menu
    • menu
    [{"orgOrder":0,"company":"Airna","sponsor":"Venrock Healthcare Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Series B Financing","leadProduct":"AIR-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Airna","amount2":0.16,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Undisclosed","sponsorNew":"Airna \/ Venrock Healthcare Capital","highestDevelopmentStatusID":"4","companyTruncated":"Airna \/ Venrock Healthcare Capital"},{"orgOrder":0,"company":"Xinvento","sponsor":"Rhythm Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Peptide","year":"2023","type":"Acquisition","leadProduct":"Setmelanotide Acetate","moa":"MC4 receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Xinvento","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Xinvento \/ Rhythm Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Xinvento \/ Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"Lixte Biotechnology","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"LB-100","moa":"||PP2A","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Lixte Biotechnology","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Lixte Biotechnology \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Lixte Biotechnology \/ Inapplicable"},{"orgOrder":0,"company":"HBM Alpha Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2025","type":"Licensing Agreement","leadProduct":"HAT001","moa":"CRH","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"HBM Alpha Therapeutics","amount2":0.40000000000000002,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.40000000000000002,"dosageForm":"Undisclosed","sponsorNew":"HBM Alpha Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"HBM Alpha Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Prime Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Prime Medicine \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Prime Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Guide Therapeutics","sponsor":"Beam Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Guide Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Guide Therapeutics \/ Beam Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Guide Therapeutics \/ Beam Therapeutics"},{"orgOrder":0,"company":"AlveoGene","sponsor":"Oxford Science Enterprises","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"AVG-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AlveoGene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"AlveoGene \/ Oxford Science Enterprises","highestDevelopmentStatusID":"4","companyTruncated":"AlveoGene \/ Oxford Science Enterprises"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Eurostars","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gain Therapeutics \/ Eurostars","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Eurostars"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"GSK","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Wave Life Sciences \/ GSK","highestDevelopmentStatusID":"4","companyTruncated":"Wave Life Sciences \/ GSK"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Wave Life Sciences \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Wave Life Sciences \/ Inapplicable"},{"orgOrder":0,"company":"Attralus","sponsor":"University of Tennessee Graduate School of Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-peptide Conjugate","year":"2022","type":"Inapplicable","leadProduct":"AT-04","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Attralus \/ University of Tennessee Graduate School of Medicine","highestDevelopmentStatusID":"4","companyTruncated":"Attralus \/ University of Tennessee Graduate School of Medicine"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Inclacumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Global Blood Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Global Blood Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Tessera Therapeutics","sponsor":"Bill & Melinda Gates Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Tessera Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Intravenous Injection","sponsorNew":"Tessera Therapeutics \/ Bill & Melinda Gates Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Tessera Therapeutics \/ Bill & Melinda Gates Foundation"},{"orgOrder":0,"company":"IMMvention Therapeutix","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Partnership","leadProduct":"IMM\u2013004","moa":"BACH1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"IMMvention Therapeutix","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"IMMvention Therapeutix \/ Novo Nordisk","highestDevelopmentStatusID":"4","companyTruncated":"IMMvention Therapeutix \/ Novo Nordisk"},{"orgOrder":0,"company":"Scribe Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Expanded Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Scribe Therapeutics","amount2":1.24,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":1.24,"dosageForm":"Undisclosed","sponsorNew":"Scribe Therapeutics \/ Sanofi","highestDevelopmentStatusID":"4","companyTruncated":"Scribe Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"Scribe Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Expanded Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Scribe Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":1.24,"dosageForm":"Undisclosed","sponsorNew":"Scribe Therapeutics \/ Sanofi","highestDevelopmentStatusID":"4","companyTruncated":"Scribe Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"ExCellThera","sponsor":"IRICoR","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Cell and Gene therapy","year":"2020","type":"Funding","leadProduct":"ECT-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ExCellThera","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ExCellThera \/ IRICoR","highestDevelopmentStatusID":"4","companyTruncated":"ExCellThera \/ IRICoR"},{"orgOrder":0,"company":"Kind Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"AND017","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Kind Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kind Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Kind Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beam Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Beam Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Lipid Nanoparticles","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Intellia Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Intellia Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Pharming","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"OTL-105","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Pharming","highestDevelopmentStatusID":"4","companyTruncated":"Orchard Therapeutics \/ Pharming"},{"orgOrder":0,"company":"Quellis Biosciences Inc","sponsor":"Catabasis Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2021","type":"Acquisition","leadProduct":"QLS-215","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Quellis Biosciences Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Quellis Biosciences Inc \/ Catabasis Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Quellis Biosciences Inc \/ Catabasis Pharmaceuticals"},{"orgOrder":0,"company":"Erytech Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Red Blood Cell-Encapsulated Arginine Deiminase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Erytech Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Erytech Pharma \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Erytech Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Tenaya Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"TN-201","moa":"MYBPC3","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Tenaya Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Tenaya Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"4","companyTruncated":"Tenaya Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Tenaya Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"TN-201","moa":"MYBPC3","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Tenaya Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Tenaya Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"4","companyTruncated":"Tenaya Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Augustine Therapeutics","sponsor":"Novo Holdings","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Other Small Molecule","year":"2025","type":"Series A Financing","leadProduct":"AGT-100216","moa":"HDAC-6","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Augustine Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Oral","sponsorNew":"Augustine Therapeutics \/ Novo Holdings","highestDevelopmentStatusID":"4","companyTruncated":"Augustine Therapeutics \/ Novo Holdings"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"CMT Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"HDAC-6","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Oryzon Genomics \/ CMT Research Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Oryzon Genomics \/ CMT Research Foundation"},{"orgOrder":0,"company":"Addex Therapeutics","sponsor":"Charcot Marie Tooth Association","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"GABA-B receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Addex Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Addex Therapeutics \/ Charcot Marie Tooth Association","highestDevelopmentStatusID":"4","companyTruncated":"Addex Therapeutics \/ Charcot Marie Tooth Association"},{"orgOrder":0,"company":"Andelyn Biosciences","sponsor":"Armatus Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Andelyn Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Andelyn Biosciences \/ Armatus Bio","highestDevelopmentStatusID":"4","companyTruncated":"Andelyn Biosciences \/ Armatus Bio"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"ORY-4001","moa":"HDAC-6","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oryzon Genomics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Oryzon Genomics \/ Inapplicable"},{"orgOrder":0,"company":"Actio Biosciences","sponsor":"Canaan Partners","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":"Preclinical","graph3":"Actio Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Actio Biosciences \/ Canaan Partners","highestDevelopmentStatusID":"4","companyTruncated":"Actio Biosciences \/ Canaan Partners"},{"orgOrder":0,"company":"AmideBio","sponsor":"NIDDK","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2022","type":"Funding","leadProduct":"Glucagon","moa":"Glucagon receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AmideBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"AmideBio \/ NIDDK","highestDevelopmentStatusID":"4","companyTruncated":"AmideBio \/ NIDDK"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal","sponsorNew":"ReCode Therapeutics \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal","sponsorNew":"ReCode Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Pfizer Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal","sponsorNew":"ReCode Therapeutics \/ Pfizer Ventures","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Pfizer Ventures"},{"orgOrder":0,"company":"Nanite","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":"Preclinical","graph3":"Nanite","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal","sponsorNew":"Nanite \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Nanite \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Prime Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal","sponsorNew":"Prime Medicine \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Prime Medicine \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Sail Biomedicines","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Sail Biomedicines","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal","sponsorNew":"Sail Biomedicines \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Sail Biomedicines \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal Inhalation","sponsorNew":"ReCode Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal Inhalation","sponsorNew":"ReCode Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal Inhalation","sponsorNew":"ReCode Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Oregon State University","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oregon State University","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal Inhalation","sponsorNew":"Oregon State University \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Oregon State University \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"The Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Funding","leadProduct":"ARCT-032","moa":"CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ The Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ The Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"RCT223","moa":"CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"ReCode Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"SpliSense","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"SPL23-ASO","moa":"CFTR RNA","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SpliSense","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"SpliSense \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"SpliSense \/ Inapplicable"},{"orgOrder":0,"company":"Kither Biotech","sponsor":"Claris Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Peptide","year":"2022","type":"Series B Financing","leadProduct":"KIT2014","moa":"cAMP","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Kither Biotech","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Kither Biotech \/ Claris Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Kither Biotech \/ Claris Ventures"},{"orgOrder":0,"company":"Spirovant Sciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Doxorubicin","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Spirovant Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Spirovant Sciences \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Spirovant Sciences \/ Inapplicable"},{"orgOrder":0,"company":"Spirovant Sciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"SPIRO-2101","moa":"CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Spirovant Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Spirovant Sciences \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Spirovant Sciences \/ Inapplicable"},{"orgOrder":0,"company":"Renovion","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2020","type":"Inapplicable","leadProduct":"Ascorbic Acid","moa":"||Mucous clearance","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Renovion","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Nebulizer","sponsorNew":"Renovion \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Renovion \/ Inapplicable"},{"orgOrder":0,"company":"Anagram Therapeutics","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":"Preclinical","graph3":"Anagram Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Anagram Therapeutics \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Anagram Therapeutics \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"ETX001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Enterprise Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Enterprise Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Porosome Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"CF PT-101","moa":"CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Porosome Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Porosome Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Porosome Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Tavanta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2022","type":"Inapplicable","leadProduct":"TAVT-135","moa":"Chloride ion","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Tavanta Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tavanta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Tavanta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Oxford Biomedica","sponsor":"BlackfinBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2025","type":"Acquisition","leadProduct":"BFB-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oxford Biomedica","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oxford Biomedica \/ BlackfinBio","highestDevelopmentStatusID":"4","companyTruncated":"Oxford Biomedica \/ BlackfinBio"},{"orgOrder":0,"company":"TikunTX","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"BPN-36964","moa":"ELP1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"TikunTX","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"TikunTX \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"TikunTX \/ Inapplicable"},{"orgOrder":0,"company":"Elastin BioSciences","sponsor":"Longaevus Technologies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2023","type":"Demerger","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Elastin BioSciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Elastin BioSciences \/ Longaevus Technologies","highestDevelopmentStatusID":"4","companyTruncated":"Elastin BioSciences \/ Longaevus Technologies"},{"orgOrder":0,"company":"ViGeneron","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"VG801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ViGeneron","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ViGeneron \/ Biogen","highestDevelopmentStatusID":"4","companyTruncated":"ViGeneron \/ Biogen"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Avrobio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2021","type":"Collaboration","leadProduct":"Briquilimab","moa":"CD117","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Jasper Therapeutics \/ Avrobio","highestDevelopmentStatusID":"4","companyTruncated":"Jasper Therapeutics \/ Avrobio"},{"orgOrder":0,"company":"Codexis","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"CDX-6316","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Codexis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Codexis \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Codexis \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"FXS01","moa":"DGKk","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"SATT Conectus","sponsor":"Lysogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"FXS01","moa":"DGKk","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SATT Conectus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"SATT Conectus \/ Lysogene","highestDevelopmentStatusID":"4","companyTruncated":"SATT Conectus \/ Lysogene"},{"orgOrder":0,"company":"Lenire Biosciences","sponsor":"University College London","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Licensing Agreement","leadProduct":"VSN16R","moa":"KCa channel","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Lenire Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Lenire Biosciences \/ University College London","highestDevelopmentStatusID":"4","companyTruncated":"Lenire Biosciences \/ University College London"},{"orgOrder":0,"company":"Nova Mentis Life Science","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Nova Mentis Life Science","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Nova Mentis Life Science \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Nova Mentis Life Science \/ Inapplicable"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"FRAXA Research Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Blarcamesine","moa":"Sigma-1 receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Liquid","sponsorNew":"Anavex Life Sciences \/ FRAXA Research Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Anavex Life Sciences \/ FRAXA Research Foundation"},{"orgOrder":0,"company":"Quris","sponsor":"Welltech Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Undisclosed","year":"2022","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Quris","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Quris \/ Welltech Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Quris \/ Welltech Ventures"},{"orgOrder":0,"company":"McQuade Center for Strategic Research and Development","sponsor":"Sentinel Oncology","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Collaboration","leadProduct":"SOL784","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"McQuade Center for Strategic Research and Development","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"McQuade Center for Strategic Research and Development \/ Sentinel Oncology","highestDevelopmentStatusID":"4","companyTruncated":"McQuade Center for Strategic Research and Development \/ Sentinel Oncology"},{"orgOrder":0,"company":"Aldevron","sponsor":"AavantiBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Aldevron","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aldevron \/ AavantiBio","highestDevelopmentStatusID":"4","companyTruncated":"Aldevron \/ AavantiBio"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Neurocrine Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Collaboration","leadProduct":"VY-FXN01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Voyager Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Voyager Therapeutics, Inc \/ Neurocrine Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"Voyager Therapeutics, Inc \/ Neurocrine Biosciences"},{"orgOrder":0,"company":"Resilience","sponsor":"AavantiBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Resilience","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Resilience \/ AavantiBio","highestDevelopmentStatusID":"4","companyTruncated":"Resilience \/ AavantiBio"},{"orgOrder":0,"company":"AavantiBio","sponsor":"Solid Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Merger","leadProduct":"AVB-202","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AavantiBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AavantiBio \/ Solid Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"AavantiBio \/ Solid Biosciences"},{"orgOrder":0,"company":"AavantiBio","sponsor":"Solid Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Merger","leadProduct":"AVB-202","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AavantiBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AavantiBio \/ Solid Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"AavantiBio \/ Solid Biosciences"},{"orgOrder":0,"company":"Redx Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"RXC007","moa":"ROCK2","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Redx Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Redx Pharma \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Redx Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Catalent Pharma Solutions","sponsor":"AavantiBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Catalent Pharma Solutions","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Catalent Pharma Solutions \/ AavantiBio","highestDevelopmentStatusID":"4","companyTruncated":"Catalent Pharma Solutions \/ AavantiBio"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Cell and Gene therapy","year":"2021","type":"Series B Financing","leadProduct":"JAG101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jaguar Gene Therapy","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jaguar Gene Therapy \/ Eli Lilly","highestDevelopmentStatusID":"4","companyTruncated":"Jaguar Gene Therapy \/ Eli Lilly"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"JAG101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jaguar Gene Therapy","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jaguar Gene Therapy \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Jaguar Gene Therapy \/ Inapplicable"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"JAG101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jaguar Gene Therapy","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jaguar Gene Therapy \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Jaguar Gene Therapy \/ Inapplicable"},{"orgOrder":0,"company":"Polaryx Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"PLX-300","moa":"PPA-alpha receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Polaryx Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Polaryx Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Polaryx Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"GT-00513","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Gain Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Theranexus","sponsor":"Exelixis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2024","type":"Licensing Agreement","leadProduct":"TX01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Theranexus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Theranexus \/ Exelixis","highestDevelopmentStatusID":"4","companyTruncated":"Theranexus \/ Exelixis"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"GT-02329","moa":"GCase level","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gain Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gain Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gain Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AVR-RD-06","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Avrobio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Avrobio \/ Inapplicable"},{"orgOrder":0,"company":"Edity Therapeutics","sponsor":"Dr. Reddy's Laboratories","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Edity Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Edity Therapeutics \/ Dr. Reddy's Laboratories","highestDevelopmentStatusID":"4","companyTruncated":"Edity Therapeutics \/ Dr. Reddy's Laboratories"},{"orgOrder":0,"company":"VECT-HORUS","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2021","type":"Series D Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"VECT-HORUS","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"VECT-HORUS \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"VECT-HORUS \/ Undisclosed"},{"orgOrder":0,"company":"Silence Therapeutics","sponsor":"Hansoh Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Silence Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Silence Therapeutics \/ Hansoh Pharma","highestDevelopmentStatusID":"4","companyTruncated":"Silence Therapeutics \/ Hansoh Pharma"},{"orgOrder":0,"company":"MaxCyte","sponsor":"Prime Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"MaxCyte","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MaxCyte \/ Prime Medicine","highestDevelopmentStatusID":"4","companyTruncated":"MaxCyte \/ Prime Medicine"},{"orgOrder":0,"company":"MaxCyte","sponsor":"Be Biopharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"MaxCyte","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MaxCyte \/ Be Biopharma","highestDevelopmentStatusID":"4","companyTruncated":"MaxCyte \/ Be Biopharma"},{"orgOrder":0,"company":"Rectify Pharma","sponsor":"Atlas Venture","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":"Preclinical","graph3":"Rectify Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rectify Pharma \/ Atlas Venture","highestDevelopmentStatusID":"4","companyTruncated":"Rectify Pharma \/ Atlas Venture"},{"orgOrder":0,"company":"Caszyme","sponsor":"Integra Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"LITHUANIA","productType":"Undisclosed","year":"2024","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Caszyme","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Caszyme \/ Integra Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Caszyme \/ Integra Therapeutics"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"ATA-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Atamyo Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Atamyo Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Warden Bio","sponsor":"Kriya Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Warden Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Warden Bio \/ Kriya Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Warden Bio \/ Kriya Therapeutics"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"mRNA-3745","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Moderna Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Moderna Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Erasmus University Medical Center","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Erasmus University Medical Center","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Erasmus University Medical Center \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Erasmus University Medical Center \/ Inapplicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"AVR-RD-03","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Avrobio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Avrobio \/ Inapplicable"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2023","type":"Licensing Agreement","leadProduct":"AT845","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ Astellas Pharma","highestDevelopmentStatusID":"4","companyTruncated":"Selecta Biosciences \/ Astellas Pharma"},{"orgOrder":0,"company":"Ensoma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"EN-374","moa":"CYBB","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ensoma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ensoma \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Ensoma \/ Inapplicable"},{"orgOrder":0,"company":"BioAxone BioSciences","sponsor":"Neurelis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Acquisition","leadProduct":"BA-1049","moa":"ROCK2","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"BioAxone BioSciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"BioAxone BioSciences \/ Neurelis","highestDevelopmentStatusID":"4","companyTruncated":"BioAxone BioSciences \/ Neurelis"},{"orgOrder":0,"company":"Generation Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"iqDNA","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Generation Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Generation Bio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Generation Bio \/ Inapplicable"},{"orgOrder":0,"company":"Generation Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Generation Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Generation Bio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Generation Bio \/ Inapplicable"},{"orgOrder":0,"company":"Metagenomi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"MGX-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ Inapplicable"},{"orgOrder":0,"company":"Metagenomi","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ J.P. Morgan","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ J.P. Morgan"},{"orgOrder":0,"company":"Metagenomi","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ J.P. Morgan","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ J.P. Morgan"},{"orgOrder":0,"company":"Metagenomi","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ J.P. Morgan","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ J.P. Morgan"},{"orgOrder":0,"company":"Metagenomi","sponsor":"Novo Holding","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ Novo Holding","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ Novo Holding"},{"orgOrder":0,"company":"Poseida Therapeutics, Inc","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"P-FVIII-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Poseida Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Poseida Therapeutics, Inc \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Poseida Therapeutics, Inc \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"E-therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ETX-148","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"E-therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"E-therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"E-therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"2Seventy Bio","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Divestment","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"2Seventy Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"2Seventy Bio \/ Novo Nordisk","highestDevelopmentStatusID":"4","companyTruncated":"2Seventy Bio \/ Novo Nordisk"},{"orgOrder":0,"company":"hC Bioscience","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Large Molecule","year":"2024","type":"Inapplicable","leadProduct":"HCB-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"hC Bioscience","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"hC Bioscience \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"hC Bioscience \/ Inapplicable"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Agreement","leadProduct":"Undisclosed","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Intellia Therapeutics \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Intellia Therapeutics \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Termination","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Intellia Therapeutics \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Intellia Therapeutics \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"2Seventy Bio","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"2Seventy Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"2Seventy Bio \/ Novo Nordisk","highestDevelopmentStatusID":"4","companyTruncated":"2Seventy Bio \/ Novo Nordisk"},{"orgOrder":0,"company":"GenScript ProBio","sponsor":"Neoletix","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Collaboration","leadProduct":"Recombinant Human Coagulation Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"GenScript ProBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GenScript ProBio \/ Neoletix","highestDevelopmentStatusID":"4","companyTruncated":"GenScript ProBio \/ Neoletix"},{"orgOrder":0,"company":"Wake Forest Institute for Regenerative Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Factor VIII transgene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Wake Forest Institute for Regenerative Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Wake Forest Institute for Regenerative Medicine \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Wake Forest Institute for Regenerative Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Seattle Children\u2019s Research Institute","sponsor":"Moderna Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Seattle Children\u2019s Research Institute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Seattle Children\u2019s Research Institute \/ Moderna Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Seattle Children\u2019s Research Institute \/ Moderna Therapeutics"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"CB 2679d-GT","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Catalyst Biosciences \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Catalyst Biosciences \/ Inapplicable"},{"orgOrder":0,"company":"Chameleon Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"AAV8 IgG","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Chameleon Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chameleon Biosciences \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Chameleon Biosciences \/ Inapplicable"},{"orgOrder":0,"company":"Telomir Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Telomir-1","moa":"IL-17 receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Telomir Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Telomir Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Telomir Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Solve GNE","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"GA-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Solve GNE","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Solve GNE \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Solve GNE \/ Undisclosed"},{"orgOrder":0,"company":"Genosera","sponsor":"Solve GNE","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Agreement","leadProduct":"GA-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Genosera","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genosera \/ Solve GNE","highestDevelopmentStatusID":"4","companyTruncated":"Genosera \/ Solve GNE"},{"orgOrder":0,"company":"Repair Biotechnologies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"REP-0003","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Repair Biotechnologies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Repair Biotechnologies \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Repair Biotechnologies \/ Inapplicable"},{"orgOrder":0,"company":"Triplet Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"TTX-3360","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Triplet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Triplet Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Triplet Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ElevateBio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"LETI-101","moa":"Mutant huntingtin protein level","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ElevateBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intraparenchymal Infusion","sponsorNew":"ElevateBio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"ElevateBio \/ Inapplicable"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"SNP3","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Wave Life Sciences \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Wave Life Sciences \/ Inapplicable"},{"orgOrder":0,"company":"Anima Medical Centre","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"BELGIUM","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Huntingtin protein","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Anima Medical Centre","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Anima Medical Centre \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Anima Medical Centre \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"VectorY","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"VectorY","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"VectorY \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"VectorY \/ Inapplicable"},{"orgOrder":0,"company":"VectorY","sponsor":"Wageningen University","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"VectorY","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"VectorY \/ Wageningen University","highestDevelopmentStatusID":"4","companyTruncated":"VectorY \/ Wageningen University"},{"orgOrder":0,"company":"LoQus23 Therapeutics","sponsor":"Dementia Discovery Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"LoQus23 Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"LoQus23 Therapeutics \/ Dementia Discovery Fund","highestDevelopmentStatusID":"4","companyTruncated":"LoQus23 Therapeutics \/ Dementia Discovery Fund"},{"orgOrder":0,"company":"Harness Therapeutics","sponsor":"Epidarex Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Harness Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Harness Therapeutics \/ Epidarex Capital","highestDevelopmentStatusID":"4","companyTruncated":"Harness Therapeutics \/ Epidarex Capital"},{"orgOrder":0,"company":"LoQus23 Therapeutics","sponsor":"Forbion","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2024","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"LoQus23 Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"LoQus23 Therapeutics \/ Forbion","highestDevelopmentStatusID":"4","companyTruncated":"LoQus23 Therapeutics \/ Forbion"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Voyager Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Voyager Therapeutics, Inc \/ Pfizer Inc","highestDevelopmentStatusID":"4","companyTruncated":"Voyager Therapeutics, Inc \/ Pfizer Inc"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Voyager Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Voyager Therapeutics, Inc \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"4","companyTruncated":"Voyager Therapeutics, Inc \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Rumi Scientific","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rumi Scientific","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Rumi Scientific \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Rumi Scientific \/ Inapplicable"},{"orgOrder":0,"company":"Affiris","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRIA","productType":"Other Large Molecule","year":"2021","type":"Inapplicable","leadProduct":"mAB C6-17","moa":"Mutant huntingtin","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Affiris","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Affiris \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Affiris \/ Inapplicable"},{"orgOrder":0,"company":"BrainVectis","sponsor":"AskBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Acquisition","leadProduct":"BV-CYP01","moa":"CYP46A1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"BrainVectis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BrainVectis \/ AskBio","highestDevelopmentStatusID":"4","companyTruncated":"BrainVectis \/ AskBio"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"BV-101","moa":"CYP46A1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AskBio \/ Bayer AG","highestDevelopmentStatusID":"4","companyTruncated":"AskBio \/ Bayer AG"},{"orgOrder":0,"company":"1cBio","sponsor":"Alesta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Licensing Agreement","leadProduct":"ALE1","moa":"PPi","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"1cBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"1cBio \/ Alesta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"1cBio \/ Alesta Therapeutics"},{"orgOrder":0,"company":"Sanford Burnham Prebys Medical Discovery Institute","sponsor":"Soft Bones","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Funding","leadProduct":"Tissue Non-Specific Alkaline Phosphatase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Sanford Burnham Prebys Medical Discovery Institute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanford Burnham Prebys Medical Discovery Institute \/ Soft Bones","highestDevelopmentStatusID":"4","companyTruncated":"Sanford Burnham Prebys Medical Discovery Institute \/ Soft Bones"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Be Biopharma \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Be Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Be Biopharma \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Be Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Paradox Immunotherapeutics","sponsor":"Symbiosis Pharmaceutical Services","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Antibody","year":"2025","type":"Financing","leadProduct":"LX-96","moa":"Diseased IgG light chain","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Paradox Immunotherapeutics","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"Paradox Immunotherapeutics \/ Symbiosis Pharmaceutical Services","highestDevelopmentStatusID":"4","companyTruncated":"Paradox Immunotherapeutics \/ Symbiosis Pharmaceutical Services"},{"orgOrder":0,"company":"SalioGen Therapeutics","sponsor":"GordonMD Global","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":"Preclinical","graph3":"SalioGen Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SalioGen Therapeutics \/ GordonMD Global","highestDevelopmentStatusID":"4","companyTruncated":"SalioGen Therapeutics \/ GordonMD Global"},{"orgOrder":0,"company":"ViGeneron","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"VG801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ViGeneron","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ViGeneron \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"ViGeneron \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"SEAL Therapeutics","sponsor":"Santhera Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2022","type":"Agreement","leadProduct":"Undisclosed","moa":"Laminin-alpha-2 protein","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SEAL Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SEAL Therapeutics \/ Santhera Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"SEAL Therapeutics \/ Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Polaryx Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"PLX-300","moa":"Transcription Factor EB","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Polaryx Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Polaryx Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Polaryx Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Abigail Wexner Research Institute","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"Calpain 3","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Abigail Wexner Research Institute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Abigail Wexner Research Institute \/ Sarepta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Abigail Wexner Research Institute \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"ASC Therapeutics","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ASC Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ASC Therapeutics \/ UMass Chan Medical School","highestDevelopmentStatusID":"4","companyTruncated":"ASC Therapeutics \/ UMass Chan Medical School"},{"orgOrder":0,"company":"University of Massachusetts","sponsor":"ASC Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"University of Massachusetts","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Massachusetts \/ ASC Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"University of Massachusetts \/ ASC Therapeutics"},{"orgOrder":0,"company":"CalciMedica","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"GB-501","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CalciMedica","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrastromal Injection","sponsorNew":"CalciMedica \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"CalciMedica \/ Inapplicable"},{"orgOrder":0,"company":"Oxford Biomedica","sponsor":"Orchard Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oxford Biomedica","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Oxford Biomedica \/ Orchard Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Oxford Biomedica \/ Orchard Therapeutics"},{"orgOrder":0,"company":"Cellectis","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"IDUA","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cellectis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cellectis \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Cellectis \/ Inapplicable"},{"orgOrder":0,"company":"Capsida","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"CAP-001","moa":"IDS","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Capsida","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Capsida \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Capsida \/ Inapplicable"},{"orgOrder":0,"company":"The University of Manchester","sponsor":"Avrobio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Licensing Agreement","leadProduct":"AVR-RD-05","moa":"IDS","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"The University of Manchester","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"The University of Manchester \/ Avrobio","highestDevelopmentStatusID":"4","companyTruncated":"The University of Manchester \/ Avrobio"},{"orgOrder":0,"company":"M6P Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"M041","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"M6P Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"M6P Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"M6P Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"M6P Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"M041","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"M6P Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"M6P Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"M6P Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Phoenix Nest","sponsor":"Cure Sanfilippo Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Funding","leadProduct":"AAV-TT","moa":"HGSNAT","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Phoenix Nest","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Phoenix Nest \/ Cure Sanfilippo Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Phoenix Nest \/ Cure Sanfilippo Foundation"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Licensing Agreement","leadProduct":"BIIB115","moa":"SMN protein synthesis","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ Biogen","highestDevelopmentStatusID":"4","companyTruncated":"Ionis Pharmaceuticals \/ Biogen"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CANbridge Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"CANbridge Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"SMN1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CANbridge Pharmaceuticals \/ UMass Chan Medical School","highestDevelopmentStatusID":"4","companyTruncated":"CANbridge Pharmaceuticals \/ UMass Chan Medical School"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CANbridge Pharmaceuticals \/ UMass Chan Medical School","highestDevelopmentStatusID":"4","companyTruncated":"CANbridge Pharmaceuticals \/ UMass Chan Medical School"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CANbridge Pharmaceuticals \/ UMass Chan Medical School","highestDevelopmentStatusID":"4","companyTruncated":"CANbridge Pharmaceuticals \/ UMass Chan Medical School"},{"orgOrder":0,"company":"Casma Therapeutics","sponsor":"The Column Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series B Financing","leadProduct":"Undisclosed","moa":"TRPML1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Casma Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Casma Therapeutics \/ The Column Group","highestDevelopmentStatusID":"4","companyTruncated":"Casma Therapeutics \/ The Column Group"},{"orgOrder":0,"company":"Hansa Biopharma","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"||Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Hansa Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Hansa Biopharma \/ Sarepta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Hansa Biopharma \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"PepGen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"PGN-EDO53","moa":"Exon-53","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"PepGen \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"PepGen \/ Inapplicable"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"ENTR-601-45","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Entrada Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Entrada Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Tideglusib","moa":"GSK-3-beta","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"AMO Pharma \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"AMO Pharma \/ Inapplicable"},{"orgOrder":0,"company":"INmune Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"INmune Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"INmune Bio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"INmune Bio \/ Inapplicable"},{"orgOrder":0,"company":"Kate Therapeutics","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Acquisition","leadProduct":"KT809","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Kate Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kate Therapeutics \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"4","companyTruncated":"Kate Therapeutics \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Ractigen Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Other Large Molecule","year":"2024","type":"Inapplicable","leadProduct":"RAG-18","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ractigen Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ractigen Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Ractigen Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Ractigen Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Other Large Molecule","year":"2024","type":"Inapplicable","leadProduct":"RAG-18","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ractigen Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ractigen Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Ractigen Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"SOM Biotech","sponsor":"University of Minnesota","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2022","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SOM Biotech","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SOM Biotech \/ University of Minnesota","highestDevelopmentStatusID":"4","companyTruncated":"SOM Biotech \/ University of Minnesota"},{"orgOrder":0,"company":"Hansa Biopharma","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Enzyme","year":"2020","type":"Licensing Agreement","leadProduct":"Imlifidase","moa":"IgG-mediated immune response","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Hansa Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hansa Biopharma \/ Sarepta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Hansa Biopharma \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"MitoRx Therapeutics","sponsor":"Wren Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"MitoRx Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MitoRx Therapeutics \/ Wren Capital","highestDevelopmentStatusID":"4","companyTruncated":"MitoRx Therapeutics \/ Wren Capital"},{"orgOrder":0,"company":"Peptris Technologies","sponsor":"Revio Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Undisclosed","year":"2025","type":"Licensing Agreement","leadProduct":"RT001","moa":"Lipid peroxidation","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Peptris Technologies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Peptris Technologies \/ Revio Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Peptris Technologies \/ Revio Therapeutics"},{"orgOrder":0,"company":"Vita Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"VTA-110","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vita Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vita Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Vita Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"FORCE-M23D","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dyne Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Dyne Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Wellington Management Company","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Entrada Therapeutics \/ Wellington Management Company","highestDevelopmentStatusID":"4","companyTruncated":"Entrada Therapeutics \/ Wellington Management Company"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Prevail Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Termination","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Precision BioSciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Precision BioSciences \/ Prevail Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Precision BioSciences \/ Prevail Therapeutics"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Precision BioSciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Precision BioSciences \/ Eli Lilly","highestDevelopmentStatusID":"4","companyTruncated":"Precision BioSciences \/ Eli Lilly"},{"orgOrder":0,"company":"Code Bio","sponsor":"Northpond Ventures","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":"Preclinical","graph3":"Code Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Code Bio \/ Northpond Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Code Bio \/ Northpond Ventures"},{"orgOrder":0,"company":"IPS Heart","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"GIVI-MPC","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"IPS Heart","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IPS Heart \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"IPS Heart \/ Inapplicable"},{"orgOrder":0,"company":"Amplo Biotechnology","sponsor":"NIH-NIAMS","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Funding","leadProduct":"AMP-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Amplo Biotechnology","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amplo Biotechnology \/ NIH-NIAMS","highestDevelopmentStatusID":"4","companyTruncated":"Amplo Biotechnology \/ NIH-NIAMS"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Stoke Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Stoke Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Stoke Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vaccine","year":"2022","type":"Inapplicable","leadProduct":"mRNA-3139","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Moderna Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Moderna Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"iECURE","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"GTP-506A","moa":"||PCSK9","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"iECURE \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"iECURE \/ Inapplicable"},{"orgOrder":0,"company":"iECURE","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"GTP-506A","moa":"||PCSK9","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"iECURE \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"iECURE \/ Inapplicable"},{"orgOrder":0,"company":"NextCure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Large Molecule","year":"2024","type":"Inapplicable","leadProduct":"NC605","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NextCure","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NextCure \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"NextCure \/ Inapplicable"},{"orgOrder":0,"company":"Somagenetix","sponsor":"Vi Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2024","type":"Series A Financing","leadProduct":"SGX-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Somagenetix","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Somagenetix \/ Vi Partners","highestDevelopmentStatusID":"4","companyTruncated":"Somagenetix \/ Vi Partners"},{"orgOrder":0,"company":"APR Applied Pharma Research","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"APR-OD031","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"APR Applied Pharma Research","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"APR Applied Pharma Research \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"APR Applied Pharma Research \/ Inapplicable"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"SLC6A19","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jnana Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jnana Therapeutics \/ RA Capital Management","highestDevelopmentStatusID":"4","companyTruncated":"Jnana Therapeutics \/ RA Capital Management"},{"orgOrder":0,"company":"Glycomine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Glycomine \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Glycomine \/ Inapplicable"},{"orgOrder":0,"company":"Anakuria","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Acquisition","leadProduct":"AT-20494","moa":"mTORC1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Anakuria","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Anakuria \/ Johnson & Johnson Innovative Medicine","highestDevelopmentStatusID":"4","companyTruncated":"Anakuria \/ Johnson & Johnson Innovative Medicine"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Disc medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Bitopertin","moa":"GlyT1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"F. Hoffmann-La Roche \/ Disc medicine","highestDevelopmentStatusID":"4","companyTruncated":"F. Hoffmann-La Roche \/ Disc medicine"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Aerosol for Inhalation","sponsorNew":"ReCode Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"MPM Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ReCode Therapeutics \/ MPM Capital","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ MPM Capital"},{"orgOrder":0,"company":"Telomir Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Telomir-1","moa":"IL-17 receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Telomir Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Telomir Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Telomir Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Mirum Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"VTX-803","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ Mirum Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ Mirum Pharmaceuticals"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"VTX-803","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"GondolaBio","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Demerger","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"GondolaBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GondolaBio \/ BridgeBio Pharma","highestDevelopmentStatusID":"4","companyTruncated":"GondolaBio \/ BridgeBio Pharma"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"4BIO Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Financing","leadProduct":"Ray-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ray Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Ray Therapeutics \/ 4BIO Capital","highestDevelopmentStatusID":"4","companyTruncated":"Ray Therapeutics \/ 4BIO Capital"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Funding","leadProduct":"Ray-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ray Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Ray Therapeutics \/ California Institute for Regenerative Medicine","highestDevelopmentStatusID":"4","companyTruncated":"Ray Therapeutics \/ California Institute for Regenerative Medicine"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"Forge Biologics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Partnership","leadProduct":"Ray-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ray Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Ray Therapeutics \/ Forge Biologics","highestDevelopmentStatusID":"4","companyTruncated":"Ray Therapeutics \/ Forge Biologics"},{"orgOrder":0,"company":"Gamut Therapeutics","sponsor":"SparingVision","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Acquisition","leadProduct":"SPVN20","moa":"GIRK","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gamut Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Gamut Therapeutics \/ SparingVision","highestDevelopmentStatusID":"4","companyTruncated":"Gamut Therapeutics \/ SparingVision"},{"orgOrder":0,"company":"SparingVision","sponsor":"Tenpoint Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Divestment","leadProduct":"SPVN20","moa":"GIRK","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"SparingVision \/ Tenpoint Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"SparingVision \/ Tenpoint Therapeutics"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-103","moa":"AAV5","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-103","moa":"AAV5","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-103","moa":"AAV5","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"NR2E3-AAV","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Eyevensys","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"EYS611","moa":"Human transferrin","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Eyevensys","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eyevensys \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Eyevensys \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Massachusetts Eye And Ear","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Massachusetts Eye And Ear","highestDevelopmentStatusID":"4","companyTruncated":"Biogen \/ Massachusetts Eye And Ear"},{"orgOrder":0,"company":"IVERIC bio","sponsor":"Opus Genetics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"IVERIC bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IVERIC bio \/ Opus Genetics","highestDevelopmentStatusID":"4","companyTruncated":"IVERIC bio \/ Opus Genetics"},{"orgOrder":0,"company":"Fujifilm Cellular Dynamics","sponsor":"BlueRock Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Licensing Agreement","leadProduct":"OpCT-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Fujifilm Cellular Dynamics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Fujifilm Cellular Dynamics \/ BlueRock Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Fujifilm Cellular Dynamics \/ BlueRock Therapeutics"},{"orgOrder":0,"company":"Axovia Therapeutics","sponsor":"A Race Against Blindness","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2025","type":"Funding","leadProduct":"AXV101","moa":"BBS1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Axovia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Axovia Therapeutics \/ A Race Against Blindness","highestDevelopmentStatusID":"4","companyTruncated":"Axovia Therapeutics \/ A Race Against Blindness"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Foundation Fighting Blindness","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"OPGx-RHO","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Opus Genetics \/ Foundation Fighting Blindness","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ Foundation Fighting Blindness"},{"orgOrder":0,"company":"Cellectis","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cellectis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Cellectis \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Cellectis \/ Inapplicable"},{"orgOrder":0,"company":"Emendo Biotherapeutics","sponsor":"AnGes","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Acquisition","leadProduct":"EMD-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Emendo Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Emendo Biotherapeutics \/ AnGes","highestDevelopmentStatusID":"4","companyTruncated":"Emendo Biotherapeutics \/ AnGes"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"TSHA-103","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Genetic Cures for Kids","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Charles River Laboratories, Inc \/ Genetic Cures for Kids","highestDevelopmentStatusID":"4","companyTruncated":"Charles River Laboratories, Inc \/ Genetic Cures for Kids"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Cure AP-4","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Charles River Laboratories, Inc \/ Cure AP-4","highestDevelopmentStatusID":"4","companyTruncated":"Charles River Laboratories, Inc \/ Cure AP-4"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Life Science Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vico Therapeutics \/ Life Science Partners","highestDevelopmentStatusID":"4","companyTruncated":"Vico Therapeutics \/ Life Science Partners"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"VMCO-I","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Nanoscope Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Nanoscope Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Intergalactic Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"IG-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intergalactic Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Intergalactic Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Intergalactic Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Oak Bay Biosciences","sponsor":"FrontFundr","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2021","type":"Funding","leadProduct":"Undisclosed","moa":"Complement pathway","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oak Bay Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oak Bay Biosciences \/ FrontFundr","highestDevelopmentStatusID":"4","companyTruncated":"Oak Bay Biosciences \/ FrontFundr"},{"orgOrder":0,"company":"reVision Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"REV-0100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"reVision Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"reVision Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"reVision Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"ABO-504","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Abeona Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Abeona Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Acadia Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Stoke Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Stoke Therapeutics \/ Acadia Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Stoke Therapeutics \/ Acadia Pharmaceuticals"},{"orgOrder":0,"company":"4M Therapeutics","sponsor":"SynGAP Research Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Funding","leadProduct":"4MT2001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"4M Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"4M Therapeutics \/ SynGAP Research Fund","highestDevelopmentStatusID":"4","companyTruncated":"4M Therapeutics \/ SynGAP Research Fund"},{"orgOrder":0,"company":"Diagonal Therapeutics","sponsor":"BVF Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Diagonal Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Diagonal Therapeutics \/ BVF Partners","highestDevelopmentStatusID":"4","companyTruncated":"Diagonal Therapeutics \/ BVF Partners"},{"orgOrder":0,"company":"Rezolute","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Ersodetug","moa":"Insulin allosteric site receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rezolute \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Rezolute \/ Inapplicable"},{"orgOrder":0,"company":"Castle Creek Biosciences","sponsor":"Paragon Biosciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"LV-FAH","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Castle Creek Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Castle Creek Biosciences \/ Paragon Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"Castle Creek Biosciences \/ Paragon Biosciences"},{"orgOrder":0,"company":"Versantis","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"VS-01","moa":"Ammonia clearance","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Versantis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intraperitoneal Injection","sponsorNew":"Versantis \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Versantis \/ Inapplicable"},{"orgOrder":0,"company":"Denovo Biopharma","sponsor":"Aytu BioScience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Enzastaurin","moa":"PKC-beta","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Denovo Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Denovo Biopharma \/ Aytu BioScience","highestDevelopmentStatusID":"4","companyTruncated":"Denovo Biopharma \/ Aytu BioScience"},{"orgOrder":0,"company":"Rumpus Therapeutics","sponsor":"Aytu BioScience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Enzastaurin","moa":"PKC-beta","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rumpus Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Rumpus Therapeutics \/ Aytu BioScience","highestDevelopmentStatusID":"4","companyTruncated":"Rumpus Therapeutics \/ Aytu BioScience"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"French Government","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"VTX-806","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ French Government","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ French Government"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"VTX-806","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"NephroDI Therapeutics","sponsor":"Xontogeny, LLC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Financing","leadProduct":"NDI-5033","moa":"AMPK","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NephroDI Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"NephroDI Therapeutics \/ Xontogeny, LLC","highestDevelopmentStatusID":"4","companyTruncated":"NephroDI Therapeutics \/ Xontogeny, LLC"}]

    Find Drugs for Genetic Disease in Preclinical Development

    Xls
    Filters Filter
    ×
    FILTER:
    filter
    Company Name
      filter

      Year

        filter

        DEALS // DEV.

          filter

          Country

            filter
            Sponsor
              filter

              Therapeutic Area

                filter

                Study Phase

                  filter

                  Deal Type

                    filter

                    Product Type

                      filter

                      Dosage Form

                        filter

                        Lead Product

                          filter

                          Target

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

                            Therapeutic Area by Lead Product

                            Study Phase by Lead Product

                            Company by Lead Product

                            Top Deals by Deal Size (USD bn)

                            01

                            Airna

                            U.S.A arrow
                            WRIB
                            Not Confirmed

                            Airna

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

                            Lead Product(s) : AIR-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Venrock Healthcare Capital

                            Deal Size : $155.0 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            Airna Raises $155 Million Series B Financing for Alpha-1 Antitrypsin Deficiency

                            Details : The financing aims to advance company's RNA editing product candidate AIR-001, which is being evaluated for the treatment of Alpha-1 antitrypsin deficiency.

                            Product Name : AIR-001

                            Product Type : Oligonucleotide

                            Upfront Cash : Undisclosed

                            April 01, 2025

                            Lead Product(s) : AIR-001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Venrock Healthcare Capital

                            Deal Size : $155.0 million

                            Deal Type : Series B Financing

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            02

                            Augustine Therapeutics

                            Belgium arrow
                            German Wound Congress
                            Not Confirmed

                            Augustine Therapeutics

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

                            Lead Product(s) : AGT-100216

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Novo Holdings

                            Deal Size : $84.8 million

                            Deal Type : Series A Financing

                            DEALS_DEV arrow-down

                            Augustine Raises EUR 78M/USD 85M in Oversubscribed Series A Financing

                            Details : The financing will support the company's lead product AGT-100216, which is being evaluated for the treatment of Charcot-Marie-Tooth.

                            Product Name : AGT-100216

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            March 24, 2025

                            Lead Product(s) : AGT-100216

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Novo Holdings

                            Deal Size : $84.8 million

                            Deal Type : Series A Financing

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            03

                            Prime Medicine

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Prime Medicine

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

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Prime Medicine Unveils Program for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)

                            Details : This program leverages the Company’s universal liver lipid nanoparticle (LNP) to edit the E342K (Pi*Z) mutation in the SERPINA1 gene, the prevalent disease-causing mutation in AATD.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            March 18, 2025

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            04

                            Peptris Technologies

                            India arrow
                            German Wound Congress
                            Not Confirmed

                            Peptris Technologies

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

                            Lead Product(s) : RT001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Revio Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Peptris Technologies out-licenses AI-discovered drug candidate to Revio Therapeutics

                            Details : Under the licensing agreement, Revio will hold commercialisation rights to PEPR124 (RT001) in all markets except in BRICS countries, which will be retained by Peptris.

                            Product Name : PEPR124

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            March 17, 2025

                            Lead Product(s) : RT001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Revio Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            05

                            HBM Alpha Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            HBM Alpha Therapeutics

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

                            Lead Product(s) : HAT001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : $395.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            HBM Alpha Enters Collaboration for Novel Endocrine Therapy Development

                            Details : Under the licensing agreement, the partner gains exclusive global rights, excluding Greater China (mainland China, Taiwan, Hong Kong, and Macau), to develop and commercialize HAT001 for treatting CAH.

                            Product Name : HAT001

                            Product Type : Antibody

                            Upfront Cash : Undisclosed

                            February 25, 2025

                            Lead Product(s) : HAT001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : $395.0 million

                            Deal Type : Licensing Agreement

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            06

                            Porosome Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Porosome Therapeutics

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

                            Lead Product(s) : CF PT-101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Grants Orphan Drug Designation to Revolutionary Cystic Fibrosis Therapy

                            Details : CF PT-101 is a porosome reconstitution therapy, which is currently being evaluated for the treatment of cystic fibrosis.

                            Product Name : CF PT-101

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 25, 2025

                            Lead Product(s) : CF PT-101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            07

                            Paradox Immunotherapeutics

                            Canada arrow
                            German Wound Congress
                            Not Confirmed

                            Paradox Immunotherapeutics

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

                            Lead Product(s) : LX-96

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Symbiosis Pharmaceutical Services

                            Deal Size : $10.0 million

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            Paradox Gets $10M from SymBiosis to Advance Protein Misfolding Disease Therapies

                            Details : The financing will be used to advance Paradox’s therapeutic pipeline, which includes LX-96, a monoclonal antibody designed for the treatment of late-stage immunoglobulin light chain (AL) amyloidosis.

                            Product Name : LX-96

                            Product Type : Antibody

                            Upfront Cash : Undisclosed

                            February 25, 2025

                            Lead Product(s) : LX-96

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Symbiosis Pharmaceutical Services

                            Deal Size : $10.0 million

                            Deal Type : Financing

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            08

                            ElevateBio

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            ElevateBio

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

                            Lead Product(s) : LETI-101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            ElevateBio Announces Compelling Preclinical Data of LETI-101 for Huntington's Disease

                            Details : LETI-101 is a selective reducer of disease-causing mutant huntingtin (mHTT) protein, It is being evaluated in patients with huntington's disease.

                            Product Name : LETI-101

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 24, 2025

                            Lead Product(s) : LETI-101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            09

                            Ensoma

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Ensoma

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

                            Lead Product(s) : EN-374

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Ensoma's EN-374 Gets Rare Pediatric, Orphan Drug Status for Granulomatous Disease

                            Details : EN-374 is a CD46/CYBB modulator, gene therapy candidate, which is currently being evaluated for the treatment of X-linked chronic granulomatous disease (X-CGD).

                            Product Name : EN-374

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 13, 2025

                            Lead Product(s) : EN-374

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            10

                            E-therapeutics

                            United Kingdom arrow
                            German Wound Congress
                            Not Confirmed

                            E-therapeutics

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

                            Lead Product(s) : ETX-148

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            e-therapeutics to Present Preclinical Data on ETX-148 at EAHAD 2025

                            Details : ETX-148 is a potential pan-hemophilia rebalancing agent. It utilizes e-therapeutics' GalOmic GalNAc-siRNA technology to silence novel gene targets in hepatocytes.

                            Product Name : ETX-148

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            February 05, 2025

                            Lead Product(s) : ETX-148

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth