[{"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,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"OMass Therapeutics \/ GV","highestDevelopmentStatusID":"2","companyTruncated":"OMass Therapeutics \/ GV"},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"Inapplicable","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":"Oral Inhalation","sponsorNew":"OMass Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"2","companyTruncated":"OMass Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Eventide Asset Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Series B Financing","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 \/ Eventide Asset Management","highestDevelopmentStatusID":"2","companyTruncated":"Korro Bio \/ Eventide Asset Management"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Genevant Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","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":"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,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CAMP4 Therapeutics \/ Fulcrum Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"CAMP4 Therapeutics \/ Fulcrum Therapeutics"},{"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,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"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":"Orna Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","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":"Precision BioSciences","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"PBGENE-HbE","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Precision BioSciences","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Precision BioSciences \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"2","companyTruncated":"Precision BioSciences \/ Novartis Pharmaceuticals Corporation"},{"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,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Fulcrum Therapeutics \/ MyoKardia","highestDevelopmentStatusID":"2","companyTruncated":"Fulcrum Therapeutics \/ MyoKardia"},{"orgOrder":0,"company":"Nanite","sponsor":"CMT Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Large Molecule","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":"Oral","sponsorNew":"Nanite \/ CMT Research Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Nanite \/ CMT Research Foundation"},{"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":"Nasal","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,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal","sponsorNew":"Flagship Pioneering \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Flagship Pioneering \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"CFTR","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal","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":"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":"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":"Evotec","sponsor":"Centogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell and 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":"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":"Design Therapeutics","sponsor":"Renaissance Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Design Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Design Therapeutics \/ Renaissance Capital","highestDevelopmentStatusID":"2","companyTruncated":"Design Therapeutics \/ Renaissance Capital"},{"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":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Scribe Therapeutics \/ Sanofi","highestDevelopmentStatusID":"2","companyTruncated":"Scribe Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"Serotiny","sponsor":"Tessera Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","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":"EdiGene","sponsor":"University of Wisconsin","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Other Large Molecule","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":"ProQR Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","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 \/ 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":"Dyno Therapeutics","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Dyno Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"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":"Ceptur Therapeutics","sponsor":"Venbio Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Ceptur Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"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":"Oligonucleotide","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Shape Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"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":"Ubiquitin-proteosome pathway","graph1":"Genetic Disease","graph2":"Discovery","graph3":"PAQ Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PAQ Therapeutics \/ Sherpa Healthcare","highestDevelopmentStatusID":"2","companyTruncated":"PAQ Therapeutics \/ Sherpa Healthcare"},{"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":"Light Chain Bioscience","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Factor VIII","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":"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":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Exicure \/ Ipsen","highestDevelopmentStatusID":"2","companyTruncated":"Exicure \/ Ipsen"},{"orgOrder":0,"company":"Bit Bio","sponsor":"Inapplicable","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":"Oral","sponsorNew":"Bit Bio \/ Inapplicable","highestDevelopmentStatusID":"2","companyTruncated":"Bit Bio \/ Inapplicable"},{"orgOrder":0,"company":"Bit Bio","sponsor":"Inapplicable","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":"Oral","sponsorNew":"Bit Bio \/ Inapplicable","highestDevelopmentStatusID":"2","companyTruncated":"Bit Bio \/ Inapplicable"},{"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":"Oral","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":"Oral","sponsorNew":"Spark Therapeutics, Inc \/ Neurochase","highestDevelopmentStatusID":"2","companyTruncated":"Spark Therapeutics, Inc \/ Neurochase"},{"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":"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":"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,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Myosana Therapeutics \/ John Ballantyne","highestDevelopmentStatusID":"2","companyTruncated":"Myosana Therapeutics \/ John Ballantyne"},{"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":"Dystrophin","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":"Harvard Stem Cell Institute","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","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":"Metrion Biosciences","sponsor":"KCNC1 Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","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":"UT Southwestern Medical Center","sponsor":"Taysha Gene Therapies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and 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":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"TSHA-105","moa":"AChE","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"2","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"UMass Chan Medical School","sponsor":"QurAlis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"FMRP","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"}]
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
Reset All
Sanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.
