[{"orgOrder":0,"company":"Light Chain Bioscience","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Large molecule","year":"2020","type":"Collaboration","leadProduct":"Bispecific antibodies","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Light Chain Bioscience","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Light Chain Bioscience \/ Takeda","highestDevelopmentStatusID":"2","companyTruncated":"Light Chain Bioscience \/ Takeda"},{"orgOrder":0,"company":"Nimbus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Endocrinology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Nimbus Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Nimbus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"Nimbus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Gero","sponsor":"Bulba Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SINGAPORE","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Gero","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Gero \/ Bulba Ventures","highestDevelopmentStatusID":"2","companyTruncated":"Gero \/ Bulba Ventures"},{"orgOrder":0,"company":"Evotec","sponsor":"Centogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Expanded Collaboration","leadProduct":"Induced pluripotent stem cell","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Evotec","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Evotec \/ CENTOGENE","highestDevelopmentStatusID":"2","companyTruncated":"Evotec \/ CENTOGENE"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Collaboration","leadProduct":"LNP based mRNA Therapeutics","moa":"CFTR","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Moderna Therapeutics","amount2":0.46000000000000002,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.46000000000000002,"dosageForm":"","sponsorNew":"Moderna Therapeutics \/ Vertex","highestDevelopmentStatusID":"2","companyTruncated":"Moderna Therapeutics \/ Vertex"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"MyoKardia","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Fulcrum Therapeutics","amount2":0.46999999999999997,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.46999999999999997,"dosageForm":"","sponsorNew":"Fulcrum Therapeutics \/ MyoKardia","highestDevelopmentStatusID":"2","companyTruncated":"Fulcrum Therapeutics \/ MyoKardia"},{"orgOrder":0,"company":"University of Massachusetts Medical School","sponsor":"Foundation to Fight H-ABC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Agreement","leadProduct":"AAV-based gene","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"University of Massachusetts Medical School","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","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":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"AAV9-based gene therapy","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"UT Southwestern Medical Center","sponsor":"Taysha Gene Therapies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Expanded Collaboration","leadProduct":"TSHA-112","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"UT Southwestern Medical Center","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"UT Southwestern Medical Center \/ Taysha Gene Therapies","highestDevelopmentStatusID":"2","companyTruncated":"UT Southwestern Medical Center \/ Taysha Gene Therapies"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Bill & Melinda Gates Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Bill & Melinda Gates Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Bill & Melinda Gates Foundation"},{"orgOrder":0,"company":"Sanofi","sponsor":"Global Blood Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Sanofi","amount2":0.34999999999999998,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"","sponsorNew":"Sanofi \/ Global Blood Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Sanofi \/ Global Blood Therapeutics"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Renaissance Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Public Offering","leadProduct":"Gene targeted chimeras","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Design Therapeutics","amount2":0.23000000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"","sponsorNew":"Design Therapeutics \/ Renaissance Capital","highestDevelopmentStatusID":"2","companyTruncated":"Design Therapeutics \/ Renaissance Capital"},{"orgOrder":0,"company":"PAQ Therapeutics","sponsor":"Sherpa Healthcare","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Series A Financing","leadProduct":"Autophagosome-tethering Compound Based Therapy","moa":"Ubiquitin-proteosome pathway","graph1":"Genetic Disease","graph2":"Discovery","graph3":"PAQ Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"PAQ Therapeutics \/ Sherpa Healthcare","highestDevelopmentStatusID":"2","companyTruncated":"PAQ Therapeutics \/ Sherpa Healthcare"},{"orgOrder":0,"company":"Shape Therapeutics","sponsor":"Decheng Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Series B Financing","leadProduct":"RNA Therapeutics","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Shape Therapeutics","amount2":0.11,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"","sponsorNew":"Shape Therapeutics \/ Decheng Capital","highestDevelopmentStatusID":"2","companyTruncated":"Shape Therapeutics \/ Decheng Capital"},{"orgOrder":0,"company":"Exicure","sponsor":"Ipsen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"SNA-based Therapeutics","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Exicure","amount2":1.02,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.02,"dosageForm":"","sponsorNew":"Exicure \/ Ipsen","highestDevelopmentStatusID":"2","companyTruncated":"Exicure \/ Ipsen"},{"orgOrder":0,"company":"Tottori University","sponsor":"GC Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Small molecule","year":"2021","type":"Collaboration","leadProduct":"Chaperone Therapy","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Tottori University","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Tottori University \/ GC Pharma","highestDevelopmentStatusID":"2","companyTruncated":"Tottori University \/ GC Pharma"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Collaboration","leadProduct":"Editing Oligonucleotides","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"ProQR Therapeutics","amount2":1.3,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.3,"dosageForm":"","sponsorNew":"ProQR Therapeutics \/ Eli Lilly","highestDevelopmentStatusID":"2","companyTruncated":"ProQR Therapeutics \/ Eli Lilly"},{"orgOrder":0,"company":"McGill University","sponsor":"Clarus","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Small molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Coenzyme Q10","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"McGill University","amount2":0.040000000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Oral","sponsorNew":"McGill University \/ Clarus","highestDevelopmentStatusID":"2","companyTruncated":"McGill University \/ Clarus"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"AAV Capsids","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Voyager Therapeutics, Inc","amount2":0.63,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.63,"dosageForm":"","sponsorNew":"Voyager Therapeutics, Inc \/ Pfizer","highestDevelopmentStatusID":"2","companyTruncated":"Voyager Therapeutics, Inc \/ Pfizer"},{"orgOrder":0,"company":"Scriptr","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Scriptr","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Scriptr \/ CANbridge Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"Scriptr \/ CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Flagship Pioneering","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Flagship Pioneering","amount2":0.11,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Dosage Strength\/Form","sponsorNew":"Flagship Pioneering \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Flagship Pioneering \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"EdiGene","sponsor":"University of Wisconsin\u2013Madison","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Collaboration","leadProduct":"Short Engineered ADAR-recruiting RNAs","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"EdiGene","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Dosage Strength\/Form","sponsorNew":"EdiGene \/ University of Wisconsin\u2013Madison","highestDevelopmentStatusID":"2","companyTruncated":"EdiGene \/ University of Wisconsin\u2013Madison"},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"OMass Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"OMass Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"OMass Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"LoQus23 Therapeutics","sponsor":"Dementia Discovery Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Financing","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"LoQus23 Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Oral","sponsorNew":"LoQus23 Therapeutics \/ Dementia Discovery Fund","highestDevelopmentStatusID":"2","companyTruncated":"LoQus23 Therapeutics \/ Dementia Discovery Fund"},{"orgOrder":0,"company":"Dyno Therapeutics","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"AAV Capsids","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Dyno Therapeutics","amount2":1.6200000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.6200000000000001,"dosageForm":"Dosage Strength\/Form","sponsorNew":"Dyno Therapeutics \/ Astellas Pharma","highestDevelopmentStatusID":"2","companyTruncated":"Dyno Therapeutics \/ Astellas Pharma"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Eventide Asset Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Series B Financing","leadProduct":"Genetic Medicine","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Korro Bio","amount2":0.12,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"","sponsorNew":"Korro Bio \/ Eventide Asset Management","highestDevelopmentStatusID":"2","companyTruncated":"Korro Bio \/ Eventide Asset Management"},{"orgOrder":0,"company":"Serotiny","sponsor":"Tessera Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"Genetic Medicine","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Serotiny","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Serotiny \/ Tessera Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Serotiny \/ Tessera Therapeutics"},{"orgOrder":0,"company":"Ceptur Therapeutics","sponsor":"venBio Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Series A Financing","leadProduct":"Oligonucleotide-based RNA Therapeutics","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Ceptur Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"Ceptur Therapeutics \/ venBio Partners","highestDevelopmentStatusID":"2","companyTruncated":"Ceptur Therapeutics \/ venBio Partners"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Yarrow Biotechnology","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Collaboration","leadProduct":"Antisense Oligonucleotide Therapy","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"ProQR Therapeutics \/ Yarrow Biotechnology","highestDevelopmentStatusID":"2","companyTruncated":"ProQR Therapeutics \/ Yarrow Biotechnology"},{"orgOrder":0,"company":"Scenic Biotech","sponsor":"Barth Syndrome Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Scenic Biotech","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Scenic Biotech \/ Barth Syndrome Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Scenic Biotech \/ Barth Syndrome Foundation"},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"GV","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Series B Financing","leadProduct":"Undisclosed","moa":"","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":"Codexis","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Codexis","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Codexis \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"Codexis \/ Not Applicable"},{"orgOrder":0,"company":"Bit Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Bit Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Bit Bio \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"Bit Bio \/ Not Applicable"},{"orgOrder":0,"company":"LUCA Science","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"Mitochondrial-based Therapy","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"LUCA Science","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"LUCA Science \/ Kyowa Kirin Co.","highestDevelopmentStatusID":"2","companyTruncated":"LUCA Science \/ Kyowa Kirin Co."},{"orgOrder":0,"company":"Bit Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Bit Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Bit Bio \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"Bit Bio \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Baylor College of Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ Baylor College of Medicine","highestDevelopmentStatusID":"2","companyTruncated":"BridgeBio Pharma \/ Baylor College of Medicine"},{"orgOrder":0,"company":"Nimbus Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"AMPK","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Nimbus Therapeutics","amount2":0.5,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.5,"dosageForm":"","sponsorNew":"Nimbus Therapeutics \/ Eli Lilly","highestDevelopmentStatusID":"2","companyTruncated":"Nimbus Therapeutics \/ Eli Lilly"},{"orgOrder":0,"company":"NeuBase Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2022","type":"Agreement","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"NeuBase Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"NeuBase Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"NeuBase Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Proteros","sponsor":"Adrestia Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Proteros","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Proteros \/ Adrestia Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Proteros \/ Adrestia Therapeutics"},{"orgOrder":0,"company":"Replay Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"SynHSV Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Replay Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Replay Bio \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"Replay Bio \/ Not Applicable"},{"orgOrder":0,"company":"Metrion Biosciences","sponsor":"KCNC1 Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Metrion Biosciences","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Metrion Biosciences \/ KCNC1 Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Metrion Biosciences \/ KCNC1 Foundation"},{"orgOrder":0,"company":"Exicure","sponsor":"Ipsen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Termination","leadProduct":"SNA-based Therapeutic","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Exicure","amount2":1.02,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.02,"dosageForm":"","sponsorNew":"Exicure \/ Ipsen","highestDevelopmentStatusID":"2","companyTruncated":"Exicure \/ Ipsen"},{"orgOrder":0,"company":"Sanegene Bio","sponsor":"Orbit Discovery","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Collaboration","leadProduct":"RNAi-based Therapy","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Sanegene Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Sanegene Bio \/ Orbit Discovery","highestDevelopmentStatusID":"2","companyTruncated":"Sanegene Bio \/ Orbit Discovery"},{"orgOrder":0,"company":"GERO.AI","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"GERO.AI","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"GERO.AI \/ Pfizer Inc","highestDevelopmentStatusID":"2","companyTruncated":"GERO.AI \/ Pfizer Inc"},{"orgOrder":0,"company":"Gensaic","sponsor":"CF Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Funding","leadProduct":"CFTR-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Gensaic","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Gensaic \/ CF Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Gensaic \/ CF Foundation"},{"orgOrder":0,"company":"Myosana Therapeutics","sponsor":"John Ballantyne","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Myosana Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"","sponsorNew":"Myosana Therapeutics \/ John Ballantyne","highestDevelopmentStatusID":"2","companyTruncated":"Myosana Therapeutics \/ John Ballantyne"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Neurochase","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Spark Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Spark Therapeutics, Inc \/ Neurochase","highestDevelopmentStatusID":"2","companyTruncated":"Spark Therapeutics, Inc \/ Neurochase"},{"orgOrder":0,"company":"Nanite","sponsor":"CMT Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Partnership","leadProduct":"Antisense Oligonucleotide","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Nanite","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","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":"","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Aitia","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Aitia \/ UCB Pharma S.A","highestDevelopmentStatusID":"2","companyTruncated":"Aitia \/ UCB Pharma S.A"},{"orgOrder":0,"company":"Kate Therapeutics","sponsor":"Westlake Village BioPartners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Series A Financing","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Kate Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"","sponsorNew":"Kate Therapeutics \/ Westlake Village BioPartners","highestDevelopmentStatusID":"2","companyTruncated":"Kate Therapeutics \/ Westlake Village BioPartners"},{"orgOrder":0,"company":"IRBM","sponsor":"Merck & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2023","type":"Expanded Collaboration","leadProduct":"Peptide Therapy","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"IRBM","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"IRBM \/ Merck & Co. Inc.","highestDevelopmentStatusID":"2","companyTruncated":"IRBM \/ Merck & Co. Inc."},{"orgOrder":0,"company":"Poolbeg Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Vaccine","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Poolbeg Pharma","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Poolbeg Pharma \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"Poolbeg Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Genevant Sciences","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Genevant Sciences","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Genevant Sciences \/ Novo Nordisk","highestDevelopmentStatusID":"2","companyTruncated":"Genevant Sciences \/ Novo Nordisk"},{"orgOrder":0,"company":"Fractyl Health","sponsor":"Michigan Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Collaboration","leadProduct":"Pancreatic Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Fractyl Health","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Fractyl Health \/ Michigan Medicine","highestDevelopmentStatusID":"2","companyTruncated":"Fractyl Health \/ Michigan Medicine"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Genetic Cures for Kids","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Charles River Laboratories, Inc \/ Genetic Cures for Kids","highestDevelopmentStatusID":"2","companyTruncated":"Charles River Laboratories, Inc \/ Genetic Cures for Kids"},{"orgOrder":0,"company":"Scribe Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Expanded Collaboration","leadProduct":"CRISPR-based Therapy","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Scribe Therapeutics","amount2":1.24,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":1.24,"dosageForm":"","sponsorNew":"Scribe Therapeutics \/ Sanofi","highestDevelopmentStatusID":"2","companyTruncated":"Scribe Therapeutics \/ Sanofi"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
Filters
×
FILTER:
Company Name
Year
DEALS // DEV.
Country
Sponsor
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target