menu
    • menu
    [{"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":"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":"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":"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":"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":"Rachel G. Greenberg, MD, MB, MHS","sponsor":"The Emmes Company, LLC | Gansu Provincial Maternal and Child Health Care Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Guanfacine","moa":"Adrenergic receptor alpha-2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rachel G. Greenberg, MD, MB, MHS","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Rachel G. Greenberg, MD, MB, MHS \/ The Emmes Company, LLC | Gansu Provincial Maternal and Child Health Care Hospital","highestDevelopmentStatusID":"8","companyTruncated":"Rachel G. Greenberg, MD, MB, MHS \/ The Emmes Company, LLC | Gansu Provincial Maternal and Child Health Care Hospital"},{"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":"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":"Scott R. Plotkin, MD, PhD","sponsor":"Takeda Pharmaceutical | The Children's Tumor Foundation | National Comprehensive Cancer Network","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","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":"Scott R. Plotkin, MD, PhD","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Scott R. Plotkin, MD, PhD \/ Takeda Pharmaceutical | The Children's Tumor Foundation | National Comprehensive Cancer Network","highestDevelopmentStatusID":"8","companyTruncated":"Scott R. Plotkin, MD, PhD \/ Takeda Pharmaceutical | The Children's Tumor Foundation | National Comprehensive Cancer Network"},{"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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 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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"Craig Erickson","sponsor":"Healx","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Gaboxadol","moa":"GABA-A receptor; agonist GABA site","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Craig Erickson","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Craig Erickson \/ Healx","highestDevelopmentStatusID":"8","companyTruncated":"Craig Erickson \/ Healx"},{"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":"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":"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":"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":"Filip Krag Knop","sponsor":"Zealand Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Hormone","year":"2021","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Filip Krag Knop","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Filip Krag Knop \/ Zealand Pharma","highestDevelopmentStatusID":"8","companyTruncated":"Filip Krag Knop \/ Zealand Pharma"},{"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":"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":"Jerry Vockley","sponsor":"Acer Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Jerry Vockley","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jerry Vockley \/ Acer Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Jerry Vockley \/ Acer Therapeutics"},{"orgOrder":0,"company":"Jerry Vockley","sponsor":"Zevra Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Jerry Vockley","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jerry Vockley \/ Zevra Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Jerry Vockley \/ Zevra Therapeutics"},{"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":"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":"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":"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":"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":"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 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":"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":"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":"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":"Amy Tang","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Amy Tang","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amy Tang \/ Pfizer Inc","highestDevelopmentStatusID":"8","companyTruncated":"Amy Tang \/ Pfizer Inc"},{"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"Romain Lazor","sponsor":"Boehringer Ingelheim GmbH","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Nintedanib Esylate","moa":"Platelet-derived growth factor receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Romain Lazor","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Romain Lazor \/ Boehringer Ingelheim GmbH","highestDevelopmentStatusID":"8","companyTruncated":"Romain Lazor \/ Boehringer Ingelheim GmbH"},{"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"Jerry Vockley","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2023","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":"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":"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":"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":"Aaron Logan","sponsor":"Incyte Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Ruxolitinib Phosphate","moa":"Tyrosine-protein kinase JAK1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aaron Logan","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aaron Logan \/ Incyte Corporation","highestDevelopmentStatusID":"8","companyTruncated":"Aaron Logan \/ Incyte Corporation"},{"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":"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":"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":"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":"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 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":"Undisclosed","sponsorNew":"Attralus \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Attralus \/ Undisclosed"},{"orgOrder":0,"company":"David Williams","sponsor":"National Heart, Lung, and Blood Institute | California Institute for Regenerative Medicine (CIRM) | Bluebird Bio | Blood and Marrow Transplant Clinical Trials Network","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Autologous CD34+ Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"David Williams","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"David Williams \/ National Heart, Lung, and Blood Institute | California Institute for Regenerative Medicine (CIRM) | Bluebird Bio | Blood and Marrow Transplant Clinical Trials Network","highestDevelopmentStatusID":"8","companyTruncated":"David Williams \/ National Heart, Lung, and Blood Institute | California Institute for Regenerative Medicine (CIRM) | Bluebird Bio | Blood and Marrow Transplant Clinical Trials Network"},{"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":"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":"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":"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":"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":"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":"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":"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","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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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":"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"}]

    Find Drugs for Genetic Disease in Phase II Clinical Development

    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) : 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

                            02

                            AbbVie Inc

                            U.S.A
                            BioJapan
                            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

                            03

                            AbbVie Inc

                            U.S.A
                            BioJapan
                            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

                            04

                            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) : SAR447537

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema

                            Details : SAR447537 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of alpha 1-Antitrypsin Deficiency.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            June 09, 2023

                            Lead Product(s) : SAR447537

                            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

                            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) : SAR447537

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema

                            Details : SAR447537 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of alpha 1-Antitrypsin Deficiency.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            May 12, 2023

                            Lead Product(s) : SAR447537

                            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

                            06

                            AbbVie Inc

                            U.S.A
                            BioJapan
                            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

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate Adverse Events and Change in Disease Activity With Oral Capsules of Galicaftor/Navocaftor/AB...

                            Details : Galicaftor is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            April 21, 2021

                            Lead Product(s) : Galicaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Abbvie Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            07

                            Avidity Biosciences

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            Avidity Biosciences

                            U.S.A arrow
                            BioJapan
                            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) : Delpacibart Zotadirsen

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Leerink Partners

                            Deal Size : $690.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Avidity Biosciences Announces Closing of Upsized Public Offering of Common Stock

                            Details : Avidity intends to use the net proceeds to advance the its three late-stage clinical programs, including AOC 1044 (delpacibart zotadirsen), designed to deliver phosphorodiamidate morpholino oligomers.

                            Product Name : AOC 1044

                            Product Type : Antibody-oligonucleotide Conjugate

                            Upfront Cash : Undisclosed

                            September 16, 2025

                            Lead Product(s) : Delpacibart Zotadirsen

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Leerink Partners

                            Deal Size : $690.0 million

                            Deal Type : Public Offering

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            08

                            Jerry Vockley

                            Country arrow
                            BioJapan
                            Not Confirmed

                            Jerry Vockley

                            Country arrow
                            BioJapan
                            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) : Triheptanoin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of Triheptanoin Use in Patients with Medium-Chain Acyl-CoA Dehydrogenase Deficiency

                            Details : Triheptanoin is a Other Small Molecule drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Medium chain acyl CoA dehydrogenase Deficiency.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            July 31, 2025

                            Lead Product(s) : Triheptanoin

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            09

                            Novartis Pharmaceuticals Corporation

                            Switzerland arrow
                            BioJapan
                            Not Confirmed

                            Novartis Pharmaceuticals Corporation

                            Switzerland arrow
                            BioJapan
                            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) : Alpelisib

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Alpelisib (BYL719) Safety & Efficacy in Pediatric & Adult PROS Patients

                            Details : Alpelisib is a Other Small Molecule drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of PIK3CA-mutated Overgrowth Spectrum.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 30, 2025

                            Lead Product(s) : Alpelisib

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            10

                            Ascendis Pharma

                            Denmark arrow
                            BioJapan
                            Not Confirmed

                            Ascendis Pharma

                            Denmark arrow
                            BioJapan
                            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) : Navepegritide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            TransCon CNP Shows Growth & Bone Gains in Children with Achondroplasia

                            Details : TransCon CNP (navepegritide) is an investigational prodrug of CNP administered once weekly providing continuous exposure of active CNP to receptors on tissues throughout the body.

                            Product Name : TransCon CNP

                            Product Type : Peptide, Unconjugated

                            Upfront Cash : Inapplicable

                            May 13, 2025

                            Lead Product(s) : Navepegritide

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth