgeneX Therapeutics","amount2":0.40999999999999998,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.40999999999999998,"dosageForm":"Injection","sponsorNew":"UgeneX Therapeutics \/ AviadoBio","highestDevelopmentStatusID":"6","companyTruncated":"UgeneX Therapeutics \/ AviadoBio"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Funding","leadProduct":"4D-710","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Inhalation","sponsorNew":"4D Molecular Therapeutics \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"BioCryst Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Acquisition","leadProduct":"Navenibart","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0.69999999999999996,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.69999999999999996,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ BioCryst Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ABO-503","moa":"RS1 gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Abeona Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Abeona Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReviR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"RTX-117","moa":"eIF2B","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReviR Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ReviR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"ReviR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Jefferies | Leerink Partners | Morgan Stanley | Cantor","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0.25,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.25,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Jefferies | Leerink Partners | Morgan Stanley | Cantor","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ Jefferies | Leerink Partners | Morgan Stanley | Cantor"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Jefferies | Leerink Partners | Morgan Stanley | Cantor","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0.22,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.25,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Jefferies | Leerink Partners | Morgan Stanley | Cantor","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ Jefferies | Leerink Partners | Morgan Stanley | Cantor"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Acquisition","leadProduct":"Delpacibart Zotadirsen","moa":"Dystrophin exon-44","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":12,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":12,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Novartis Pharmaceuticals Corporation"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

    Xls
    Filters Filter
    ×
    FILTER:
    filter
    Company Name
      filter

      Year

        filter

        DEALS // DEV.

          filter

          Country

            filter
            Sponsor
              filter

              Therapeutic Area

                filter

                Study Phase

                  filter

                  Deal Type

                    filter

                    Product Type

                      filter

                      Dosage Form

                        filter

                        Lead Product

                          filter

                          Target

                            Loading...
                            refresh Filters
                            refresh Reset Filter
                            refresh Reset Filter
                            price-trend All Developments

                            Therapeutic Area by Lead Product

                            Study Phase by Lead Product

                            Company by Lead Product

                            Top Deals by Deal Size (USD bn)

                            01

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Rilzabrutinib

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efficacy & Safety of Rilzabrutinib in Patients with Sickle-cell Disease

                            Details : Rilzabrutinib is a Other Small Molecule drug candidate, which is currently being evaluated in Phase III clinical studies for the treatment of Anemia, Sickle Cell.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 16, 2025

                            Lead Product(s) : Rilzabrutinib

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            02

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alfa

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sp...

                            Details : Olipudase Alfa is a Enzyme drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Niemann-Pick Diseases.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            April 29, 2025

                            Lead Product(s) : Olipudase Alfa

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            03

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alfa

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy

                            Details : Efanesoctocog Alfa is a Protein drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Hemophilia A.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            April 24, 2025

                            Lead Product(s) : Efanesoctocog Alfa

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            04

                            AbbVie Inc

                            U.S.A
                            ISPE Annual Meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : ABBV-CLS-628

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Calico Life Sciences

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Assess Adverse Events and Effectiveness of IntraVenous Infusions of ABBV-CLS-628 in Adult Participa...

                            Details : ABBV-CLS-628 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Polycystic Kidney, Autosomal Dominant.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            March 30, 2025

                            Lead Product(s) : ABBV-CLS-628

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Calico Life Sciences

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Abbvie Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            05

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : SAR446268

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-m...

                            Details : SAR446268 is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Myotonic Dystrophy.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            February 25, 2025

                            Lead Product(s) : SAR446268

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            06

                            AbbVie Inc

                            U.S.A
                            ISPE Annual Meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Fosigotifator

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Expanded Access to Fosigotifator

                            Details : Fosigotifator is a Other Small Molecule drug candidate, which is currently being evaluated in clinical studies for the treatment of Leukoencephalopathies.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            September 19, 2024

                            Lead Product(s) : Fosigotifator

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Abbvie Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            07

                            AbbVie Inc

                            U.S.A
                            ISPE Annual Meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Galicaftor,Navocaftor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Sionna Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Sionna Expands CF Pipeline with AbbVie License Agreement

                            Details : Under the terms of the agreement, Sionna will assume all development for ABBV-2222 (galicaftor), which is being evaluated in combination with navocaftor for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Undisclosed

                            July 16, 2024

                            Lead Product(s) : Galicaftor,Navocaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Sionna Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            Abbvie Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            08

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth

                            Details : Eliglustat is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Gaucher Disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            January 05, 2024

                            Lead Product(s) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            09

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or...

                            Details : Eliglustat is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Gaucher Disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            January 03, 2024

                            Lead Product(s) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            10

                            AbbVie Inc

                            U.S.A
                            ISPE Annual Meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Etentamig

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Assess Change in Disease Activity and Adverse Events (AE)s in Adult Participants With Immunoglobuli...

                            Details : Etentamig is a Antibody drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Immunoglobulin Light-chain Amyloidosis.

                            Product Name : Undisclosed

                            Product Type : Antibody, Unconjugated

                            Upfront Cash : Inapplicable

                            December 06, 2023

                            Lead Product(s) : Etentamig

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Abbvie Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth