[{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Lysogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Termination","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Sarepta Therapeutics \/ Lysogene","highestDevelopmentStatusID":"9","companyTruncated":"Sarepta Therapeutics \/ Lysogene"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Partnership","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0.81000000000000005,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.81000000000000005,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Regenxbio \/ Nippon Shinyaku","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Nippon Shinyaku"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"RGX-121","moa":"Iduronate 2-sulfatase (IDS)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Azidus Brazil","sponsor":"Cellavita","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Mesenchymal Stem Cell","moa":"Stem cell proliferation","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Azidus Brazil","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"Azidus Brazil \/ Cellavita","highestDevelopmentStatusID":"9","companyTruncated":"Azidus Brazil \/ Cellavita"},{"orgOrder":0,"company":"Avrobio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Avrobio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Organovo Holdings","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Series C Financing","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0.12,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Organovo Holdings","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Organovo Holdings"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Public Offering","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Glycomine","sponsor":"CTI Life Sciences Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Series C Financing","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0.12,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Intravenous Infusion","sponsorNew":"Glycomine \/ CTI Life Sciences Fund","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ CTI Life Sciences Fund"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Seelos Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Seelos Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"The Lundquist Institute","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Anakinra","moa":"Interleukin-1 receptor","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"The Lundquist Institute","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"The Lundquist Institute \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"The Lundquist Institute \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Public Offering","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0.28000000000000003,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.32000000000000001,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Goldman Sachs & Co","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Public Offering","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0.32000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.32000000000000001,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Goldman Sachs & Co","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Idursulfase","moa":"||IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Private Placement","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0.5,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.5,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Tividenofusp Alfa","moa":"IDS*","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Tividenofusp Alfa","moa":"IDS*","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"CSL889","moa":"Hemojuvelin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"CSL Behring \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"CSL Behring \/ Inapplicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Glycomine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Inapplicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Glycomine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Inapplicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Glycomine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Inapplicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Glycomine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Inapplicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Glycomine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Inapplicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Glycomine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Inapplicable"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Seelos Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Seelos Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Avrobio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Inapplicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Avrobio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Inapplicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Avrobio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Inapplicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Avrobio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Inapplicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-02","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Avrobio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Avrobio \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Portfolio Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Syncona Portfolio Limited","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Syncona Portfolio Limited"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Portfolio Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Syncona Portfolio Limited","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Syncona Portfolio Limited"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"University College London","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ University College London","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ University College London"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Tividenofusp Alfa","moa":"IDS*","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"CSL889","moa":"Hemojuvelin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"CSL Behring \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"CSL Behring \/ Inapplicable"},{"orgOrder":0,"company":"Generium","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"GNR-055","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"Generium \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Generium \/ Inapplicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Glycomine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Inapplicable"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Ultevursen","moa":"usherin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Laboratoires Thea","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Laboratoires Thea \/ Sepul Bio","highestDevelopmentStatusID":"9","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"QR-421a","moa":"Usherin messenger RNA (USH2A mRNA)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Laboratoires Thea","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Laboratoires Thea \/ Sepul Bio","highestDevelopmentStatusID":"9","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Ultevursen","moa":"usherin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Laboratoires Thea","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Laboratoires Thea \/ Sepul Bio","highestDevelopmentStatusID":"9","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Osivelotor","moa":"Hemoglobin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Pfizer Inc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Pfizer Inc \/ Inapplicable"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Osivelotor","moa":"Hemoglobin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Pfizer Inc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Pfizer Inc \/ Inapplicable"},{"orgOrder":0,"company":"Vanderbilt University Medical Center","sponsor":"Teva Pharmaceutical Industries","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Deutetrabenazine","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Vanderbilt University Medical Center","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Vanderbilt University Medical Center \/ Teva Pharmaceutical Industries","highestDevelopmentStatusID":"9","companyTruncated":"Vanderbilt University Medical Center \/ Teva Pharmaceutical Industries"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"AMO Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Inapplicable"},{"orgOrder":0,"company":"QED Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Infigratinib","moa":"Fibroblast growth factor receptor","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"QED Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"QED Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"QED Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"SKY-0515","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Skyhawk Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Skyhawk Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"GBT601","moa":"Hemoglobin polymerization","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Global Blood Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Global Blood Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Retrotope","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2020","type":"Inapplicable","leadProduct":"Linoleic Acid","moa":"Lipid peroxidation","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Retrotope","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Retrotope \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Retrotope \/ Inapplicable"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"Applied Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Applied Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Public Offering","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Oral Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Jefferies","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Jefferies"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Novo Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Series B Financing","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Oral Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Novo Ventures","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Novo Ventures"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"AMO Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Inapplicable"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"AMO Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Inapplicable"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"AMO Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Inapplicable"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"AMO Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Inapplicable"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"AMO Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Morgan Stanley","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Morgan Stanley"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.14000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Morgan Stanley","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Morgan Stanley"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.16,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Morgan Stanley","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Morgan Stanley"},{"orgOrder":0,"company":"Disc medicine","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Hercules Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Financing","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Hercules Capital","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Hercules Capital"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Inapplicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Inapplicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.23000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Jefferies","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Jefferies"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.23000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Jefferies","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Jefferies"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Bain Capital Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Private Placement","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Bain Capital Life Sciences","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Bain Capital Life Sciences"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Gemini Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Merger","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.17999999999999999,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17999999999999999,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Gemini Therapeutics","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Gemini Therapeutics"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Inapplicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Inapplicable"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"GBT601","moa":"Hemoglobin polymerization","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Global Blood Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Global Blood Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"GBT601","moa":"Hemoglobin polymerization","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Global Blood Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Global Blood Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"GBT601","moa":"Hemoglobin polymerization","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Global Blood Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Global Blood Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"SKY-0515","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Skyhawk Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Skyhawk Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"SKY-0515","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Skyhawk Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Skyhawk Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"SKY-0515","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Skyhawk Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Skyhawk Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Mavodelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Reneo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Reneo Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Reneo Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"OrbiMed Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Series B Financing","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ OrbiMed Advisors","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ OrbiMed Advisors"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Collaboration","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":1.04,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":1.04,"dosageForm":"Subcutaneous Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"9","companyTruncated":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Licensing Agreement","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":1.04,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":1.04,"dosageForm":"Subcutaneous Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"9","companyTruncated":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2022","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2023","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Lyophilized Powder","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Millendo Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2020","type":"Inapplicable","leadProduct":"Livoletide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Millendo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Millendo Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Millendo Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"RO7204239","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2022","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Lyophilized Powder","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2023","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2024","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2024","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2025","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2020","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2022","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2025","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Pozelimab","moa":"Complement C5","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous","sponsorNew":"Regeneron Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Regeneron Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0.12,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ Syncona Limited","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Syncona Limited"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Cantor Fitzgerald","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0.01,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Cantor Fitzgerald","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Cantor Fitzgerald"},{"orgOrder":0,"company":"Agtc","sponsor":"H.C. Wainwright & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ H.C. Wainwright & Co","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ H.C. Wainwright & Co"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Acquisition","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0.02,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Syncona Limited","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Syncona Limited"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"TCGX","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Series B Financing","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0.17000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17000000000000001,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ TCGX","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ TCGX"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Cotoretigene Toliparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"TCGX","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Series B Financing","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17000000000000001,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ TCGX","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ TCGX"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ligand Pharmaceuticals","sponsor":"Palvella Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2020","type":"Agreement","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ligand Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical","sponsorNew":"Ligand Pharmaceuticals \/ Palvella Therapeutics","highestDevelopmentStatusID":"9","companyTruncated":"Ligand Pharmaceuticals \/ Palvella Therapeutics"},{"orgOrder":0,"company":"Electra Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"ELA026","moa":"SIRP-alpha","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Electra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Electra Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Electra Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Inapplicable"},{"orgOrder":0,"company":"Bambino Ges\u00f9 Hospital and Research Institute","sponsor":"University of Exeter | Radboud University Medical Center | Consorzio per Valutazioni Biologiche e Farmacologiche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Posaconazole","moa":"Cytochrome P450 51","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Bambino Ges\u00f9 Hospital and Research Institute","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bambino Ges\u00f9 Hospital and Research Institute \/ University of Exeter | Radboud University Medical Center | Consorzio per Valutazioni Biologiche e Farmacologiche","highestDevelopmentStatusID":"9","companyTruncated":"Bambino Ges\u00f9 Hospital and Research Institute \/ University of Exeter | Radboud University Medical Center | Consorzio per Valutazioni Biologiche e Farmacologiche"},{"orgOrder":0,"company":"Assistance Publique \u2013 H\u00f4pitaux de Paris","sponsor":"INSERM U1163","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Famotidine","moa":"Histamine H2 receptor","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Assistance Publique \u2013 H\u00f4pitaux de Paris","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ INSERM U1163","highestDevelopmentStatusID":"9","companyTruncated":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ INSERM U1163"},{"orgOrder":0,"company":"OnKure Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"REN001","moa":"PARP delta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"OnKure Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"OnKure Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"OnKure Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Indiana University","sponsor":"Global Health Uganda | Makerere University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Zinc Sulphate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Indiana University","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Indiana University \/ Global Health Uganda | Makerere University","highestDevelopmentStatusID":"9","companyTruncated":"Indiana University \/ Global Health Uganda | Makerere University"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Applied Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Applied Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Camurus","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Peptide","year":"2022","type":"Inapplicable","leadProduct":"CAM2029","moa":"SST receptor","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Camurus","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Camurus \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Camurus \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"TAK-672","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"rAAV2tYF-GRK1-hRPGRco","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beacon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Generium","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"GNR-038","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Generium \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Generium \/ Inapplicable"},{"orgOrder":0,"company":"Generium","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"GNR-055","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Lyophilized Powder","sponsorNew":"Generium \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Generium \/ Inapplicable"},{"orgOrder":0,"company":"PT Prodia StemCell Indonesia","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"Umbilical Cord Mesenchymal Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"PT Prodia StemCell Indonesia","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PT Prodia StemCell Indonesia \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"PT Prodia StemCell Indonesia \/ Inapplicable"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AMO Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2024","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"PT Prodia StemCell Indonesia","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Umbilical Cord Mesenchymal Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"PT Prodia StemCell Indonesia","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PT Prodia StemCell Indonesia \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"PT Prodia StemCell Indonesia \/ Inapplicable"},{"orgOrder":0,"company":"Electra Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"ELA026","moa":"SIRP-alpha","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Electra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Electra Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Electra Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Electra Therapeutics","sponsor":"Westlake Village BioPartners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2022","type":"Series B Financing","leadProduct":"ELA026","moa":"SIRP-alpha","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Electra Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Electra Therapeutics \/ Westlake Village BioPartners","highestDevelopmentStatusID":"9","companyTruncated":"Electra Therapeutics \/ Westlake Village BioPartners"},{"orgOrder":0,"company":"Generium","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"GNR-038","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Generium \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Generium \/ Inapplicable"}]
Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development
Filters
×
FILTER:
Company Name
Year
DEALS // DEV.
Country
Sponsor
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target