menu
    • menu
    [{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Deep Track Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Private Placement","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Deep Track Capital","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Deep Track Capital"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"KRRO-110","moa":"A1AT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Korro Bio \/ Undisclosed"},{"orgOrder":0,"company":"AM-Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Ilofotase Alpha","moa":"A2AR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AM-Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AM-Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AM-Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AM-Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Ilofotase Alpha","moa":"A2AR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AM-Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AM-Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AM-Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Adrenas Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"AAV BBP-631","moa":"AAV","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Adrenas Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Adrenas Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Adrenas Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"UX111","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Abeona Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Abeona Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"UX111","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Abeona Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Abeona Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Acquisition","leadProduct":"UX111","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Abeona Therapeutics \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Abeona Therapeutics \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Lantu Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Vesemnogene Lantuparvovec","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lantu Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Lantu Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lantu Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"AAVB-039","moa":"ABCA4 protein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AAVantgarde Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Undisclosed"},{"orgOrder":0,"company":"H. Lundbeck AS","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Lu AG13909","moa":"ACTH receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"H. Lundbeck AS","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"H. Lundbeck AS \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"H. Lundbeck AS \/ Undisclosed"},{"orgOrder":0,"company":"Vaderis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"VAD044","moa":"AKT kinase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vaderis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vaderis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vaderis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vaderis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"VAD044","moa":"AKT kinase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vaderis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vaderis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vaderis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vaderis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"VAD044","moa":"AKT kinase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vaderis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vaderis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vaderis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vaderis Therapeutics","sponsor":"Medicxi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"VAD044","moa":"AKT kinase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vaderis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Vaderis Therapeutics \/ Medicxi","highestDevelopmentStatusID":"7","companyTruncated":"Vaderis Therapeutics \/ Medicxi"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"DCR-A1AT","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dicerna Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Dicerna Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dicerna Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Viking Global Investors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Series C Financing","leadProduct":"4D-310","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"4D Molecular Therapeutics \/ Viking Global Investors","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Viking Global Investors"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"Isaralgagene Civaparvovec","moa":"Alpha-galactosidase A (GLA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sangamo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sangamo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sangamo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"RGX-111","moa":"Alpha-L-iduronidase (IDUA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"RGX-111","moa":"Alpha-L-iduronidase (IDUA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"RGX-111","moa":"Alpha-L-iduronidase (IDUA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"AM-Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Ilofotase Alfa","moa":"ALPI","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AM-Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AM-Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AM-Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"WVE-120101","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"WVE-120102","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"WVE-120102","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"BlackfinBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"BFB-101","moa":"AP4B1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BlackfinBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BlackfinBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BlackfinBio \/ Undisclosed"},{"orgOrder":0,"company":"BlackfinBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"BFB-101","moa":"AP4B1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BlackfinBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BlackfinBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BlackfinBio \/ Undisclosed"},{"orgOrder":0,"company":"Rajshekhar Chakraborty, MD","sponsor":"Genentech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Venetoclax","moa":"Apoptosis regulator Bcl-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Rajshekhar Chakraborty, MD","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Rajshekhar Chakraborty, MD \/ Genentech","highestDevelopmentStatusID":"7","companyTruncated":"Rajshekhar Chakraborty, MD \/ Genentech"},{"orgOrder":0,"company":"Alfred Chung","sponsor":"Johnson & Johnson Innovative Medicine | AbbVie Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Venetoclax","moa":"Apoptosis regulator Bcl-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alfred Chung","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alfred Chung \/ Johnson & Johnson Innovative Medicine | AbbVie Inc","highestDevelopmentStatusID":"7","companyTruncated":"Alfred Chung \/ Johnson & Johnson Innovative Medicine | AbbVie Inc"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"Hawaii Pacific Neuroscience","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"nL-ATN1-002","moa":"ATN1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"n-Lorem Foundation \/ Hawaii Pacific Neuroscience","highestDevelopmentStatusID":"7","companyTruncated":"n-Lorem Foundation \/ Hawaii Pacific Neuroscience"},{"orgOrder":0,"company":"Clarametyx Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"CMTX-101","moa":"Bacterial biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Clarametyx Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Clarametyx Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Clarametyx Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Clarametyx Biosciences","sponsor":"KAMRA Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Funding","leadProduct":"CMTX-101","moa":"Bacterial biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Clarametyx Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Clarametyx Biosciences \/ KAMRA Fund","highestDevelopmentStatusID":"7","companyTruncated":"Clarametyx Biosciences \/ KAMRA Fund"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"DFT383","moa":"BCL-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Zentalis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"ZN-d5","moa":"BCL-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Zentalis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zentalis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Zentalis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Zentalis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"ZN-D5","moa":"BCL-2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Zentalis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zentalis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Zentalis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Nexcella","sponsor":"Immix Biopharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Immix Biopharma","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Immix Biopharma"},{"orgOrder":0,"company":"Nexcella","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Undisclosed"},{"orgOrder":0,"company":"Nexcella","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Undisclosed"},{"orgOrder":0,"company":"Nexcella","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Undisclosed"},{"orgOrder":0,"company":"Nexcella","sponsor":"Cirm","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Funding","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Cirm","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Cirm"},{"orgOrder":0,"company":"Nexcella","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Cyclophosphamide","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Undisclosed"},{"orgOrder":0,"company":"Nexcella","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NXC-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nexcella","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Nexcella \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nexcella \/ Undisclosed"},{"orgOrder":0,"company":"Viralgen","sponsor":"Sio Gene Therapies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell & Gene Therapy","year":"2020","type":"Partnership","leadProduct":"AXO-AAV-GM1","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Viralgen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Viralgen \/ Sio Gene Therapies","highestDevelopmentStatusID":"7","companyTruncated":"Viralgen \/ Sio Gene Therapies"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AXO-AAV-GM1","moa":"||Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sio Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sio Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AXO-AAV-GM1","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sio Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sio Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AXO-AAV-GM1","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sio Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sio Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AXO-AAV-GM1","moa":"||Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sio Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sio Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"National Human Genome Research Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AXO-AAV-GM1","moa":"||Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sio Gene Therapies \/ National Human Genome Research Institute","highestDevelopmentStatusID":"7","companyTruncated":"Sio Gene Therapies \/ National Human Genome Research Institute"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"GEMMA Biotherapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Licensing Agreement","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0.13,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ GEMMA Biotherapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ GEMMA Biotherapeutics"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Public Offering","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0.13,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ J.P. Morgan"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PBGM01","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"BMN 331","moa":"C1-INH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARO-C3","moa":"C3 receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Moderna Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"VX-522","moa":"cAMP-dependent chloride channel (CFTR)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"Vertex Pharmaceuticals \/ Moderna Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Vertex Pharmaceuticals \/ Moderna Therapeutics"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"VX-522","moa":"cAMP-dependent chloride channel (CFTR)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"EDIT-301","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"DRI Healthcare Trust","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Financing","leadProduct":"Renizgamglogene Autogedtemcel","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ DRI Healthcare Trust","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ DRI Healthcare Trust"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Renizgamglogene Autogedtemcel","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Azzur Group","sponsor":"Editas Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Agreement","leadProduct":"Renizgamglogene Autogedtemcel","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Azzur Group","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Azzur Group \/ Editas Medicine","highestDevelopmentStatusID":"7","companyTruncated":"Azzur Group \/ Editas Medicine"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Azzur Group","sponsor":"Editas Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Partnership","leadProduct":"Renizgamglogene Autogedtemcel","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Azzur Group","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Azzur Group \/ Editas Medicine","highestDevelopmentStatusID":"7","companyTruncated":"Azzur Group \/ Editas Medicine"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Renizgamglogene Autogedtemcel","moa":"CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Editas Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Sorrento Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"STI-6129","moa":"CD38","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sorrento Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sorrento Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sorrento Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sorrento Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"STI-6129","moa":"CD38","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sorrento Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sorrento Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sorrento Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jiayin Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"EXG110","moa":"CD39","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jiayin Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Jiayin Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jiayin Biotech \/ Undisclosed"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Etentamig","moa":"CD3E","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AbbVie Inc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AbbVie Inc \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AT-GTX-501","moa":"Ceroid-lipofuscinosis neuronal protein 6 (CLN6)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AT-GTX-501","moa":"Ceroid-lipofuscinosis neuronal protein 6 (CLN6)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"4D-710","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"4D-710","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"4D-710","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"4D-710","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"4D-710","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"OXB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"BI 3720931","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Boehringer Ingelheim GmbH \/ OXB","highestDevelopmentStatusID":"7","companyTruncated":"Boehringer Ingelheim GmbH \/ OXB"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"MRT5005","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"MRT5005","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BAY2599023","moa":"Coagulation factor VIII (F8)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Bayer AG \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"UX701","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"UX701","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"UX701","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"UX701","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"UX701","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"UX701","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"VTX-801","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vivet Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vivet Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"VTX-801","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vivet Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"7","companyTruncated":"Vivet Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"VTX-801","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vivet Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Vivet Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Agreement","leadProduct":"VTX-801","moa":"Copper-transporting ATPase 2 (ATP7B)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pfizer Inc \/ Vivet Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Pfizer Inc \/ Vivet Therapeutics"},{"orgOrder":0,"company":"Bristol Myers Squibb","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"BMS-986470","moa":"CRBN\/ZBTB7A\/WIZ","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bristol Myers Squibb","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bristol Myers Squibb \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bristol Myers Squibb \/ Undisclosed"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Kamau Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Agreement","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Kamau Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Kamau Therapeutics"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Undisclosed"},{"orgOrder":0,"company":"Xenetic Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"SHP656","moa":"Cryptochrome circadian clock 1 (CRY1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Xenetic Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xenetic Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Xenetic Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Ensoma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EN-374","moa":"CYBB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ensoma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ensoma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ensoma \/ Undisclosed"},{"orgOrder":0,"company":"Ensoma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EN-374","moa":"CYBB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ensoma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ensoma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ensoma \/ Undisclosed"},{"orgOrder":0,"company":"Ensoma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EN-374","moa":"CYBB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ensoma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ensoma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ensoma \/ Undisclosed"},{"orgOrder":0,"company":"Instituto Nacional de Investigacao em Saude, Angola","sponsor":"Fundaci\u00f3n Bancaria Caixa D'estalvis I Pensions De Barcelona | Funda\u00e7ao Calouste Gulbenkian | CLINCOORD SERVICES, INC. | CLINCOORD PRESTA\u00c7\u00c3O DE SEVI\u00c7OS, LDA ANGOLA | Escola Superior de Tecnologia da Sa\u00fade de Lisboa | ClinCoord Research Center at Luanda Med","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ANGOLA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Aspirin","moa":"Cyclooxygenase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Instituto Nacional de Investigacao em Saude, Angola","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Instituto Nacional de Investigacao em Saude, Angola \/ Fundaci\u00f3n Bancaria Caixa D'estalvis I Pensions De Barcelona | Funda\u00e7ao Calouste Gulbenkian | CLINCOORD SERVICES, INC. | CLINCOORD PRESTA\u00c7\u00c3O DE SEVI\u00c7OS, LDA ANGOLA | Escola Superior de Tecnologia da Sa\u00fade de Lisboa | ClinCoord Research Center at Luanda Med","highestDevelopmentStatusID":"7","companyTruncated":"Instituto Nacional de Investigacao em Saude, Angola \/ Fundaci\u00f3n Bancaria Caixa D'estalvis I Pensions De Barcelona | Funda\u00e7ao Calouste Gulbenkian | CLINCOORD SERVICES, INC. | CLINCOORD PRESTA\u00c7\u00c3O DE SEVI\u00c7OS, LDA ANGOLA | Escola Superior de Tecnologia da Sa\u00fade de Lisboa | ClinCoord Research Center at Luanda Med"},{"orgOrder":0,"company":"Proteostasis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Dirocaftor","moa":"||cystic fibrosis transmembrane regulator (CFTR) protein modulators","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Proteostasis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proteostasis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Proteostasis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Proteostasis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Dirocaftor","moa":"||cystic fibrosis transmembrane regulator (CFTR) protein modulators","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Proteostasis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proteostasis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Proteostasis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-04","moa":"Cystinosin (CTNS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-04","moa":"Cystinosin (CTNS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AVR-RD-04","moa":"Cystinosin (CTNS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AVR-RD-04","moa":"Cystinosin (CTNS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-04","moa":"Cystinosin (CTNS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"University of California","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AVR-RD-04","moa":"Cystinosin (CTNS)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ University of California","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ University of California"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"JR-171","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Lepunafusp Alpha","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Lepunafusp Alpha","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Lepunafusp Alpha","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Lepunafusp Alpha","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"BMN 351","moa":"DMD exon 51","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARO-DM1","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ARO-DM1","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARO-DM1","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Licensing Agreement","leadProduct":"ARO-DM1","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":11.380000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":11.380000000000001,"dosageForm":"Infusion","sponsorNew":"Arrowhead Pharmaceuticals \/ Sarepta Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0.33000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0.37,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Public Offering","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0.23000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2021","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-101","moa":"DMPK","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Essen Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Busulfan","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Essen Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Essen Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Essen Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Oncopeptides","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Melphalan Flufenamide","moa":"||DNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Oncopeptides","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Oncopeptides \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Oncopeptides \/ Undisclosed"},{"orgOrder":0,"company":"Johns Hopkins University","sponsor":"Neurofibromatosis Therapeutic Acceleration Program | SpringWorks Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Mirdametinib","moa":"Dual specificity mitogen-activated protein kinase kinase 2 | Dual specificity mitogen-activated protein kinase kinase 1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Johns Hopkins University","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johns Hopkins University \/ Neurofibromatosis Therapeutic Acceleration Program | SpringWorks Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Johns Hopkins University \/ Neurofibromatosis Therapeutic Acceleration Program | SpringWorks Therapeutics"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Licensing Agreement","leadProduct":"ARO-DUX4","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":11.380000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":11.380000000000001,"dosageForm":"Infusion","sponsorNew":"Arrowhead Pharmaceuticals \/ Sarepta Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARO-DUX4","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Licensing Agreement","leadProduct":"ARO-DUX4","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":11.380000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":11.380000000000001,"dosageForm":"Undisclosed","sponsorNew":"Arrowhead Pharmaceuticals \/ Sarepta Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ARO-DUX4","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Epicrispr Biotechnologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Epicrispr Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Epicrispr Biotechnologies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Epicrispr Biotechnologies \/ Undisclosed"},{"orgOrder":0,"company":"Epicrispr Biotechnologies","sponsor":"Springbok Analytics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Partnership","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Epicrispr Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Epicrispr Biotechnologies \/ Springbok Analytics","highestDevelopmentStatusID":"7","companyTruncated":"Epicrispr Biotechnologies \/ Springbok Analytics"},{"orgOrder":0,"company":"Epicrispr Biotechnologies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Epicrispr Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Epicrispr Biotechnologies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Epicrispr Biotechnologies \/ Undisclosed"},{"orgOrder":0,"company":"Epicrispr Biotechnologies","sponsor":"Ally Bridge Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Series B Financing","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Epicrispr Biotechnologies","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Injection","sponsorNew":"Epicrispr Biotechnologies \/ Ally Bridge Group","highestDevelopmentStatusID":"7","companyTruncated":"Epicrispr Biotechnologies \/ Ally Bridge Group"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2021","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2021","type":"Inapplicable","leadProduct":"DYNE-251","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Dyne Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Public Offering","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0.17999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17999999999999999,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Goldman Sachs & Co","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Janus Henderson Investors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Public Offering","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Janus Henderson Investors","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Janus Henderson Investors"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ENTR-601-45","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-45","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-45","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-45","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Entrada Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Entrada Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Megan Waldrop","sponsor":"Audentes Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"scAAV9.U7.ACCA","moa":"Dystrophin exon-51","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Megan Waldrop","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Megan Waldrop \/ Audentes Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Megan Waldrop \/ Audentes Therapeutics"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Thomas Jefferson University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Thomas Jefferson University","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ Thomas Jefferson University"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"IQVIA","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ IQVIA","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ IQVIA"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Thomas Jefferson University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Thomas Jefferson University","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ Thomas Jefferson University"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Calico Life Sciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Fosigotifator","moa":"EIF2B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AbbVie Inc \/ Calico Life Sciences","highestDevelopmentStatusID":"7","companyTruncated":"AbbVie Inc \/ Calico Life Sciences"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Fosigotifator","moa":"EIF2B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AbbVie Inc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AbbVie Inc \/ Undisclosed"},{"orgOrder":0,"company":"Calico Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Fosigotifator","moa":"EIF2B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Calico Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Calico Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Calico Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Calico Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Fosigotifator","moa":"EIF2B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Calico Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Calico Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Calico Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Aro-Enac","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"ARO-ENaC","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ARO-ENaC","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ARO-ENaC","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ARO-ENaC","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Arrowhead Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Arrowhead Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"IONIS-ENAC-2.5Rx","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"London Health Sciences Centre Research Institute (Lawson)","sponsor":"Greenstone Biosciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Genistein","moa":"Estrogen receptor beta (ESR2)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"London Health Sciences Centre Research Institute (Lawson)","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"London Health Sciences Centre Research Institute (Lawson) \/ Greenstone Biosciences","highestDevelopmentStatusID":"7","companyTruncated":"London Health Sciences Centre Research Institute (Lawson) \/ Greenstone Biosciences"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AAV5-hFIX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Thermo Fisher Scientific","sponsor":"Spur Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Agreement","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Thermo Fisher Scientific","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Thermo Fisher Scientific \/ Spur Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Thermo Fisher Scientific \/ Spur Therapeutics"},{"orgOrder":0,"company":"ASC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ASC618","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ASC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ASC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ASC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"ASC Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Expanded Collaboration","leadProduct":"ASC618","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Charles River Laboratories, Inc \/ ASC Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Charles River Laboratories, Inc \/ ASC Therapeutics"},{"orgOrder":0,"company":"ASC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ASC618","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ASC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ASC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ASC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Octapharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"OCTA12","moa":"||Factor VIII","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Octapharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Octapharma \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"NXT007","moa":"Factor VIIIa mimetic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"AskBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AB-1003","moa":"FKRP","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Undisclosed"},{"orgOrder":0,"company":"AskBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AB-1003","moa":"FKRP","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Undisclosed"},{"orgOrder":0,"company":"AskBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AB-1003","moa":"FKRP","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Undisclosed"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"N-Acetylcysteine Amide","moa":"Free radical","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nacuity Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Nacuity Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nacuity Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Foundation Fighting Blindness","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Series B Financing","leadProduct":"N-Acetylcysteine Amide","moa":"Free radical","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nacuity Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Tablet","sponsorNew":"Nacuity Pharmaceuticals \/ Foundation Fighting Blindness","highestDevelopmentStatusID":"7","companyTruncated":"Nacuity Pharmaceuticals \/ Foundation Fighting Blindness"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"N-Acetylcysteine Amide","moa":"Free radical","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nacuity Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Nacuity Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nacuity Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Cornell University","sponsor":"Lexeo Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Licensing Agreement","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Cornell University","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cornell University \/ Lexeo Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Cornell University \/ Lexeo Therapeutics"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Private Placement","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ J.P. Morgan"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Public Offering","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ J.P. Morgan"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Private Placement","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ J.P. Morgan"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"D1 Capital Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Series B Financing","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ D1 Capital Partners","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ D1 Capital Partners"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LX2006","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lexeo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"BEAM-301","moa":"GAA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Aelis Farma","sponsor":"European Commission","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"AEF0217","moa":"GABAA-alpha-5 receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aelis Farma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Aelis Farma \/ European Commission","highestDevelopmentStatusID":"7","companyTruncated":"Aelis Farma \/ European Commission"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Forge Biologics","sponsor":"Perceptive Xontogeny Venture Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Series A Financing","leadProduct":"FBX-101","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Forge Biologics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Infusion","sponsorNew":"Forge Biologics \/ Perceptive Xontogeny Venture Fund","highestDevelopmentStatusID":"7","companyTruncated":"Forge Biologics \/ Perceptive Xontogeny Venture Fund"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"PBKR03","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"PBKR03","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"PBKR03","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Undisclosed"},{"orgOrder":0,"company":"Passage Bio","sponsor":"University of Pennsylvania","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"PBKR03","moa":"Galactocerebrosidase (GALC)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ University of Pennsylvania","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ University of Pennsylvania"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Hanmi Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"HM15421","moa":"GLP-1R","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GC Biopharma \/ Hanmi Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"GC Biopharma \/ Hanmi Pharmaceutical"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"CAN103","moa":"Glucocerebrosidase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CANbridge Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"University Medical Center Groningen","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"6,6-2H2 Glucose","moa":"Glucose transporter","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"University Medical Center Groningen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pill","sponsorNew":"University Medical Center Groningen \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"University Medical Center Groningen \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"mRNA-3745","moa":"Glucose-6-phosphatase catalytic subunit 1 messenger RNA (G6PC1 mRNA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Moderna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"mRNA-3745","moa":"Glucose-6-phosphatase catalytic subunit 1 messenger RNA (G6PC1 mRNA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Moderna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Disc medicine \/ Undisclosed"},{"orgOrder":0,"company":"Disc medicine","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Collaboration","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ National Institutes of Health","highestDevelopmentStatusID":"7","companyTruncated":"Disc medicine \/ National Institutes of Health"},{"orgOrder":0,"company":"Asklepion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GREECE","productType":"Amino Acid","year":"2021","type":"Inapplicable","leadProduct":"Citrulline","moa":"G-protein coupled receptor GPCR33 (GPRC6A)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Asklepion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Asklepion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Asklepion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"MeiraGTx","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ MeiraGTx","highestDevelopmentStatusID":"7","companyTruncated":"Johnson & Johnson Innovative Medicine \/ MeiraGTx"},{"orgOrder":0,"company":"University of Illinois, Chicago","sponsor":"Global Blood Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"University of Illinois, Chicago","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Illinois, Chicago \/ Global Blood Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"University of Illinois, Chicago \/ Global Blood Therapeutics"},{"orgOrder":0,"company":"Hitachi Chemicals","sponsor":"Bluebird Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell & Gene Therapy","year":"2020","type":"Agreement","leadProduct":"Lentiglobin","moa":"Hemoglobin subunit beta (HBB)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Hitachi Chemicals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hitachi Chemicals \/ Bluebird Bio","highestDevelopmentStatusID":"7","companyTruncated":"Hitachi Chemicals \/ Bluebird Bio"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Lepunafusp Alfa","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Helixmith","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Donaperminogene Seltoplasmid","moa":"Hepatocyte growth factor (HGF)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Helixmith","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Helixmith \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Helixmith \/ Undisclosed"},{"orgOrder":0,"company":"GEMMA Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"GB221","moa":"HER2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GEMMA Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GEMMA Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GEMMA Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Hillhurst Biopharmaceuticals","sponsor":"National Heart, Lung, and Blood Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"HBI-002","moa":"HO metabolic pathway","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Hillhurst Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Hillhurst Biopharmaceuticals \/ National Heart, Lung, and Blood Institute","highestDevelopmentStatusID":"7","companyTruncated":"Hillhurst Biopharmaceuticals \/ National Heart, Lung, and Blood Institute"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ABO-101","moa":"HS","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Abeona Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Abeona Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Leerink Partners","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Leerink Partners"},{"orgOrder":0,"company":"Uniqure","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Leerink Partners","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Leerink Partners"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Minaris Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Agreement","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Minaris Regenerative Medicine","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Minaris Regenerative Medicine"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"MB-107","moa":"IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Theris Medical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Deferoxamine Mesylate","moa":"Iron","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Theris Medical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Patch","sponsorNew":"Theris Medical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Theris Medical \/ Undisclosed"},{"orgOrder":0,"company":"Atlas","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Ciclopirox","moa":"Iron | Aluminium","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atlas","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Atlas \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atlas \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Jasper Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PR001","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Prevail Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Acquisition","leadProduct":"Methylprednisolone","moa":"||Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":1.04,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":1.04,"dosageForm":"Injection","sponsorNew":"Prevail Therapeutics \/ Eli Lilly","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Eli Lilly"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Methylprednisolone","moa":"||Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Prevail Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"PR001","moa":"Lysosomal acid glucosylceramidase (GBA1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Prevail Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"AZD0120","moa":"MAPK pathway","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Alexion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Undisclosed"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Undisclosed"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Columbus Venture Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2023","type":"Series B Financing","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Columbus Venture Partners","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Columbus Venture Partners"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Undisclosed"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Undisclosed"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Undisclosed"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"EIC Accelerator","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2022","type":"Funding","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ EIC Accelerator","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ EIC Accelerator"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"REC-4881","moa":"MEK1 receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Recursion Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Recursion Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Recursion Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"REC-4881","moa":"MEK1 receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Recursion Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Recursion Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Recursion Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"REC-4881","moa":"MEK1 receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Recursion Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Recursion Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Recursion Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Fosun Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Luvometinib","moa":"MEK1|MEK2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Fosun Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Fosun Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Fosun Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Armatus Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Licensing Agreement","leadProduct":"SGT-003","moa":"Microdystrophin DNA sequence","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Solid Biosciences \/ Armatus Bio","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Armatus Bio"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Public Offering","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0.13,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Jefferies"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Collaboration","leadProduct":"SGT-001","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Solid Biosciences","amount2":0.29999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.29999999999999999,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Solid Biosciences \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AAVB-081","moa":"MYO7A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AAVantgarde Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Undisclosed"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AAVB-081","moa":"MYO7A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AAVantgarde Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Undisclosed"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AAVB-081","moa":"MYO7A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AAVantgarde Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Undisclosed"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"PYC-001","moa":"OPA1\/Tubulin\/VEGF","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"PYC-001","moa":"OPA1\/Tubulin\/VEGF","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"STK-002","moa":"Optic atrophy-1 protein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Stoke Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stoke Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Stoke Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"STK-002","moa":"Optic atrophy-1 protein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Stoke Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stoke Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Stoke Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"STK-002","moa":"Optic atrophy-1 protein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Stoke Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stoke Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Stoke Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"BiomX","sponsor":"Laidlaw & Company","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Microorganism","year":"2025","type":"Private Placement","leadProduct":"BX004","moa":"P. aeruginosa biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BiomX","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Inhalation","sponsorNew":"BiomX \/ Laidlaw & Company","highestDevelopmentStatusID":"7","companyTruncated":"BiomX \/ Laidlaw & Company"},{"orgOrder":0,"company":"BiomX","sponsor":"Deerfield Management Company","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Microorganism","year":"2024","type":"Private Placement","leadProduct":"BX004","moa":"P. aeruginosa biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BiomX","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Solution for Nebulizer","sponsorNew":"BiomX \/ Deerfield Management Company","highestDevelopmentStatusID":"7","companyTruncated":"BiomX \/ Deerfield Management Company"},{"orgOrder":0,"company":"BiomX","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Microorganism","year":"2021","type":"Inapplicable","leadProduct":"BX004","moa":"P. aeruginosa biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BiomX","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"BiomX \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BiomX \/ Undisclosed"},{"orgOrder":0,"company":"BiomX","sponsor":"Deerfield Management Company","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Microorganism","year":"2024","type":"Private Placement","leadProduct":"BX004","moa":"P. aeruginosa biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BiomX","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Solution for Nebulizer","sponsorNew":"BiomX \/ Deerfield Management Company","highestDevelopmentStatusID":"7","companyTruncated":"BiomX \/ Deerfield Management Company"},{"orgOrder":0,"company":"Adaptive Phage Therapeutics","sponsor":"BiomX","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2024","type":"Merger","leadProduct":"BX004","moa":"P. aeruginosa biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Adaptive Phage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Adaptive Phage Therapeutics \/ BiomX","highestDevelopmentStatusID":"7","companyTruncated":"Adaptive Phage Therapeutics \/ BiomX"},{"orgOrder":0,"company":"Adaptive Phage Therapeutics","sponsor":"BiomX","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2024","type":"Merger","leadProduct":"BX004","moa":"P. aeruginosa biofilm","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Adaptive Phage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Adaptive Phage Therapeutics \/ BiomX","highestDevelopmentStatusID":"7","companyTruncated":"Adaptive Phage Therapeutics \/ BiomX"},{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"CTx-PDE6b","moa":"PDE6B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Coave Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Laboratoires Thea","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"CTx-PDE6b","moa":"PDE6B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Injection","sponsorNew":"Coave Therapeutics \/ Laboratoires Thea","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Laboratoires Thea"},{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"CTx-PDE6b","moa":"PDE6B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Coave Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ASC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"ASC61","moa":"PD-L1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ASC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ASC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ASC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Children's Hospital of Philadelphia","sponsor":"Friedreich's Ataxia Research Alliance | Stealth Biotherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Elamipretide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Children's Hospital of Philadelphia","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Children's Hospital of Philadelphia \/ Friedreich's Ataxia Research Alliance | Stealth Biotherapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Children's Hospital of Philadelphia \/ Friedreich's Ataxia Research Alliance | Stealth Biotherapeutics"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Elamipretide","moa":"peptides","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Stealth Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Stealth Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Financing","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Pfizer Inc","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Pfizer Inc"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"HMI-102","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Homology Medicines \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Homology Medicines \/ Undisclosed"},{"orgOrder":0,"company":"HaiHe Biopharma Co., Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"CYH33","moa":"PI3K\u03b1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"HaiHe Biopharma Co., Ltd","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"HaiHe Biopharma Co., Ltd \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"HaiHe Biopharma Co., Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Perceptive Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Private Placement","leadProduct":"QLS-215","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Catabasis Pharmaceuticals","amount2":0.11,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"Catabasis Pharmaceuticals \/ Perceptive Advisors","highestDevelopmentStatusID":"7","companyTruncated":"Catabasis Pharmaceuticals \/ Perceptive Advisors"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"QLS-215","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Catabasis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Catabasis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Endogena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"EA-2353","moa":"Progenitor cell","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Endogena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Endogena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Endogena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Endogena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"EA-2353","moa":"Progenitor cell","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Endogena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Endogena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Endogena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Endogena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"EA-2353","moa":"Progenitor cell","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Endogena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Endogena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Endogena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Endogena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"EA-2353","moa":"Progenitor cell","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Endogena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Endogena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Endogena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Serpinpc","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Centessa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Centessa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Centessa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Serpinpc","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Centessa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Centessa Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Centessa Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Oberland Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Agreement","leadProduct":"Serpinpc","moa":"Protein C","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Centessa Pharmaceuticals","amount2":0.29999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.29999999999999999,"dosageForm":"Infusion","sponsorNew":"Centessa Pharmaceuticals \/ Oberland Capital Management","highestDevelopmentStatusID":"7","companyTruncated":"Centessa Pharmaceuticals \/ Oberland Capital Management"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"VP-001","moa":"PRPF31","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"VP-001","moa":"PRPF31","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"VP-001","moa":"PRPF31","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"VP-001","moa":"PRPF31","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"VP-001","moa":"PRPF31","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PYC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PYC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Children's Hospital Los Angeles","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Children's Hospital Los Angeles","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Children's Hospital Los Angeles \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"7","companyTruncated":"Children's Hospital Los Angeles \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"RBP4","sponsor":"Belite Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tinlarebant","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"RBP4","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"RBP4 \/ Belite Bio","highestDevelopmentStatusID":"7","companyTruncated":"RBP4 \/ Belite Bio"},{"orgOrder":0,"company":"SparingVision","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Undisclosed"},{"orgOrder":0,"company":"SparingVision","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Undisclosed"},{"orgOrder":0,"company":"SparingVision","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Undisclosed"},{"orgOrder":0,"company":"SparingVision","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Undisclosed"},{"orgOrder":0,"company":"SparingVision","sponsor":"4BIO Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Financing","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Injection","sponsorNew":"SparingVision \/ 4BIO Capital","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ 4BIO Capital"},{"orgOrder":0,"company":"SparingVision","sponsor":"Jeito Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2022","type":"Series B Financing","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Injection","sponsorNew":"SparingVision \/ Jeito Capital","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Jeito Capital"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Vico Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Vico Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Vico Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vico Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vico Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Seroba","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2024","type":"Series B Financing","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Undisclosed","sponsorNew":"Vico Therapeutics \/ Seroba","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Seroba"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"4D-125","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Public Offering","leadProduct":"4D-125","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"4D Molecular Therapeutics \/ Goldman Sachs & Co","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"4D-125","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"4D-125","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"4D Molecular Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"4D Molecular Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"4D Molecular Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"rAAV2tYF-GRK1-RPGR","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Agtc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Agtc \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"SRP-9004","moa":"Sarcoglycan alpha (SGCA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Lingyi Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"LY-M001","moa":"SARS-CoV-2 spike protein","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lingyi Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lingyi Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lingyi Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"WVE-003","moa":"SNP3","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-003","moa":"SNP3","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"WVE-003","moa":"SNP3","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Termination","leadProduct":"WVE-003","moa":"SNP3","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0.26000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.26000000000000001,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BBP-631","moa":"Steroid 21-hydroxylase (CYP21A2)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"BBP-631","moa":"Steroid 21-hydroxylase (CYP21A2)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Columbia University","sponsor":"ITBMed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Siplizumab","moa":"T-cell surface antigen CD2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Columbia University","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Columbia University \/ ITBMed","highestDevelopmentStatusID":"7","companyTruncated":"Columbia University \/ ITBMed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Linvoseltamab","moa":"TNFRSF17","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"YolTech Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"YOLT-201","moa":"TTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"YolTech Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"YolTech Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"YolTech Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"UT Southwestern Medical Center","sponsor":"GSK","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-drug Conjugate","year":"2021","type":"Inapplicable","leadProduct":"Belantamab Mafodotin","moa":"Tumor necrosis factor receptor superfamily member 17 | Tubulin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"UT Southwestern Medical Center","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"UT Southwestern Medical Center \/ GSK","highestDevelopmentStatusID":"7","companyTruncated":"UT Southwestern Medical Center \/ GSK"},{"orgOrder":0,"company":"St. Jude Children","sponsor":"Incyte Corporation | North American Consortium for Histiocytosis | Cures Within Reach","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ruxolitinib Phosphate","moa":"Tyrosine-protein kinase JAK1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"St. Jude Children","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"St. Jude Children \/ Incyte Corporation | North American Consortium for Histiocytosis | Cures Within Reach","highestDevelopmentStatusID":"7","companyTruncated":"St. Jude Children \/ Incyte Corporation | North American Consortium for Histiocytosis | Cures Within Reach"},{"orgOrder":0,"company":"Shanghai Vitalgen BioPharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"VGB-R04","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Shanghai Vitalgen BioPharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Shanghai Vitalgen BioPharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Shanghai Vitalgen BioPharma \/ Undisclosed"},{"orgOrder":0,"company":"AskBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"AB-1001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AskBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Undisclosed"},{"orgOrder":0,"company":"Ascidian Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"ACDN-01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ascidian Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ascidian Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ascidian Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ascidian Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"ACDN-01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ascidian Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ascidian Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ascidian Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Ace Cells Lab","sponsor":"European Wellness Academy","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2025","type":"Inapplicable","leadProduct":"Ace Retino","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ace Cells Lab","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ace Cells Lab \/ European Wellness Academy","highestDevelopmentStatusID":"7","companyTruncated":"Ace Cells Lab \/ European Wellness Academy"},{"orgOrder":0,"company":"AC Immune","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Vaccine","year":"2024","type":"Inapplicable","leadProduct":"ACI-24.060","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AC Immune","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AC Immune \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AC Immune \/ Undisclosed"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ACTX-401","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alcyone Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alcyone Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Alcyone Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"AMT-191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"AMT-191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Uniqure \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Undisclosed"},{"orgOrder":0,"company":"Biocad","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"ANB-004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Biocad","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Biocad \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Biocad \/ Undisclosed"},{"orgOrder":0,"company":"Biocad","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"ANB-010","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Biocad","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Biocad \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Biocad \/ Undisclosed"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2020","type":"Inapplicable","leadProduct":"AP-PA02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Armata Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2023","type":"Inapplicable","leadProduct":"AP-PA02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Armata Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2020","type":"Funding","leadProduct":"AP-PA02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Inhalation","sponsorNew":"Armata Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Innoviva","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2020","type":"Financing","leadProduct":"AP-PA02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Inhalation","sponsorNew":"Armata Pharmaceuticals \/ Innoviva","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Innoviva"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Innoviva","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2020","type":"Agreement","leadProduct":"AP-PA02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Inhalation","sponsorNew":"Armata Pharmaceuticals \/ Innoviva","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Innoviva"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Innoviva","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2020","type":"Financing","leadProduct":"AP-PA02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Inhalation","sponsorNew":"Armata Pharmaceuticals \/ Innoviva","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Innoviva"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Sickle Cell Disease Association Of America","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Partnership","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Sickle Cell Disease Association Of America","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Sickle Cell Disease Association Of America"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aruvant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aruvant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Aruvant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Lonza Group","sponsor":"Aruvant Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2021","type":"Agreement","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lonza Group","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Lonza Group \/ Aruvant Sciences","highestDevelopmentStatusID":"7","companyTruncated":"Lonza Group \/ Aruvant Sciences"},{"orgOrder":0,"company":"Aldevron","sponsor":"Aruvant Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Partnership","leadProduct":"ARU-1801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aldevron","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Aldevron \/ Aruvant Sciences","highestDevelopmentStatusID":"7","companyTruncated":"Aldevron \/ Aruvant Sciences"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ASP2957","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Astellas Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Astellas Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"The Dion Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Partnership","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ The Dion Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ The Dion Foundation"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Bpifrance","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Financing","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Infusion","sponsorNew":"Atamyo Therapeutics \/ Bpifrance","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Bpifrance"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Bain Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Series C Financing","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14999999999999999,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Bain Capital","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Bain Capital"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"ATSN-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"University of Manchester","sponsor":"CTI Clinical Trial and Consulting | Great Ormond Street Hospital for Children NHS Foundation Trust | Manchester University NHS FT | LifeArc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Autologous CD34+ Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"University of Manchester","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"University of Manchester \/ CTI Clinical Trial and Consulting | Great Ormond Street Hospital for Children NHS Foundation Trust | Manchester University NHS FT | LifeArc","highestDevelopmentStatusID":"7","companyTruncated":"University of Manchester \/ CTI Clinical Trial and Consulting | Great Ormond Street Hospital for Children NHS Foundation Trust | Manchester University NHS FT | LifeArc"},{"orgOrder":0,"company":"Avrobio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"AVR\u2011RD\u201104","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Avrobio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Undisclosed"},{"orgOrder":0,"company":"Belief BioMed","sponsor":"Shanghai Mianyi Biopharmaceutical Co., Ltd. | Shanghai Xinzhi BioMed Co., Ltd","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"BBM-D101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Belief BioMed","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Belief BioMed \/ Shanghai Mianyi Biopharmaceutical Co., Ltd. | Shanghai Xinzhi BioMed Co., Ltd","highestDevelopmentStatusID":"7","companyTruncated":"Belief BioMed \/ Shanghai Mianyi Biopharmaceutical Co., Ltd. | Shanghai Xinzhi BioMed Co., Ltd"},{"orgOrder":0,"company":"Belief BioMed","sponsor":"Shanghai MYGT Biopharmaceutical LLC","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"BBM-H803","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Belief BioMed","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Belief BioMed \/ Shanghai MYGT Biopharmaceutical LLC","highestDevelopmentStatusID":"7","companyTruncated":"Belief BioMed \/ Shanghai MYGT Biopharmaceutical LLC"},{"orgOrder":0,"company":"Belief BioMed","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BBM-H803","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Belief BioMed","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Belief BioMed \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Belief BioMed \/ Undisclosed"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BE-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Be Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Be Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BE-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Be Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Be Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"BE-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Be Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Be Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Arch Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Financing","leadProduct":"BE-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Be Biopharma","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"Be Biopharma \/ Arch Ventures","highestDevelopmentStatusID":"7","companyTruncated":"Be Biopharma \/ Arch Ventures"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Nextech","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Series C Financing","leadProduct":"BE-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Be Biopharma","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Infusion","sponsorNew":"Be Biopharma \/ Nextech","highestDevelopmentStatusID":"7","companyTruncated":"Be Biopharma \/ Nextech"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BE-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Be Biopharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Be Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"BEAM-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BEAM-101","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"BEAM-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"BEAM-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"BEAM-302","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beam Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"BI3720931","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"BI3720931","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Bionic Sight","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BS01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bionic Sight","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bionic Sight \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bionic Sight \/ Undisclosed"},{"orgOrder":0,"company":"Bionic Sight","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"BS01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bionic Sight","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bionic Sight \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bionic Sight \/ Undisclosed"},{"orgOrder":0,"company":"Bionic Sight","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"BS01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bionic Sight","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bionic Sight \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bionic Sight \/ Undisclosed"},{"orgOrder":0,"company":"Bionic Sight","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BS01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bionic Sight","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bionic Sight \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bionic Sight \/ Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Divestment","leadProduct":"CTNS-RD-04","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Avrobio \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2024","type":"Inapplicable","leadProduct":"Delpacibart Braxlosiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"Delpacibart Braxlosiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2023","type":"Inapplicable","leadProduct":"Delpacibart Braxlosiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Avidity Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"DNL126","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"DNL126","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Denali Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Denali Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sumitomo Pharma America","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"DSP-3077","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sumitomo Pharma America","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sumitomo Pharma America \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sumitomo Pharma America \/ Undisclosed"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"DT-216P2","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Design Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Design Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"DT-216P2","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Design Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Design Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"iECURE","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ECUR-506","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"iECURE \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"iECURE \/ Undisclosed"},{"orgOrder":0,"company":"iECURE","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ECUR-506","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"iECURE \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"iECURE \/ Undisclosed"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ECUR-506","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Precision BioSciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Precision BioSciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Precision BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"iECURE","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"ECUR-506","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"iECURE \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"iECURE \/ Undisclosed"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"iECURE","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"ECUR-506","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Precision BioSciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Precision BioSciences \/ iECURE","highestDevelopmentStatusID":"7","companyTruncated":"Precision BioSciences \/ iECURE"},{"orgOrder":0,"company":"ENCell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"EN001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ENCell","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ENCell \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ENCell \/ Undisclosed"},{"orgOrder":0,"company":"ENCell","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EN001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ENCell","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ENCell \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ENCell \/ Undisclosed"},{"orgOrder":0,"company":"Jiayin Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EXG001-307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jiayin Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Jiayin Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jiayin Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Jiayin Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"EXG001-307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jiayin Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Jiayin Biotech \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jiayin Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Exegenesis Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"EXG001-307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Exegenesis Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Exegenesis Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Exegenesis Bio \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Acquisition","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Syncona Limited","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Syncona Limited"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Acquisition","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Syncona Limited","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Syncona Limited"},{"orgOrder":0,"company":"Frontera Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"FT-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Frontera Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Frontera Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Frontera Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Frontera Therapeutics","sponsor":"Gene Therapy Research Center, Shariati Hospital, Tehran University of Medical Sciences, Iran | Peking Union Medical College Hospital | Henan Provincial People's Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FT-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Frontera Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Frontera Therapeutics \/ Gene Therapy Research Center, Shariati Hospital, Tehran University of Medical Sciences, Iran | Peking Union Medical College Hospital | Henan Provincial People's Hospital","highestDevelopmentStatusID":"7","companyTruncated":"Frontera Therapeutics \/ Gene Therapy Research Center, Shariati Hospital, Tehran University of Medical Sciences, Iran | Peking Union Medical College Hospital | Henan Provincial People's Hospital"},{"orgOrder":0,"company":"GeneCradle Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"GC101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GeneCradle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GeneCradle Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GeneCradle Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"GS030","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GenSight Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GenSight Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GenSight Biologics \/ Undisclosed"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"GS030","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GenSight Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GenSight Biologics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GenSight Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Grace Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"GS-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grace Science \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Grace Science \/ Undisclosed"},{"orgOrder":0,"company":"Grace Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"GS-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grace Science \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Grace Science \/ Undisclosed"},{"orgOrder":0,"company":"Grace Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"GS-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grace Science \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Grace Science \/ Undisclosed"},{"orgOrder":0,"company":"Andelyn Biosciences","sponsor":"Grace Science","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Partnership","leadProduct":"GS-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Andelyn Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Andelyn Biosciences \/ Grace Science","highestDevelopmentStatusID":"7","companyTruncated":"Andelyn Biosciences \/ Grace Science"},{"orgOrder":0,"company":"Grace Science","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"GS-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grace Science \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Grace Science \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"HGB-206","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"HGB-206","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"HGB-206","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Bluebird Bio \/ Undisclosed"},{"orgOrder":0,"company":"ReNeuron","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"hRPC Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReNeuron","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ReNeuron \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReNeuron \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"ITU512","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"JAG201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jaguar Gene Therapy","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jaguar Gene Therapy \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jaguar Gene Therapy \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JR-446","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Medipal Holdings Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JR-446","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Medipal Holdings Corporation","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Medipal Holdings Corporation"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Medipal Holdings Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"JR-446","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Medipal Holdings Corporation","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Medipal Holdings Corporation"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"JR-446","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"AskBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LION-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AskBio \/ Undisclosed"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"LY3884961","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prevail Therapeutics \/ Eli Lilly","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Eli Lilly"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"LY3884961","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Prevail Therapeutics \/ Eli Lilly","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Eli Lilly"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Bpifrance","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Financing","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Bpifrance","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Bpifrance"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Sirion Biotech","sponsor":"Mustang Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2020","type":"Licensing Agreement","leadProduct":"MB-207","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sirion Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sirion Biotech \/ Mustang Bio","highestDevelopmentStatusID":"7","companyTruncated":"Sirion Biotech \/ Mustang Bio"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Busulfan","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-207","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-207","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Undisclosed"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"mRNA-3210","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Moderna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"NGGT Suzhou Biotechnology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NGGT002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"NGGT Suzhou Biotechnology","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NGGT Suzhou Biotechnology \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"NGGT Suzhou Biotechnology \/ Undisclosed"},{"orgOrder":0,"company":"Next Generation Gene Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NGGT002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Next Generation Gene Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Next Generation Gene Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Next Generation Gene Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"NGGT","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NGGT002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"NGGT","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NGGT \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"NGGT \/ Undisclosed"},{"orgOrder":0,"company":"Neurogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"NGN-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"NS-050","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NS Pharma \/ Nippon Shinyaku","highestDevelopmentStatusID":"7","companyTruncated":"NS Pharma \/ Nippon Shinyaku"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"NS-050\/NCNP-03","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"NS-050\/NCNP-03","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NS Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"NS Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Kamau Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Nula-Cel","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Kamau Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kamau Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Kamau Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Memorial Sloan Kettering Cancer Center","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTQ923","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Memorial Sloan Kettering Cancer Center","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Memorial Sloan Kettering Cancer Center \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Memorial Sloan Kettering Cancer Center \/ Undisclosed"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTQ923","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Intellia Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Intellia Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"PM359","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prime Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prime Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Prime Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"PM359","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prime Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prime Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Prime Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"PM359","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prime Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Prime Medicine \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Prime Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"PM359","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prime Medicine","amount2":0.13,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Infusion","sponsorNew":"Prime Medicine \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Prime Medicine \/ J.P. Morgan"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Public Offering","leadProduct":"PM359","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Prime Medicine","amount2":0.16,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Infusion","sponsorNew":"Prime Medicine \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Prime Medicine \/ J.P. Morgan"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"RCT2100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"RCT2100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"RCT2100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"RCT2100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Intellia Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"REGV131","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regeneron Pharmaceuticals \/ Intellia Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Regeneron Pharmaceuticals \/ Intellia Therapeutics"},{"orgOrder":0,"company":"Respirion Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"RSP-1502","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Respirion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Respirion Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Respirion Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SAR444836","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"Medicinova","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"SAR444836","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Medicinova","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Medicinova \/ Sanofi","highestDevelopmentStatusID":"7","companyTruncated":"Medicinova \/ Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"SAR446268","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SB-007","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SpliceBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SpliceBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SpliceBio \/ Undisclosed"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SB-007","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SpliceBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SpliceBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SpliceBio \/ Undisclosed"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"SB-007","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SpliceBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SpliceBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SpliceBio \/ Undisclosed"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"SHP648","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Baxalta Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Sigilon Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"SIG-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eli Lilly \/ Sigilon Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Eli Lilly \/ Sigilon Therapeutics"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Public Offering","leadProduct":"SIG-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sigilon Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Sigilon Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"7","companyTruncated":"Sigilon Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Canada Pension Plan Investment Board","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Series B Financing","leadProduct":"SIG-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sigilon Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Sigilon Therapeutics \/ Canada Pension Plan Investment Board","highestDevelopmentStatusID":"7","companyTruncated":"Sigilon Therapeutics \/ Canada Pension Plan Investment Board"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"SIG-005","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sigilon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sigilon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sigilon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"SIG-005","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sigilon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sigilon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sigilon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"SIG-005","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sigilon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sigilon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sigilon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SIG-005","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sigilon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sigilon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sigilon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spirovant Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SP-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spirovant Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Spirovant Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spirovant Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"SPK-10001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Spark Therapeutics, Inc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spark Therapeutics, Inc \/ Undisclosed"},{"orgOrder":0,"company":"Sqy Therapeutics","sponsor":"Biotrial","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"SQY51","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sqy Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sqy Therapeutics \/ Biotrial","highestDevelopmentStatusID":"7","companyTruncated":"Sqy Therapeutics \/ Biotrial"},{"orgOrder":0,"company":"Jiangsu Gensciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"SS109","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jiangsu Gensciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jiangsu Gensciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jiangsu Gensciences \/ Undisclosed"},{"orgOrder":0,"company":"Jiangsu Gensciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"SS109","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Jiangsu Gensciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jiangsu Gensciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Jiangsu Gensciences \/ Undisclosed"},{"orgOrder":0,"company":"PRG Science & Technology Co., Ltd.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Trineumin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"PRG Science & Technology Co., Ltd.","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PRG Science & Technology Co., Ltd. \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"PRG Science & Technology Co., Ltd. \/ Undisclosed"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"vMCO-I","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nanoscope Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nanoscope Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Adaptive Phage Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2023","type":"Inapplicable","leadProduct":"WRAIR_EPa11","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Adaptive Phage Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Adaptive Phage Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Adaptive Phage Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"National Institutes of Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Microorganism","year":"2022","type":"Inapplicable","leadProduct":"WRAIR_EPa11","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"National Institutes of Health","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"National Institutes of Health \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"National Institutes of Health \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Public Offering","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ J.P. Morgan"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Public Offering","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ J.P. Morgan"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Chigenovo","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"ZVS203e","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Chigenovo","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chigenovo \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Chigenovo \/ Undisclosed"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"QR-421a","moa":"Usherin messenger RNA (USH2A mRNA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ProQR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"HMB-002","moa":"VMF\/Factor VII level","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Hemab Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"HMB-002","moa":"VMF\/Factor VII level","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hemab Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sapience Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Irinotecan","moa":"||Wnt\/beta-catenin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sapience Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sapience Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sapience Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AGTC-501","moa":"X-linked retinitis pigmentosa GTPase regulator (RPGR)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Agtc \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Leiden University Medical Centre","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Leiden University Medical Centre","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Leiden University Medical Centre"},{"orgOrder":0,"company":"Ensoma","sponsor":"Gilead Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Financing","leadProduct":"EN-374","moa":"CYBB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ensoma","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Infusion","sponsorNew":"Ensoma \/ Gilead Sciences","highestDevelopmentStatusID":"7","companyTruncated":"Ensoma \/ Gilead Sciences"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"AAVB-039","moa":"ABCA4","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AAVantgarde Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Undisclosed"},{"orgOrder":0,"company":"GeneCradle Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"GC310","moa":"ATP7B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GeneCradle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneCradle Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GeneCradle Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Alesta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"ALE1","moa":"PPi","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alesta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alesta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Alesta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Hercules Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Financing","leadProduct":"Antimony pentoxide","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Hercules Capital","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Hercules Capital"},{"orgOrder":0,"company":"Uniqure","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"Antimony pentoxide","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Leerink Partners","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Leerink Partners"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Financing","leadProduct":"RCT2100","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"7","companyTruncated":"ReCode Therapeutics \/ Cystic Fibrosis Foundation"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

    Xls
    Filters Filter
    ×
    FILTER:
    filter
    Company Name
      filter

      Year

        filter

        DEALS // DEV.

          filter

          Country

            filter
            Sponsor
              filter

              Therapeutic Area

                filter

                Study Phase

                  filter

                  Deal Type

                    filter

                    Product Type

                      filter

                      Dosage Form

                        filter

                        Lead Product

                          filter

                          Target

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

                            Therapeutic Area by Lead Product

                            Study Phase by Lead Product

                            Company by Lead Product

                            Top Deals by Deal Size (USD bn)

                            01

                            Sanofi

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

                            Lead Product(s) : SAR446268

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

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

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

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            February 25, 2025

                            Lead Product(s) : SAR446268

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            02

                            AbbVie Inc

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

                            Lead Product(s) : Etentamig

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

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

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

                            Product Name : Undisclosed

                            Product Type : Antibody, Unconjugated

                            Upfront Cash : Inapplicable

                            December 06, 2023

                            Lead Product(s) : Etentamig

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Abbvie Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            03

                            Sanofi

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

                            Lead Product(s) : SAR444836

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous ...

                            Details : SAR444836 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Phenylketonurias.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            August 02, 2023

                            Lead Product(s) : SAR444836

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            04

                            AbbVie Inc

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

                            Lead Product(s) : Fosigotifator

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Calico Life Sciences

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease

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

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 07, 2023

                            Lead Product(s) : Fosigotifator

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Calico Life Sciences

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Abbvie Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            05

                            Sanofi

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

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Translate Bio Provides MRT5005 Program Updates

                            Details : Response to COVID-19 pandemic anticipated to cause interruptions in certain clinical trial operations; updated interim data readout timing to be provided at later date.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            March 26, 2020

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            06

                            Sanofi

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

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Translate Bio Receives FDA Fast Track Designation for MRT5005 for the Treatment of Cystic Fibrosis

                            Details : First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial in the third quarter of 2020.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            February 26, 2020

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            07

                            Uniqure

                            Netherlands arrow
                            Aus. Peptide Conference
                            Not Confirmed

                            Uniqure

                            Netherlands arrow
                            Aus. Peptide Conference
                            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) : Antimony pentoxide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Leerink Partners

                            Deal Size : $345.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            uniQure Announces Closing of Upsized Public Offering

                            Details : uniQure intends to use the net proceeds to fund its commercialization readiness activities, the potential commercial launch of AMT-130, a one-time gene therapy for Huntington's disease.

                            Product Name : AMT-130

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            September 29, 2025

                            Lead Product(s) : Antimony pentoxide

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Leerink Partners

                            Deal Size : $345.0 million

                            Deal Type : Public Offering

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            08

                            Uniqure

                            Netherlands arrow
                            Aus. Peptide Conference
                            Not Confirmed

                            Uniqure

                            Netherlands arrow
                            Aus. Peptide Conference
                            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) : Antimony pentoxide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Hercules Capital

                            Deal Size : Undisclosed

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            Uniqure Secures $125M Non-Dilutive Funding, Refinances $50M Debt

                            Details : The financing deal will support the development of AMT-130, a gene therapy for the treatment of Huntington’s disease.

                            Product Name : AMT-130

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            September 24, 2025

                            Lead Product(s) : Antimony pentoxide

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Hercules Capital

                            Deal Size : Undisclosed

                            Deal Type : Financing

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            09

                            Alesta Therapeutics

                            Country arrow
                            Aus. Peptide Conference
                            Not Confirmed

                            Alesta Therapeutics

                            Country arrow
                            Aus. Peptide Conference
                            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) : ALE1

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Ad...

                            Details : ALE1 is a Small Molecule drug candidate drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Hypophosphatasia.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            September 18, 2025

                            Lead Product(s) : ALE1

                            Therapeutic Area : Genetic Disease

                            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

                            GEMMA Biotherapeutics

                            Country arrow
                            Aus. Peptide Conference
                            Not Confirmed

                            GEMMA Biotherapeutics

                            Country arrow
                            Aus. Peptide Conference
                            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) : GB221

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Safety, Tolerability & Efficacy of GB221 in Infants with Spinal Muscular Atrophy Type 1

                            Details : GB221 is a drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Spinal Muscular Atrophies of Childhood.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            July 17, 2025

                            Lead Product(s) : GB221

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth