[{"orgOrder":0,"company":"Modalis Therapeutics","sponsor":"SOLVE FSHD","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Collaboration","leadProduct":"MDL-103","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Modalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Modalis Therapeutics \/ SOLVE FSHD","highestDevelopmentStatusID":"2","companyTruncated":"Modalis Therapeutics \/ SOLVE FSHD"},{"orgOrder":0,"company":"Evotec","sponsor":"Centogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2020","type":"Expanded Collaboration","leadProduct":"Induced Pluripotent Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Evotec","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Evotec \/ Centogene","highestDevelopmentStatusID":"2","companyTruncated":"Evotec \/ Centogene"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Collaboration","leadProduct":"PBGENE-HbE","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Precision BioSciences","amount2":1.48,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.48,"dosageForm":"Undisclosed","sponsorNew":"Precision BioSciences \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"2","companyTruncated":"Precision BioSciences \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"UT Southwestern Medical Center","sponsor":"Taysha Gene Therapies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Expanded Collaboration","leadProduct":"TSHA-112","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"UT Southwestern Medical Center","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"UT Southwestern Medical Center \/ Taysha Gene Therapies","highestDevelopmentStatusID":"2","companyTruncated":"UT Southwestern Medical Center \/ Taysha Gene Therapies"},{"orgOrder":0,"company":"Tottori University","sponsor":"GC Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Tottori University","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tottori University \/ GC Pharma","highestDevelopmentStatusID":"2","companyTruncated":"Tottori University \/ GC Pharma"},{"orgOrder":0,"company":"GERO.AI","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SINGAPORE","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"GERO.AI","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GERO.AI \/ Pfizer Inc","highestDevelopmentStatusID":"2","companyTruncated":"GERO.AI \/ Pfizer Inc"},{"orgOrder":0,"company":"EdiGene","sponsor":"University of Wisconsin","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"EdiGene","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"EdiGene \/ University of Wisconsin","highestDevelopmentStatusID":"2","companyTruncated":"EdiGene \/ University of Wisconsin"},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"OMass Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"OMass Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"OMass Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"GV","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2022","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"OMass Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Inhalation","sponsorNew":"OMass Therapeutics \/ GV","highestDevelopmentStatusID":"2","companyTruncated":"OMass Therapeutics \/ GV"},{"orgOrder":0,"company":"Exicure","sponsor":"Ipsen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Exicure","amount2":1.02,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.02,"dosageForm":"Undisclosed","sponsorNew":"Exicure \/ Ipsen","highestDevelopmentStatusID":"2","companyTruncated":"Exicure \/ Ipsen"},{"orgOrder":0,"company":"Bit Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Bit Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bit Bio \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"Bit Bio \/ Undisclosed"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"ProQR Therapeutics","amount2":1.3,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.3,"dosageForm":"Undisclosed","sponsorNew":"ProQR Therapeutics \/ Eli Lilly","highestDevelopmentStatusID":"2","companyTruncated":"ProQR Therapeutics \/ Eli Lilly"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Yarrow Biotechnology","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ProQR Therapeutics \/ Yarrow Biotechnology","highestDevelopmentStatusID":"2","companyTruncated":"ProQR Therapeutics \/ Yarrow Biotechnology"},{"orgOrder":0,"company":"Bit Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Bit Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bit Bio \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"Bit Bio \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Global Blood Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2021","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Sanofi","amount2":0.34999999999999998,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Global Blood Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Sanofi \/ Global Blood Therapeutics"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Bill & Melinda Gates Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Bill & Melinda Gates Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Bill & Melinda Gates Foundation"},{"orgOrder":0,"company":"Proteros","sponsor":"Adrestia Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Proteros","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Proteros \/ Adrestia Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Proteros \/ Adrestia Therapeutics"},{"orgOrder":0,"company":"Light Chain Bioscience","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Light Chain Bioscience","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Light Chain Bioscience \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"2","companyTruncated":"Light Chain Bioscience \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Genflow Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Genflow Biosciences","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genflow Biosciences \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"Genflow Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"University of Massachusetts Medical School","sponsor":"Foundation To Fight H-Abc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"University of Massachusetts Medical School","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Massachusetts Medical School \/ Foundation To Fight H-Abc","highestDevelopmentStatusID":"2","companyTruncated":"University of Massachusetts Medical School \/ Foundation To Fight H-Abc"},{"orgOrder":0,"company":"Harvard Stem Cell Institute","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Harvard Stem Cell Institute","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Harvard Stem Cell Institute \/ Sarepta Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Harvard Stem Cell Institute \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"UMass Chan Medical School","sponsor":"QurAlis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"UMass Chan Medical School","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"UMass Chan Medical School \/ QurAlis","highestDevelopmentStatusID":"2","companyTruncated":"UMass Chan Medical School \/ QurAlis"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Eventide Asset Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Korro Bio","amount2":0.12,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Undisclosed","sponsorNew":"Korro Bio \/ Eventide Asset Management","highestDevelopmentStatusID":"2","companyTruncated":"Korro Bio \/ Eventide Asset Management"},{"orgOrder":0,"company":"CAMP4 Therapeutics","sponsor":"Fulcrum Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"CAMP4 Therapeutics","amount2":0.070000000000000007,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"Undisclosed","sponsorNew":"CAMP4 Therapeutics \/ Fulcrum Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"CAMP4 Therapeutics \/ Fulcrum Therapeutics"},{"orgOrder":0,"company":"Gensaic","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Gensaic","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gensaic \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Gensaic \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Flagship Pioneering","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Flagship Pioneering","amount2":0.11,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"Flagship Pioneering \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Flagship Pioneering \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Scribe Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Expanded Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Scribe Therapeutics","amount2":1.24,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.24,"dosageForm":"Undisclosed","sponsorNew":"Scribe Therapeutics \/ Sanofi","highestDevelopmentStatusID":"2","companyTruncated":"Scribe Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"Dyno Therapeutics","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Dyno Therapeutics","amount2":1.6200000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.6200000000000001,"dosageForm":"Undisclosed","sponsorNew":"Dyno Therapeutics \/ Astellas Pharma","highestDevelopmentStatusID":"2","companyTruncated":"Dyno Therapeutics \/ Astellas Pharma"},{"orgOrder":0,"company":"Sanegene Bio","sponsor":"Orbit Discovery","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Sanegene Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanegene Bio \/ Orbit Discovery","highestDevelopmentStatusID":"2","companyTruncated":"Sanegene Bio \/ Orbit Discovery"},{"orgOrder":0,"company":"Genevant Sciences","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Genevant Sciences","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genevant Sciences \/ Novo Nordisk","highestDevelopmentStatusID":"2","companyTruncated":"Genevant Sciences \/ Novo Nordisk"},{"orgOrder":0,"company":"Metrion Biosciences","sponsor":"KCNC1 Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Metrion Biosciences","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Metrion Biosciences \/ KCNC1 Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Metrion Biosciences \/ KCNC1 Foundation"},{"orgOrder":0,"company":"Kate Therapeutics","sponsor":"Westlake Village BioPartners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Kate Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Kate Therapeutics \/ Westlake Village BioPartners","highestDevelopmentStatusID":"2","companyTruncated":"Kate Therapeutics \/ Westlake Village BioPartners"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Genevant Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Korro Bio \/ Genevant Sciences","highestDevelopmentStatusID":"2","companyTruncated":"Korro Bio \/ Genevant Sciences"},{"orgOrder":0,"company":"Nanite","sponsor":"CMT Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Nanite","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nanite \/ CMT Research Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Nanite \/ CMT Research Foundation"},{"orgOrder":0,"company":"Aitia","sponsor":"UCB Pharma S.A","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Aitia","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aitia \/ UCB Pharma S.A","highestDevelopmentStatusID":"2","companyTruncated":"Aitia \/ UCB Pharma S.A"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Neurochase","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Spark Therapeutics, Inc \/ Neurochase","highestDevelopmentStatusID":"2","companyTruncated":"Spark Therapeutics, Inc \/ Neurochase"},{"orgOrder":0,"company":"Orna Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Orna Therapeutics","amount2":0.69999999999999996,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.69999999999999996,"dosageForm":"Undisclosed","sponsorNew":"Orna Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"Orna Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"MyoKardia","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Fulcrum Therapeutics","amount2":0.46999999999999997,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.46999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Fulcrum Therapeutics \/ MyoKardia","highestDevelopmentStatusID":"2","companyTruncated":"Fulcrum Therapeutics \/ MyoKardia"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Moderna Therapeutics","amount2":0.46000000000000002,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.46000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Moderna Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"Moderna Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Scriptr","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Scriptr","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Scriptr \/ CANbridge Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"Scriptr \/ CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Renaissance Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Design Therapeutics","amount2":0.23000000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Design Therapeutics \/ Renaissance Capital","highestDevelopmentStatusID":"2","companyTruncated":"Design Therapeutics \/ Renaissance Capital"},{"orgOrder":0,"company":"Serotiny","sponsor":"Tessera Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Serotiny","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Serotiny \/ Tessera Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Serotiny \/ Tessera Therapeutics"},{"orgOrder":0,"company":"Ceptur Therapeutics","sponsor":"Venbio Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Ceptur Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Ceptur Therapeutics \/ Venbio Partners","highestDevelopmentStatusID":"2","companyTruncated":"Ceptur Therapeutics \/ Venbio Partners"},{"orgOrder":0,"company":"NeuBase Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"NeuBase Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NeuBase Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"NeuBase Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Shape Therapeutics","sponsor":"Decheng Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Shape Therapeutics","amount2":0.11,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"Shape Therapeutics \/ Decheng Capital","highestDevelopmentStatusID":"2","companyTruncated":"Shape Therapeutics \/ Decheng Capital"},{"orgOrder":0,"company":"PAQ Therapeutics","sponsor":"Sherpa Healthcare","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"PAQ Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Undisclosed","sponsorNew":"PAQ Therapeutics \/ Sherpa Healthcare","highestDevelopmentStatusID":"2","companyTruncated":"PAQ Therapeutics \/ Sherpa Healthcare"},{"orgOrder":0,"company":"Myosana Therapeutics","sponsor":"John Ballantyne","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Myosana Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"Myosana Therapeutics \/ John Ballantyne","highestDevelopmentStatusID":"2","companyTruncated":"Myosana Therapeutics \/ John Ballantyne"},{"orgOrder":0,"company":"Altay Therapeutics","sponsor":"SOLVE FSHD","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Altay Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Altay Therapeutics \/ SOLVE FSHD","highestDevelopmentStatusID":"2","companyTruncated":"Altay Therapeutics \/ SOLVE FSHD"},{"orgOrder":0,"company":"Myosana Therapeutics","sponsor":"Cureduchenne Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Myosana Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Myosana Therapeutics \/ Cureduchenne Ventures","highestDevelopmentStatusID":"2","companyTruncated":"Myosana Therapeutics \/ Cureduchenne Ventures"},{"orgOrder":0,"company":"Shape Therapeutics","sponsor":"VectorY","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Licensing Agreement","leadProduct":"VTx-003","moa":"\u03b1v\u03b21","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Shape Therapeutics","amount2":1.2,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.2,"dosageForm":"Undisclosed","sponsorNew":"Shape Therapeutics \/ VectorY","highestDevelopmentStatusID":"2","companyTruncated":"Shape Therapeutics \/ VectorY"}]

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...

                          Therapeutic Area by Lead Product

                          Study Phase by Lead Product

                          Company by Lead Product

                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Study Phase : Discovery

                          Sponsor : Global Blood Therapeutics

                          Deal Size : $353.0 million

                          Deal Type : Licensing Agreement

                          Details : These mechanisms of the two licensed novel small moleculeare are distinct and potentially complementary to that of Oxbryta tablets, a novel hemoglobin S polymerization inhibitor approved in the United States for the treatment of SCD in patients ages 12 y...

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          March 16, 2021

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Global Blood Therapeutics

                          Deal Size : $353.0 million

                          Deal Type : Licensing Agreement

                          Sanofi Company Banner

                          02

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : VectorY gains an exclusive option to evaluate Shape’s deep-brain penetrating AAV capsid for vectorized antibody payloads, including VTx-003 in Huntington’s Disease.

                          Product Name : VTx-003

                          Product Type : Antibody, Unconjugated

                          Upfront Cash : Undisclosed

                          September 18, 2025

                          Lead Product(s) : VTx-003

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : VectorY

                          Deal Size : $1,200.0 million

                          Deal Type : Licensing Agreement

                          blank

                          03

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : The collaboration aims to advance the clinical development of company's lead program MDL-103, which is being evaluated for the treatment of FSHD,

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Undisclosed

                          June 08, 2025

                          Lead Product(s) : MDL-103

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : SOLVE FSHD

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

                          blank

                          04

                          UMass Chan Medical School

                          Country arrow
                          Pharma MES 2025
                          Not Confirmed

                          UMass Chan Medical School

                          Country arrow
                          Pharma MES 2025
                          Not Confirmed

                          Details : Under the licensing agreement, QurAlis explore the biology of FXS to determine and confirm relevant targets that could enable antisense oligonucleotide (ASO)-mediated correction for FXS.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          May 15, 2025

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : QurAlis

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

                          blank

                          05

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : Genflow launch of a new development program, focused on advancing a novel gene therapy. It is designed to combat ocular problems including several pathologies of the cornea and glaucoma.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          April 10, 2025

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          06

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : Under the collaboration, Sanofi has exercised its option for a second target as part of the companies' research collaboration to develop CRISPR-based therapeutics using Scribe’s CRISPR technologies.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : $40.0 million

                          January 13, 2025

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Sanofi

                          Deal Size : $1,240.0 million

                          Deal Type : Expanded Collaboration

                          blank

                          07

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : Through the collaboration, Vertex will utilize Orna's proprietary LNP delivery solutions to enhance it's efforts in developing next-gen gene editing therapies for patients with SCD and TDT.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : $65.0 million

                          January 07, 2025

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Vertex Pharmaceuticals

                          Deal Size : $700.0 million

                          Deal Type : Collaboration

                          blank

                          08

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : Under the collaboration, Sanofi has exercised its option for a second target as part of the companies' research collaboration to develop CRISPR-based therapeutics using Scribe’s CRISPR technologies.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : $40.0 million

                          January 03, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Sanofi

                          Deal Size : $1,240.0 million

                          Deal Type : Expanded Collaboration

                          blank

                          09

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : Under the collaboration, GC4K will develop a gene therapy for the treatment of Hereditary Spastic Paraplegia Type 56 (SPG56), a progressive neurological disease characterised by varying degrees of spasticity and muscle weakness.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          November 28, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Genetic Cures for Kids

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

                          blank

                          10

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : The collaboration aims to combine Genevant’s proprietary LNP technology with innovative mRNA-based megaTAL technology to develop an in vivo gene editing treatment for hemophilia A.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          November 06, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Novo Nordisk

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

                          blank