menu
    • menu
    [{"orgOrder":0,"company":"Aspa Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"AAV9 BBP-812","moa":"AAV9","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aspa Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aspa Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aspa Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Viralgen","sponsor":"Elpida","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SPAIN","productType":"Cell & Gene Therapy","year":"2023","type":"Partnership","leadProduct":"Melpida","moa":"AP4M1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Viralgen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Viralgen \/ Elpida","highestDevelopmentStatusID":"7","companyTruncated":"Viralgen \/ Elpida"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BBP-812","moa":"Aspartoacylase (ASPA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Resilience","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Collaboration","leadProduct":"BBP-812","moa":"Aspartoacylase (ASPA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Resilience","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Resilience \/ BridgeBio Pharma","highestDevelopmentStatusID":"7","companyTruncated":"Resilience \/ BridgeBio Pharma"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"BBP-812","moa":"Aspartoacylase (ASPA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"rAAV-Olig001-ASPA","moa":"Aspartoacylase gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Myrtelle \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Undisclosed"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"rAAV-Olig001-ASPA","moa":"Aspartoacylase gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Myrtelle \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Undisclosed"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"rAAV-Olig001-ASPA","moa":"Aspartoacylase gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Myrtelle \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Undisclosed"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"rAAV-Olig001-ASPA","moa":"Aspartoacylase gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Myrtelle \/ Pfizer Inc","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Pfizer Inc"},{"orgOrder":0,"company":"Lantu Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Vebeglogene Autotemcel","moa":"Beta-globin","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Lantu Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Lantu Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lantu Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AXO-AAV-GM2","moa":"Beta-hexosaminidase subunit alpha (HEXA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sio Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sio Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Fidelity Management & Research Company","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Series B Financing","leadProduct":"TSHA-101","moa":"Beta-hexosaminidase subunit alpha (HEXA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Fidelity Management & Research Company","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Fidelity Management & Research Company"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-101","moa":"Beta-hexosaminidase subunit alpha (HEXA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"TSHA-101","moa":"Beta-hexosaminidase subunit alpha (HEXA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"TSHA-101","moa":"Beta-hexosaminidase subunit alpha (HEXA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Anupam Sehgal","sponsor":"Taysha Gene Therapies | GlycoNet","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"TSHA-101","moa":"Beta-hexosaminidase subunit alpha (HEXA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Anupam Sehgal","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Anupam Sehgal \/ Taysha Gene Therapies | GlycoNet","highestDevelopmentStatusID":"7","companyTruncated":"Anupam Sehgal \/ Taysha Gene Therapies | GlycoNet"},{"orgOrder":0,"company":"CARE Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"CAN106","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CARE Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CARE Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"CARE Pharma \/ Undisclosed"},{"orgOrder":0,"company":"CARE Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"CAN106","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CARE Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CARE Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"CARE Pharma \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"WuXi Biologics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ WuXi Biologics","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ WuXi Biologics"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"CARE Pharma Shanghai Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ CARE Pharma Shanghai Ltd.","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ CARE Pharma Shanghai Ltd."},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"WuXi Biologics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ WuXi Biologics","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ WuXi Biologics"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"WuXi Biologics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ WuXi Biologics","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ WuXi Biologics"},{"orgOrder":0,"company":"ImmuneCare Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"IMC-002","moa":"CD20\/CD47","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"ImmuneCare Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"ImmuneCare Biopharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ImmuneCare Biopharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-301","moa":"CD34+","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AGN-151587","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"AbbVie Inc","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Termination","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Editas Medicine \/ AbbVie Inc","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ AbbVie Inc"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Theranexus","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Theranexus","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Theranexus \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Theranexus \/ Beyond Batten Disease Foundation"},{"orgOrder":0,"company":"Theranexus","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Theranexus","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Theranexus \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Theranexus \/ Beyond Batten Disease Foundation"},{"orgOrder":0,"company":"Beyond Batten Disease Foundation","sponsor":"Theranexus","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Beyond Batten Disease Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Beyond Batten Disease Foundation \/ Theranexus","highestDevelopmentStatusID":"7","companyTruncated":"Beyond Batten Disease Foundation \/ Theranexus"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Nationwide Children\u2019s Hospital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Financing","leadProduct":"CLN-301","moa":"CLN3","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Alcyone Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alcyone Therapeutics \/ Nationwide Children\u2019s Hospital","highestDevelopmentStatusID":"7","companyTruncated":"Alcyone Therapeutics \/ Nationwide Children\u2019s Hospital"},{"orgOrder":0,"company":"Aegle Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AGLE-102","moa":"COL7A1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aegle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aegle Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aegle Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aegle Therapeutics","sponsor":"US Department of Defense","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Funding","leadProduct":"AGLE-102","moa":"COL7A1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aegle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aegle Therapeutics \/ US Department of Defense","highestDevelopmentStatusID":"7","companyTruncated":"Aegle Therapeutics \/ US Department of Defense"},{"orgOrder":0,"company":"Orphan Technologies","sponsor":"Travere Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Enzyme","year":"2020","type":"Acquisition","leadProduct":"OT-58","moa":"Cystathionine beta-synthase (CBS)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Orphan Technologies","amount2":0.52000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.52000000000000002,"dosageForm":"Injection","sponsorNew":"Orphan Technologies \/ Travere Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Orphan Technologies \/ Travere Therapeutics"},{"orgOrder":0,"company":"Orphan Technologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"OT-58","moa":"Cystathionine beta-synthase (CBS)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Orphan Technologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Orphan Technologies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orphan Technologies \/ Undisclosed"},{"orgOrder":0,"company":"Orphan Technologies","sponsor":"Travere Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Enzyme","year":"2020","type":"Acquisition","leadProduct":"OT-58","moa":"Cystathionine beta-synthase (CBS)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Orphan Technologies","amount2":0.52000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.52000000000000002,"dosageForm":"Injection","sponsorNew":"Orphan Technologies \/ Travere Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Orphan Technologies \/ Travere Therapeutics"},{"orgOrder":0,"company":"Phoenicia BioScience","sponsor":"National Heart, Lung, and Blood Institute","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"LEBANON","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Benserazide Hydrochloride","moa":"DOPA decarboxylase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Phoenicia BioScience","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Phoenicia BioScience \/ National Heart, Lung, and Blood Institute","highestDevelopmentStatusID":"7","companyTruncated":"Phoenicia BioScience \/ National Heart, Lung, and Blood Institute"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"HMB-001","moa":"Factor VIIa","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Hemab Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"HMB-001","moa":"Factor VIIa","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Hemab Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Haemnet","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"HMB-001","moa":"Factor VIIa","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Hemab Therapeutics \/ Haemnet","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Haemnet"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"N-Acetylcysteine Amide","moa":"Free radical","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Nacuity Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Nacuity Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nacuity Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Foundation Fighting Blindness","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"NPI-001","moa":"Free radical","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Nacuity Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Nacuity Pharmaceuticals \/ Foundation Fighting Blindness","highestDevelopmentStatusID":"7","companyTruncated":"Nacuity Pharmaceuticals \/ Foundation Fighting Blindness"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"hLB-001","moa":"G6PC","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"LogicBio Therapeutics \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"GeneDx","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ GeneDx","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ GeneDx"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Silicon Valley Bank","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Financing","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Silicon Valley Bank","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Silicon Valley Bank"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneTx Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2022","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneTx Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneTx Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneTx Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2022","type":"Acquisition","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneTx Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GRIN Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Radiprodil","moa":"Glutamate NMDA receptor; GRIN1\/GRIN2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GRIN Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GRIN Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GRIN Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GRIN Therapeutics","sponsor":"Avance Clinical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Radiprodil","moa":"Glutamate NMDA receptor; GRIN1\/GRIN2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GRIN Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GRIN Therapeutics \/ Avance Clinical","highestDevelopmentStatusID":"7","companyTruncated":"GRIN Therapeutics \/ Avance Clinical"},{"orgOrder":0,"company":"GRIN Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Radiprodil","moa":"Glutamate NMDA receptor; GRIN1\/GRIN2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GRIN Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GRIN Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GRIN Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GRIN Therapeutics","sponsor":"Avance Clinical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Radiprodil","moa":"Glutamate NMDA receptor| GRIN1|GRIN2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GRIN Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GRIN Therapeutics \/ Avance Clinical","highestDevelopmentStatusID":"7","companyTruncated":"GRIN Therapeutics \/ Avance Clinical"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"ATB200","moa":"Glycogen","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Pegtarviliase","moa":"Homocysteine","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aeglea BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aeglea BioTherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aeglea BioTherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Pegtarviliase","moa":"Homocysteine","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aeglea BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aeglea BioTherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aeglea BioTherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegtarviliase","moa":"Homocysteine","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aeglea BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aeglea BioTherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aeglea BioTherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegtarviliase","moa":"Homocysteine","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aeglea BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aeglea BioTherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aeglea BioTherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegtarviliase","moa":"Homocysteine","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aeglea BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aeglea BioTherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aeglea BioTherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Oppenheimer & Co. Inc.","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Oppenheimer & Co. Inc.","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Oppenheimer & Co. Inc."},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Oppenheimer & Co. Inc.","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Oppenheimer & Co. Inc.","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Oppenheimer & Co. Inc."},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Piper Sandler","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Piper Sandler","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Piper Sandler"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Piper Sandler","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Piper Sandler","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Piper Sandler"},{"orgOrder":0,"company":"Arbor Biotechnologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ABO-101","moa":"HS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Arbor Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arbor Biotechnologies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arbor Biotechnologies \/ Undisclosed"},{"orgOrder":0,"company":"Arbor Biotechnologies","sponsor":"Arch Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Series C Financing","leadProduct":"ABO-101","moa":"HS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Arbor Biotechnologies","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.070000000000000007,"dosageForm":"Infusion","sponsorNew":"Arbor Biotechnologies \/ Arch Ventures","highestDevelopmentStatusID":"7","companyTruncated":"Arbor Biotechnologies \/ Arch Ventures"},{"orgOrder":0,"company":"Arbor Biotechnologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ABO-101","moa":"HS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Arbor Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arbor Biotechnologies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arbor Biotechnologies \/ Undisclosed"},{"orgOrder":0,"company":"Arbor Biotechnologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ABO-101","moa":"HS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Arbor Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arbor Biotechnologies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arbor Biotechnologies \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nona Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Batoclimab","moa":"IgG receptor FcRn large subunit p51","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Nona Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nona Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nona Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Argenx","sponsor":"M. Peter Marinkovich","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Efgartigimod","moa":"IgG receptor FcRn large subunit p51","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Argenx","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Argenx \/ M. Peter Marinkovich","highestDevelopmentStatusID":"7","companyTruncated":"Argenx \/ M. Peter Marinkovich"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Lonza Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Partnership","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Lonza Group","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Lonza Group"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Briquilimab","moa":"||KIT","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Briquilimab","moa":"||KIT","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-101","moa":"||LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ATSN-101","moa":"LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-101","moa":"LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Licensing Agreement","leadProduct":"ATSN-101","moa":"LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Nippon Shinyaku","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Nippon Shinyaku"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATSN-101","moa":"||LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-101","moa":"LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-101","moa":"LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"AAV8.hLCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Opus Genetics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Undisclosed"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"AAV8.hLCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Opus Genetics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Undisclosed"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OPGx-001","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Opus Genetics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Undisclosed"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"RD Fund","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Financing","leadProduct":"OPGx-001","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Opus Genetics \/ RD Fund","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ RD Fund"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OPGx-LCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Opus Genetics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Undisclosed"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OPGx-LCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Opus Genetics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Undisclosed"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OPGx-LCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Opus Genetics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Undisclosed"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"OPGx-LCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Opus Genetics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Undisclosed"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Perceptive Advisors","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"OPGx-LCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Injection","sponsorNew":"Opus Genetics \/ Perceptive Advisors","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Perceptive Advisors"},{"orgOrder":0,"company":"Hospices Civils de Lyon","sponsor":"Janssen-Cilag | INSERM U1111","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Daratumumab","moa":"Lymphocyte differentiation antigen CD38","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Hospices Civils de Lyon","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Hospices Civils de Lyon \/ Janssen-Cilag | INSERM U1111","highestDevelopmentStatusID":"7","companyTruncated":"Hospices Civils de Lyon \/ Janssen-Cilag | INSERM U1111"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"Kennedy Krieger Institute","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SPAIN","productType":"Miscellaneous","year":"2022","type":"Collaboration","leadProduct":"Vafidemstat","moa":"Lysine-specific histone demethylase 1 (LSD)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Oryzon Genomics \/ Kennedy Krieger Institute","highestDevelopmentStatusID":"7","companyTruncated":"Oryzon Genomics \/ Kennedy Krieger Institute"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"Kabuki Syndrome Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SPAIN","productType":"Miscellaneous","year":"2021","type":"Funding","leadProduct":"Vafidemstat","moa":"Lysine-specific histone demethylase 1 (LSD)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Oryzon Genomics \/ Kabuki Syndrome Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Oryzon Genomics \/ Kabuki Syndrome Foundation"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AT845","moa":"Lysosomal alpha-glucosidase (GAA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Astellas Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Astellas Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AT845","moa":"Lysosomal alpha-glucosidase (GAA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Astellas Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Astellas Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AT845","moa":"Lysosomal alpha-glucosidase (GAA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Astellas Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Astellas Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AT845","moa":"Lysosomal alpha-glucosidase (GAA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Astellas Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Astellas Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Audentes Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AT845","moa":"Lysosomal alpha-glucosidase (GAA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Audentes Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Audentes Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Audentes Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Neurogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Undisclosed"},{"orgOrder":0,"company":"Neurogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Undisclosed"},{"orgOrder":0,"company":"Neurogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Undisclosed"},{"orgOrder":0,"company":"Neurogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Undisclosed"},{"orgOrder":0,"company":"Neurogene","sponsor":"Great Point Partners","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Private Placement","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Great Point Partners","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Great Point Partners"},{"orgOrder":0,"company":"Neurogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Undisclosed"},{"orgOrder":0,"company":"Neurogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Undisclosed"},{"orgOrder":0,"company":"Neoleukin Therapeutics","sponsor":"Neurogene","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Merger","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neoleukin Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neoleukin Therapeutics \/ Neurogene","highestDevelopmentStatusID":"7","companyTruncated":"Neoleukin Therapeutics \/ Neurogene"},{"orgOrder":0,"company":"Neoleukin Therapeutics","sponsor":"Neurogene","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Merger","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neoleukin Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neoleukin Therapeutics \/ Neurogene","highestDevelopmentStatusID":"7","companyTruncated":"Neoleukin Therapeutics \/ Neurogene"},{"orgOrder":0,"company":"Neurogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Financing","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.050000000000000003,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Astellas Pharma","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Astellas Pharma"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"GordonMD Global Investments","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Private Placement","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ GordonMD Global Investments","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ GordonMD Global Investments"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Private Placement","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ RA Capital Management","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ RA Capital Management"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"LogicBio Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"LogicBio Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"LogicBio Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"LogicBio Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"LogicBio Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"LogicBio Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Collaboration","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0.58999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.58999999999999997,"dosageForm":"Infusion","sponsorNew":"LogicBio Therapeutics \/ CANbridge Pharmaceuticals","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"LogicBio Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Public Offering","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"LogicBio Therapeutics \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Jefferies"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"LogicBio Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"mRNA-3704","moa":"Methylmalonyl-CoA mutase (MMUT)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Moderna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Foresee Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"FP-045","moa":"Mitochondrial aldehyde dehydrogenase (ALDH2)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Foresee Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Foresee Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Foresee Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Foresee Pharmaceuticals","sponsor":"TRPharm","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Collaboration","leadProduct":"FP-045","moa":"Mitochondrial aldehyde dehydrogenase (ALDH2)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Foresee Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Foresee Pharmaceuticals \/ TRPharm","highestDevelopmentStatusID":"7","companyTruncated":"Foresee Pharmaceuticals \/ TRPharm"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"National Human Genome Research Institute","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SEL-302","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ National Human Genome Research Institute","highestDevelopmentStatusID":"7","companyTruncated":"Selecta Biosciences \/ National Human Genome Research Institute"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"SEL-302","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Selecta Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"SEL-302","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Selecta Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"ITALY","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AAVB-081","moa":"MYO7A","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"AAVantgarde Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Undisclosed"},{"orgOrder":0,"company":"AnnJi Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"TAIWAN","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"AnnJi Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Liquid","sponsorNew":"AnnJi Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AnnJi Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"AnnJi Pharmaceutical","sponsor":"Avenue Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"TAIWAN","productType":"Miscellaneous","year":"2023","type":"Licensing Agreement","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"AnnJi Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.25,"dosageForm":"Liquid","sponsorNew":"AnnJi Pharmaceutical \/ Avenue Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"AnnJi Pharmaceutical \/ Avenue Therapeutics"},{"orgOrder":0,"company":"AnnJi Pharmaceutical","sponsor":"Avenue Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"TAIWAN","productType":"Miscellaneous","year":"2023","type":"Licensing Agreement","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"AnnJi Pharmaceutical","amount2":0.25,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.25,"dosageForm":"Liquid","sponsorNew":"AnnJi Pharmaceutical \/ Avenue Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"AnnJi Pharmaceutical \/ Avenue Therapeutics"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Avenue Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Avenue Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"Maxim Group LLC","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Avenue Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Liquid","sponsorNew":"Avenue Therapeutics \/ Maxim Group LLC","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ Maxim Group LLC"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"AnnJi Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Avenue Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Avenue Therapeutics \/ AnnJi Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ AnnJi Pharmaceutical"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Avenue Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Avenue Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"Maxim Group LLC","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Avenue Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Liquid","sponsorNew":"Avenue Therapeutics \/ Maxim Group LLC","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ Maxim Group LLC"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Public Offering","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ JMP Securities","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ JMP Securities"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Public Offering","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ JMP Securities","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ JMP Securities"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Citizens JMP","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Private Placement","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.040000000000000001,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Citizens JMP","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Citizens JMP"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Public Offering","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ JMP Securities","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ JMP Securities"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Leerink Partners","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Leerink Partners"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Public Offering","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ JMP Securities","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ JMP Securities"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Venthera","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"BBP-681","moa":"PI3K","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"BridgeBio Pharma \/ Venthera","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Venthera"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"VT30","moa":"PI3K","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Tern Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"TTX-381","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Tern Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Tern Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Tern Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Andelyn Biosciences","sponsor":"Tern Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Partnership","leadProduct":"TTX-381","moa":"TPP1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Andelyn Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Andelyn Biosciences \/ Tern Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Andelyn Biosciences \/ Tern Therapeutics"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Longitude Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Series A Financing","leadProduct":"LX1004","moa":"Tripeptidyl-peptidase I (TPP1)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.089999999999999997,"dosageForm":"Injection","sponsorNew":"Lexeo Therapeutics \/ Longitude Capital","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Longitude Capital"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LX1004","moa":"Tripeptidyl-peptidase I (TPP1)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"LX1004","moa":"Tripeptidyl-peptidase I (TPP1)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AskBio","sponsor":"Scottish Enterprise","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Funding","leadProduct":"AAV2\/8LSPhGAA","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Scottish Enterprise","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Scottish Enterprise"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Acquisition","leadProduct":"AAV2\/8LSPhGAA","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":4,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":4,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Bayer AG","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Bayer AG"},{"orgOrder":0,"company":"AskBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ACTUS-101","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Undisclosed"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"ADARx Pharmaceuticals | Novotech","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"ADX-038","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"ADARx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ADARx Pharmaceuticals \/ ADARx Pharmaceuticals | Novotech","highestDevelopmentStatusID":"7","companyTruncated":"ADARx Pharmaceuticals \/ ADARx Pharmaceuticals | Novotech"},{"orgOrder":0,"company":"Vitro Biopharma Inc.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Allorx Stem Cell","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Vitro Biopharma Inc.","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vitro Biopharma Inc. \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vitro Biopharma Inc. \/ Undisclosed"},{"orgOrder":0,"company":"Wacker Chemie AG","sponsor":"Caeregen Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"GERMANY","productType":"Miscellaneous","year":"2023","type":"Collaboration","leadProduct":"CTR-107","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Wacker Chemie AG","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wacker Chemie AG \/ Caeregen Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Wacker Chemie AG \/ Caeregen Therapeutics"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"DS-2325a","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Daiichi Sankyo \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Daiichi Sankyo \/ Undisclosed"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"DS-2325a","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Daiichi Sankyo \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Daiichi Sankyo \/ Undisclosed"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"DS-2325a","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Daiichi Sankyo \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Daiichi Sankyo \/ Undisclosed"},{"orgOrder":0,"company":"Eyestem","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"INDIA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Eyecyte-RPE","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Eyestem","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Eyestem \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Eyestem \/ Undisclosed"},{"orgOrder":0,"company":"GeneCradle Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"GC301","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneCradle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GeneCradle Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GeneCradle Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GeneCradle Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"GC301","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneCradle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GeneCradle Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GeneCradle Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Genethon","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"GNT-003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Genethon \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Genethon \/ Undisclosed"},{"orgOrder":0,"company":"HuidaGene Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"HG004","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"HuidaGene Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"HuidaGene Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"HuidaGene Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"HuidaGene Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"HG004","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"HuidaGene Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"HuidaGene Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"HuidaGene Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Gangnam Severance Hospital","sponsor":"Daewoong Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Human Umbilical Cord Blood Derived Mesenchymal Stem Cell","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Gangnam Severance Hospital","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gangnam Severance Hospital \/ Daewoong Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Gangnam Severance Hospital \/ Daewoong Pharmaceutical"},{"orgOrder":0,"company":"Berge Minassian","sponsor":"Elpida Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"ION283","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Berge Minassian","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Berge Minassian \/ Elpida Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Berge Minassian \/ Elpida Therapeutics"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Kyowa Kirin","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"KK8123","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kyowa Kirin \/ Kyowa Kirin","highestDevelopmentStatusID":"7","companyTruncated":"Kyowa Kirin \/ Kyowa Kirin"},{"orgOrder":0,"company":"Kanglin Biotechnology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"KL003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Kanglin Biotechnology","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Kanglin Biotechnology \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Kanglin Biotechnology \/ Undisclosed"},{"orgOrder":0,"company":"Kanglin Biotechnology","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell & Gene Therapy","year":"2024","type":"Series A Financing","leadProduct":"KL003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Kanglin Biotechnology","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Injection","sponsorNew":"Kanglin Biotechnology \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Kanglin Biotechnology \/ Undisclosed"},{"orgOrder":0,"company":"Minovia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"MNV-BLD","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Minovia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Minovia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Minovia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"mRNA-3705","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Moderna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"mRNA-3705","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Moderna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"mRNA-3705","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Moderna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"mRNA-3705","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Moderna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"MYR-101","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Myrtelle \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Undisclosed"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"MYR-101","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Myrtelle \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Undisclosed"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Levetiracetam","moa":"||Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Myrtelle \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Undisclosed"},{"orgOrder":0,"company":"Mahzi Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"MZ-1866","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Mahzi Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Mahzi Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mahzi Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"NGGT Suzhou Biotechnology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"NGGT001","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"NGGT Suzhou Biotechnology","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"NGGT Suzhou Biotechnology \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"NGGT Suzhou Biotechnology \/ Undisclosed"},{"orgOrder":0,"company":"Fenix Innovation Group","sponsor":"Neurotech International","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"AUSTRALIA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"NTI164","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Fenix Innovation Group","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Fenix Innovation Group \/ Neurotech International","highestDevelopmentStatusID":"7","companyTruncated":"Fenix Innovation Group \/ Neurotech International"},{"orgOrder":0,"company":"Phoenicis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"PTW-002","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Phoenicis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Hydrogel","sponsorNew":"Phoenicis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Phoenicis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SAR439483","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Sofinnova Investments","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Series A Financing","leadProduct":"SAR439483","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Sofinnova Investments","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Sofinnova Investments"},{"orgOrder":0,"company":"MyoPax","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"GERMANY","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Satori-01","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"MyoPax","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"MyoPax \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"MyoPax \/ Undisclosed"},{"orgOrder":0,"company":"MyoPax","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"GERMANY","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Satori-01","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"MyoPax","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"MyoPax \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"MyoPax \/ Undisclosed"},{"orgOrder":0,"company":"Sixera Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWEDEN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"SXR1096","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Sixera Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Cream","sponsorNew":"Sixera Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sixera Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"UX053","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"UX053","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"OBiO Technology","sponsor":"Chinagene","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Collaboration","leadProduct":"ZVS101e","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"OBiO Technology","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"OBiO Technology \/ Chinagene","highestDevelopmentStatusID":"7","companyTruncated":"OBiO Technology \/ Chinagene"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"QR-421a","moa":"Usherin messenger RNA (USH2A mRNA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"ProQR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EXG-001","moa":"||ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"EXG-34217","moa":"ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EXG-34217","moa":"ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EXG-34217","moa":"ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Acquisition","leadProduct":"CLN-301","moa":"CLN3","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Alcyone Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alcyone Therapeutics \/ Biogen","highestDevelopmentStatusID":"7","companyTruncated":"Alcyone Therapeutics \/ Biogen"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

    Xls
    Filters Filter
    ×
    FILTER:
    filter
    Company Name
      filter

      Year

        filter

        DEALS // DEV.

          filter

          Country

            filter
            Sponsor
              filter

              Therapeutic Area

                filter

                Study Phase

                  filter

                  Deal Type

                    filter

                    Product Type

                      filter

                      Dosage Form

                        filter

                        Lead Product

                          filter

                          Target

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

                            Therapeutic Area by Lead Product

                            Study Phase by Lead Product

                            Company by Lead Product

                            Top Deals by Deal Size (USD bn)

                            01

                            Alcyone Therapeutics

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            Alcyone Therapeutics

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

                            Lead Product(s) : CLN-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Biogen

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

                            DEALS_DEV arrow-down

                            Biogen to Acquire Alcyone Therapeutics, Expanding Drug Delivery Solution Portfolio

                            Details : With the acquisition, Biogen Alcyone’s remaining therapeutic assets, including CLN-301 will be divested into a new independent company, Neela Therapeutics.

                            Product Name : CLN-301

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            September 18, 2025

                            Lead Product(s) : CLN-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Biogen

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            02

                            Taysha Gene Therapies

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            Taysha Gene Therapies

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

                            Lead Product(s) : TSHA-102

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Jefferies

                            Deal Size : Undisclosed

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Taysha Gene Therapies Announces Offering of Common Stock and Pre-Funded Warrants

                            Details : The proceeds will be used to fund clinical, regulatory and manufacturing development for TSHA-102, a self-complementary intrathecally delivered AAV9 gene transfer therapy, in Rett syndrome.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            May 28, 2025

                            Lead Product(s) : TSHA-102

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Jefferies

                            Deal Size : Undisclosed

                            Deal Type : Public Offering

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            03

                            Taysha Gene Therapies

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            Taysha Gene Therapies

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

                            Lead Product(s) : TSHA-102

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Jefferies

                            Deal Size : $200.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Taysha Gene Therapies Announces Pricing of Stock and Pre-Funded Warrants

                            Details : The company will fund clinical, regulatory and manufacturing development for TSHA-102, a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy, in Rett syndrome.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            May 28, 2025

                            Lead Product(s) : TSHA-102

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Jefferies

                            Deal Size : $200.0 million

                            Deal Type : Public Offering

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            04

                            Theranexus

                            France arrow
                            BioJapan
                            Not Confirmed

                            Theranexus

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

                            Lead Product(s) : Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Beyond Batten Disease Foundation

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Theranexus & BBDF Reveal Real-World Data Supporting Batten-1 for Batten

                            Details : Batten-1 is a novel and exclusive proprietary drug containing the active ingredient miglustat. The MOA of this substance blocks the accumulation of glycosphingolipids and neuroinflammation.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 13, 2025

                            Lead Product(s) : Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Beyond Batten Disease Foundation

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            05

                            Opus Genetics

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            Opus Genetics

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

                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Opus gets RMAT Designation for OPGx-LCA5 Gene Therapy in Vision Restoration

                            Details : OPGx-LCA5 is an investigational gene therapy, which is currently being evaluated for the treatment of patients with leber congenital amaurosis.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            May 06, 2025

                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            06

                            Opus Genetics

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            Opus Genetics

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

                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Opus Shares One-Month Data from Pediatric Gene Therapy OPGx-LCA5 Trial

                            Details : OPGx-LCA5 is an investigational gene therapy, which is currently being evaluated for the treatment of patients with leber congenital amaurosis.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            April 08, 2025

                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            07

                            Benitec Biopharma

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            Benitec Biopharma

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

                            Lead Product(s) : BB-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Leerink Partners

                            Deal Size : $30.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Benitec Biopharma Inc. Announces Pricing of $30 Million Common Stock Offering

                            Details : The net proceeds from the proposed financing will support the clinical development of BB-301, which is being evaluated for Oculopharyngeal Muscular Dystrophy.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            March 25, 2025

                            Lead Product(s) : BB-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Leerink Partners

                            Deal Size : $30.0 million

                            Deal Type : Public Offering

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            08

                            Opus Genetics

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            Opus Genetics

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

                            Lead Product(s) : OPGx-LCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Perceptive Advisors

                            Deal Size : $21.4 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Opus Genetics Prices Public Offering & Private Placement Over $20M

                            Details : Opus intends to use the net proceeds to fund clinical development of its lead gene therapy programs, OPGx-LCA5 for the treatment of leber congenital amaurosis.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            March 21, 2025

                            Lead Product(s) : OPGx-LCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Perceptive Advisors

                            Deal Size : $21.4 million

                            Deal Type : Public Offering

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            09

                            Elixirgen Therapeutics

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            Elixirgen Therapeutics

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

                            Lead Product(s) : EXG-34217

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Elixirgen Gets FDA Orphan Drug Designation for Telomere Disorders

                            Details : EXG-34217 is a dose of autologous CD34+ hematopoietic stem cells (HSCs) that is being investigated for the treatment of telomere biology disorders.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            February 20, 2025

                            Lead Product(s) : EXG-34217

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            10

                            Opus Genetics

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            Opus Genetics

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

                            Lead Product(s) : OPGx-LCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Opus Genetics Announces Updates on OPGx-LCA5 Clinical Program

                            Details : OPGx-LCA5 is an investigational gene therapy, which is currently being evaluated for the treatment of patients with leber congenital amaurosis.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            February 18, 2025

                            Lead Product(s) : OPGx-LCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth