menu
    • menu
    [{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"ETX001","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Enterprise Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Enterprise Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Enterprise Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","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 \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Wave Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"APR Applied Pharma Research","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2020","type":"Not Applicable","leadProduct":"APR-OD031","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"APR Applied Pharma Research","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"APR Applied Pharma Research \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"APR Applied Pharma Research \/ Not Applicable"},{"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":"","sponsorNew":"BrainVectis \/ AskBio","highestDevelopmentStatusID":"4","companyTruncated":"BrainVectis \/ AskBio"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"AskBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Licensing Agreement","leadProduct":"AAV gene therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"University of North Carolina","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"University of North Carolina \/ AskBio","highestDevelopmentStatusID":"4","companyTruncated":"University of North Carolina \/ AskBio"},{"orgOrder":0,"company":"Phoenix Nest","sponsor":"Cure Sanfilippo Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Funding","leadProduct":"AAV-TT","moa":"HGSNAT gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Phoenix Nest","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Phoenix Nest \/ Cure Sanfilippo Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Phoenix Nest \/ Cure Sanfilippo Foundation"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"TANGO antisense oligonucleotides","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Stoke Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Stoke Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Stoke Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"FORCE-M23D","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Dyne Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Dyne Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"PBKR03","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intra-cisterna Magna Injection\n","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"REGENERON","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Agreement","leadProduct":"CRISPR\/Cas9 based gene therapy","moa":"Factor IX gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intellia Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"","sponsorNew":"Intellia Therapeutics \/ REGENERON","highestDevelopmentStatusID":"4","companyTruncated":"Intellia Therapeutics \/ REGENERON"},{"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":"Not Applicable","leadProduct":"FVIII mRNA","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Seattle Children\u2019s Research Institute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Seattle Children\u2019s Research Institute \/ Moderna Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Seattle Children\u2019s Research Institute \/ Moderna Therapeutics"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"VTX-803","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Vivet Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Evox Therapeutics","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Large molecule","year":"2020","type":"Collaboration","leadProduct":"Protein therapeutics","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Evox Therapeutics","amount2":0.88,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.88,"dosageForm":"","sponsorNew":"Evox Therapeutics \/ Takeda","highestDevelopmentStatusID":"4","companyTruncated":"Evox Therapeutics \/ Takeda"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"mRNA-3745","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Moderna Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Moderna Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Hansa Biopharma","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Licensing Agreement","leadProduct":"Imlifidase","moa":"IgG-mediated immune response","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Hansa Biopharma","amount2":0.40999999999999998,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.40999999999999998,"dosageForm":"","sponsorNew":"Hansa Biopharma \/ Sarepta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Hansa Biopharma \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"SparingVision","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"SPVN06","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"SparingVision \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"SparingVision \/ Not Applicable"},{"orgOrder":0,"company":"Erasmus University Medical Center","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Hematopoietic Stem Cell Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Erasmus University Medical Center","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Erasmus University Medical Center \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Erasmus University Medical Center \/ Not Applicable"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"FIX-Gene Therapy","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 \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Catalyst Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Renovion","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ascorbic Acid","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Renovion","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nebulizer","sponsorNew":"Renovion \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Renovion \/ Not Applicable"},{"orgOrder":0,"company":"Triplet Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"TTX-3360","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Triplet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Triplet Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Triplet Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Vita Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"VTA-110","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vita Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Vita Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Vita Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Life Science Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Series A Financing","leadProduct":"Antisense OligoNucleotides","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vico Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"Vico Therapeutics \/ Life Science Partners","highestDevelopmentStatusID":"4","companyTruncated":"Vico Therapeutics \/ Life Science Partners"},{"orgOrder":0,"company":"Wake Forest Institute for Regenerative Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Cell-based gene therapy","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":"","sponsorNew":"Wake Forest Institute for Regenerative Medicine \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Wake Forest Institute for Regenerative Medicine \/ Not Applicable"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Vida Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Financing","leadProduct":"Antibody-oligonucleotide conjugates","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Dyne Therapeutics","amount2":0.12,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"","sponsorNew":"Dyne Therapeutics \/ Vida Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Dyne Therapeutics \/ Vida Ventures"},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AAV-based engineered transgene","moa":"CLN7 gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Neurogene \/ Not Applicable"},{"orgOrder":0,"company":"Foundation for Angelman Syndrome Therapeutics","sponsor":"Lixte Biotechnology","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Collaboration","leadProduct":"LB-100","moa":"Protein phosphatase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Foundation for Angelman Syndrome Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Foundation for Angelman Syndrome Therapeutics \/ Lixte Biotechnology","highestDevelopmentStatusID":"4","companyTruncated":"Foundation for Angelman Syndrome Therapeutics \/ Lixte Biotechnology"},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Gene therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Neurogene \/ Not Applicable"},{"orgOrder":0,"company":"ExCellThera","sponsor":"IRICoR","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Funding","leadProduct":"ECT-001","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ExCellThera","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"ExCellThera \/ IRICoR","highestDevelopmentStatusID":"4","companyTruncated":"ExCellThera \/ IRICoR"},{"orgOrder":0,"company":"Casma Therapeutics","sponsor":"The Column Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Series B Financing","leadProduct":"Undisclosed","moa":"TRPML1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Casma Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"","sponsorNew":"Casma Therapeutics \/ The Column Group","highestDevelopmentStatusID":"4","companyTruncated":"Casma Therapeutics \/ The Column Group"},{"orgOrder":0,"company":"Spirovant Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"SPIRO-2101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Spirovant Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Spirovant Sciences \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Spirovant Sciences \/ Not Applicable"},{"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":"Lentiviral gene therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"The University of Manchester","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"The University of Manchester \/ Avrobio","highestDevelopmentStatusID":"4","companyTruncated":"The University of Manchester \/ Avrobio"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"AskBio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"ImmTOR","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Selecta Biosciences \/ Asklepios BioPharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Selecta Biosciences \/ Asklepios BioPharmaceutical"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Branaplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Versantis","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","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 \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Versantis \/ Not Applicable"},{"orgOrder":0,"company":"Vedere Bio","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Acquisition","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vedere Bio","amount2":0.28000000000000003,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.28000000000000003,"dosageForm":"Intravitreal Injection","sponsorNew":"Vedere Bio \/ Novartis","highestDevelopmentStatusID":"4","companyTruncated":"Vedere Bio \/ Novartis"},{"orgOrder":0,"company":"Axplora","sponsor":"Sensorion","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Agreement","leadProduct":"AAV vectors","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Axplora","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Axplora \/ Sensorion","highestDevelopmentStatusID":"4","companyTruncated":"Axplora \/ Sensorion"},{"orgOrder":0,"company":"reVision Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2020","type":"Not Applicable","leadProduct":"REV-0100","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"reVision Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"reVision Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"reVision Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Catalent Pharma Solutions","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Partnership","leadProduct":"TSHA-118","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Taysha Gene Therapies \/ Catalent","highestDevelopmentStatusID":"4","companyTruncated":"Taysha Gene Therapies \/ Catalent"},{"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":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Sanford Burnham Prebys Medical Discovery Institute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Sanford Burnham Prebys Medical Discovery Institute \/ Soft Bones","highestDevelopmentStatusID":"4","companyTruncated":"Sanford Burnham Prebys Medical Discovery Institute \/ Soft Bones"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AVR-RD-06","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Amylyx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Tauroursodeoxycholic Acid","moa":"Apoptosis","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Amylyx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Amylyx Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Amylyx Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"AskBio","sponsor":"Selecta Biosciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"ImmTOR","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"AskBio \/ Selecta Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"AskBio \/ Selecta Biosciences"},{"orgOrder":0,"company":"Polaryx Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"PLX-300","moa":"PPAR alpha","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Polaryx Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Polaryx Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Polaryx Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"AskBio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"University of North Carolina","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"University of North Carolina \/ Asklepios Biopharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"University of North Carolina \/ Asklepios Biopharmaceutical"},{"orgOrder":0,"company":"Autobahn Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"ABX-002","moa":"TR-Beta","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Autobahn Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Autobahn Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Autobahn Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"TSHA-103","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Inclacumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Global Blood Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Global Blood Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Emendo Biotherapeutics","sponsor":"AnGes","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Acquisition","leadProduct":"EMD-101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Emendo Biotherapeutics","amount2":0.25,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.25,"dosageForm":"","sponsorNew":"Emendo Biotherapeutics \/ AnGes","highestDevelopmentStatusID":"4","companyTruncated":"Emendo Biotherapeutics \/ AnGes"},{"orgOrder":0,"company":"Polaryx Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"PLX-300","moa":"PPAR alpha","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Polaryx Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Polaryx Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Polaryx Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"VECT-HORUS","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Series D Financing","leadProduct":"Nucleic acid based therapeutics","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"VECT-HORUS","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"","sponsorNew":"VECT-HORUS \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"VECT-HORUS \/ Undisclosed"},{"orgOrder":0,"company":"Vera Therapeutics","sponsor":"NeuBase Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Acquisition","leadProduct":"NT0200","moa":"DMPK protein degradation","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vera Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Vera Therapeutics \/ NeuBase Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Vera Therapeutics \/ NeuBase Therapeutics"},{"orgOrder":0,"company":"Quellis Biosciences Inc","sponsor":"Catabasis Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","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":"","sponsorNew":"Quellis Biosciences Inc \/ Catabasis Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Quellis Biosciences Inc \/ Catabasis Pharmaceuticals"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"VMCO-I","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Nanoscope Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Nanoscope Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Polaryx Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"PLX-300","moa":"TFEB","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Polaryx Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Polaryx Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Polaryx Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"M6P Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Not Applicable","leadProduct":"M011","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"M6P Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"M6P Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"M6P Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"JAG101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jaguar Gene Therapy","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Jaguar Gene Therapy \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Jaguar Gene Therapy \/ Not Applicable"},{"orgOrder":0,"company":"SalioGen Therapeutics","sponsor":"PBM Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Series A Financing","leadProduct":"Non-viral gene therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SalioGen Therapeutics","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"","sponsorNew":"SalioGen Therapeutics \/ PBM Capital","highestDevelopmentStatusID":"4","companyTruncated":"SalioGen Therapeutics \/ PBM Capital"},{"orgOrder":0,"company":"Actelion Pharmaceuticals Ltd","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Collaboration","leadProduct":"Miglustat","moa":"GCS","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Actelion Pharmaceuticals Ltd","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Actelion Pharmaceuticals Ltd \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Actelion Pharmaceuticals Ltd \/ Beyond Batten Disease Foundation"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Lipid Nanoparticles","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Intellia Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Intellia Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Evox Therapeutics","sponsor":"Redmile Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Series C Financing","leadProduct":"EVX-101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Evox Therapeutics","amount2":0.10000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"","sponsorNew":"Evox Therapeutics \/ Redmile Group","highestDevelopmentStatusID":"4","companyTruncated":"Evox Therapeutics \/ Redmile Group"},{"orgOrder":0,"company":"Anima Medical Centre","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Huntingtin protein","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Anima Medical Centre","amount2":2.4199999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":2.4199999999999999,"dosageForm":"","sponsorNew":"Anima Medical Centre \/ Takeda","highestDevelopmentStatusID":"4","companyTruncated":"Anima Medical Centre \/ Takeda"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Wellington Management Company","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Series B Financing","leadProduct":"Oligonucleotide antibody","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Entrada Therapeutics","amount2":0.12,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"","sponsorNew":"Entrada Therapeutics \/ Wellington Management Company","highestDevelopmentStatusID":"4","companyTruncated":"Entrada Therapeutics \/ Wellington Management Company"},{"orgOrder":0,"company":"Biom Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Not Applicable","leadProduct":"BIO017","moa":"TRPV1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Biom Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Biom Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Biom Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Series B Financing","leadProduct":"JAG101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jaguar Gene Therapy","amount2":0.14000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14000000000000001,"dosageForm":"","sponsorNew":"Jaguar Gene Therapy \/ Eli Lilly","highestDevelopmentStatusID":"4","companyTruncated":"Jaguar Gene Therapy \/ Eli Lilly"},{"orgOrder":0,"company":"Denovo Biopharma","sponsor":"Aytu BioScience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"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 BioPharma","highestDevelopmentStatusID":"4","companyTruncated":"Denovo Biopharma \/ Aytu BioPharma"},{"orgOrder":0,"company":"Rumpus Therapeutics","sponsor":"Aytu BioScience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"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 BioPharma","highestDevelopmentStatusID":"4","companyTruncated":"Rumpus Therapeutics \/ Aytu BioPharma"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Mirum Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"VTX-803","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Vivet Therapeutics \/ Mirum Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ Mirum Pharmaceuticals"},{"orgOrder":0,"company":"GAMUT Therapeutics","sponsor":"SparingVision","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Acquisition","leadProduct":"SPVN20","moa":"","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":"Catalent Pharma Solutions","sponsor":"AavantiBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Partnership","leadProduct":"AAV-based gene therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Catalent Pharma Solutions","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Catalent Pharma Solutions \/ AavantiBio","highestDevelopmentStatusID":"4","companyTruncated":"Catalent Pharma Solutions \/ AavantiBio"},{"orgOrder":0,"company":"Autobahn Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"ABX-002","moa":"TR-Beta","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Autobahn Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Autobahn Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Autobahn Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Affiris","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"mAB C6-17","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Affiris","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Affiris \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Affiris \/ Not Applicable"},{"orgOrder":0,"company":"NephroDI Therapeutics","sponsor":"Xontogeny, LLC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"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"},{"orgOrder":0,"company":"NeuBase Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"NT0200","moa":"DMPK protein","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NeuBase Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"NeuBase Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"NeuBase Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"RNA-based therapy","moa":"CFTR gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"GenScript ProBio","sponsor":"Neoletix","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Collaboration","leadProduct":"Recombinant Human Coagulation Factor VIII","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"GenScript ProBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"GenScript ProBio \/ Neoletix","highestDevelopmentStatusID":"4","companyTruncated":"GenScript ProBio \/ Neoletix"},{"orgOrder":0,"company":"Resilience","sponsor":"AavantiBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Resilience","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Resilience \/ AavantiBio","highestDevelopmentStatusID":"4","companyTruncated":"Resilience \/ AavantiBio"},{"orgOrder":0,"company":"SATT Conectus","sponsor":"Lysogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","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":"","sponsorNew":"SATT Conectus \/ Lysogene","highestDevelopmentStatusID":"4","companyTruncated":"SATT Conectus \/ Lysogene"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"RTW Investments","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Funding","leadProduct":"ACTX-101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Alcyone Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Alcyone Therapeutics \/ RTW Investments","highestDevelopmentStatusID":"4","companyTruncated":"Alcyone Therapeutics \/ RTW Investments"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"RCT223","moa":"CFTR gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"SpliSense","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"SPL23-ASO","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SpliSense","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"SpliSense \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"SpliSense \/ Not Applicable"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Dyne Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Dyne Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"GLM101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Glycomine \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Glycomine \/ Not Applicable"},{"orgOrder":0,"company":"Usher III Initiative","sponsor":"Foundation Fighting Blindness","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Funding","leadProduct":"BF844","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Usher III Initiative","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Usher III Initiative \/ Foundation Fighting Blindness","highestDevelopmentStatusID":"4","companyTruncated":"Usher III Initiative \/ Foundation Fighting Blindness"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Pharming","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"Autologous Hematopoietic Stem Cell Gene Therapy","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Orchard Therapeutics","amount2":0.20999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20999999999999999,"dosageForm":"","sponsorNew":"Orchard Therapeutics \/ Pharming Group","highestDevelopmentStatusID":"4","companyTruncated":"Orchard Therapeutics \/ Pharming Group"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"QLS-215","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Catabasis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Catabasis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Catabasis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"University of Massachusetts","sponsor":"ASC Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Partnership","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"University of Massachusetts","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"University of Massachusetts \/ ASC Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"University of Massachusetts \/ ASC Therapeutics"},{"orgOrder":0,"company":"Aldevron","sponsor":"AavantiBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Partnership","leadProduct":"Gene Therapy","moa":"","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":"Guide Therapeutics","sponsor":"Beam Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Acquisition","leadProduct":"Undisclosed","moa":"","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":"Lixte Biotechnology","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"LB-100","moa":"PP2A","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Lixte Biotechnology","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Lixte Biotechnology \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Lixte Biotechnology \/ Not Applicable"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"ImmTOR","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Selecta Biosciences \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Selecta Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Vico Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Vico Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Vico Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"M6P Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"M041","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"M6P Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"M6P Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"M6P Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Abigail Wexner Research Institute at Nationwide Children\u2019s Hospital","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":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Abigail Wexner Research Institute at Nationwide Children\u2019s Hospital","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Abigail Wexner Research Institute at Nationwide Children\u2019s Hospital \/ Sarepta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Abigail Wexner Research Institute at Nationwide Children\u2019s Hospital \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"SLC6A19","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jnana Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"","sponsorNew":"Jnana Therapeutics \/ RA Capital Management","highestDevelopmentStatusID":"4","companyTruncated":"Jnana Therapeutics \/ RA Capital Management"},{"orgOrder":0,"company":"McQuade Center for Strategic Research and Development","sponsor":"Sentinel Oncology","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Collaboration","leadProduct":"SOL784","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"McQuade Center for Strategic Research and Development","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","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":"The Texas A&M University System","sponsor":"Engrail Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Elesclomol","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"The Texas A&M University System","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"The Texas A&M University System \/ Engrail Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"The Texas A&M University System \/ Engrail Therapeutics"},{"orgOrder":0,"company":"Versantis","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"VS-01","moa":"Ammonia clearance","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Versantis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Versantis \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Versantis \/ Not Applicable"},{"orgOrder":0,"company":"Addex Therapeutics","sponsor":"The Charcot\u2013Marie\u2013Tooth Association","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","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 \/ The Charcot\u2013Marie\u2013Tooth Association","highestDevelopmentStatusID":"4","companyTruncated":"Addex Therapeutics \/ The Charcot\u2013Marie\u2013Tooth Association"},{"orgOrder":0,"company":"Oak Bay Biosciences","sponsor":"FrontFundr","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Funding","leadProduct":"Undisclosed","moa":"Complement","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oak Bay Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Oak Bay Biosciences \/ FrontFundr","highestDevelopmentStatusID":"4","companyTruncated":"Oak Bay Biosciences \/ FrontFundr"},{"orgOrder":0,"company":"Nabriva Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Lefamulin Acetate","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Nabriva Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Nabriva Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Nabriva Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Rectify Pharma","sponsor":"Atlas Venture","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Positive functional","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rectify Pharma","amount2":0.10000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"","sponsorNew":"Rectify Pharma \/ Atlas Venture","highestDevelopmentStatusID":"4","companyTruncated":"Rectify Pharma \/ Atlas Venture"},{"orgOrder":0,"company":"Poseida Therapeutics, Inc","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"P-FVIII-101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Poseida Therapeutics, Inc","amount2":3.6000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":3.6000000000000001,"dosageForm":"","sponsorNew":"Poseida Therapeutics, Inc \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Poseida Therapeutics, Inc \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"ImmTOR","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Selecta Biosciences \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Selecta Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Comet Therapeutics","sponsor":"VectivBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Acquisition","leadProduct":"VB-1197","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Comet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Comet Therapeutics \/ VectivBio Holding","highestDevelopmentStatusID":"4","companyTruncated":"Comet Therapeutics \/ VectivBio Holding"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Pfizer Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Series B Financing","leadProduct":"LNP-based RNA Therapy","moa":"CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"ReCode Therapeutics \/ Pfizer Ventures","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Pfizer Ventures"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"mRNA-based antibody","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"NTLA-3001","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intellia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Intellia Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Intellia Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Massachusetts Eye and Ear and Harvard Medical School","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Agreement","leadProduct":"OPGx-003","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Dosage Strength\/Form","sponsorNew":"Opus Genetics \/ Massachusetts Eye and Ear and Harvard Medical School","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ Massachusetts Eye and Ear and Harvard Medical School"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Avrobio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Collaboration","leadProduct":"Humanized Anti-CD117 Monoclonal Antibody","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":"Spirovant Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"SP-101","moa":"CFTR","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Spirovant Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Spirovant Sciences \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Spirovant Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Nova Mentis Life Science","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Nova Mentis Life Science","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Nova Mentis Life Science \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Nova Mentis Life Science \/ Not Applicable"},{"orgOrder":0,"company":"Codexis","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"CDX-6512","moa":"Enzyme Replacement","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Codexis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Codexis \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Codexis \/ Not Applicable"},{"orgOrder":0,"company":"Variant","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"VAR002","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Variant","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Variant \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Variant \/ Not Applicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"mRNA therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"ATA-100","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Atamyo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Atamyo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Altasciences Company Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Oxypurinol","moa":"Xanthine oxidase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Xortx Therapeutics \/ Altasciences","highestDevelopmentStatusID":"4","companyTruncated":"Xortx Therapeutics \/ Altasciences"},{"orgOrder":0,"company":"Generation Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Generation Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Generation Bio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Generation Bio \/ Not Applicable"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"Lonza Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Agreement","leadProduct":"Anc80L65","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Affinia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Lumbar Puncture","sponsorNew":"Affinia Therapeutics \/ Lonza Group","highestDevelopmentStatusID":"4","companyTruncated":"Affinia Therapeutics \/ Lonza Group"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"4BIO Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Financing","leadProduct":"Ray-001","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ray Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Intravitreal Injection","sponsorNew":"Ray Therapeutics \/ 4BIO Capital","highestDevelopmentStatusID":"4","companyTruncated":"Ray Therapeutics \/ 4BIO Capital"},{"orgOrder":0,"company":"SalioGen Therapeutics","sponsor":"GordonMD Global","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Series B Financing","leadProduct":"Genetic Medicine","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SalioGen Therapeutics","amount2":0.12,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"","sponsorNew":"SalioGen Therapeutics \/ GordonMD Global","highestDevelopmentStatusID":"4","companyTruncated":"SalioGen Therapeutics \/ GordonMD Global"},{"orgOrder":0,"company":"Warden Bio","sponsor":"Kriya Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Acquisition","leadProduct":"AAV-mediated Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Warden Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Warden Bio \/ Kriya Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Warden Bio \/ Kriya Therapeutics"},{"orgOrder":0,"company":"Quris","sponsor":"Welltech Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2022","type":"Financing","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Quris","amount2":0.029999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"Quris \/ Welltech Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Quris \/ Welltech Ventures"},{"orgOrder":0,"company":"M6P Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"M041","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"M6P Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"M6P Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"M6P Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Erytech Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Red Blood Cell-encapsulated Arginine Deiminase","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Erytech Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Erytech Pharma \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Erytech Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Gain Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"M6P Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"M011","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"M6P Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"M6P Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"M6P Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Anakuria","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"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":"Inhalation","sponsorNew":"Anakuria \/ Janssen","highestDevelopmentStatusID":"4","companyTruncated":"Anakuria \/ Janssen"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","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":"","sponsorNew":"Ionis Pharmaceuticals \/ Biogen","highestDevelopmentStatusID":"4","companyTruncated":"Ionis Pharmaceuticals \/ Biogen"},{"orgOrder":0,"company":"SEAL Therapeutics","sponsor":"Santhera Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","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":"","sponsorNew":"SEAL Therapeutics \/ Santhera","highestDevelopmentStatusID":"4","companyTruncated":"SEAL Therapeutics \/ Santhera"},{"orgOrder":0,"company":"NeuBase Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"NT-0200","moa":"mRNA splicing","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NeuBase Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"NeuBase Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"NeuBase Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"Forge Biologics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Partnership","leadProduct":"Ray-001","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ray Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal","sponsorNew":"Ray Therapeutics \/ Forge Biologics","highestDevelopmentStatusID":"4","companyTruncated":"Ray Therapeutics \/ Forge Biologics"},{"orgOrder":0,"company":"Tonix Pharmaceuticals Holding Corp","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2022","type":"Not Applicable","leadProduct":"Oxytocin","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Tonix Pharmaceuticals Holding Corp","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intranasal","sponsorNew":"Tonix Pharmaceuticals Holding Corp \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Tonix Pharmaceuticals Holding Corp \/ Not Applicable"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Wave Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Wave Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"NeuBase Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"NT-0231.F","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NeuBase Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"NeuBase Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"NeuBase Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Kither Biotech","sponsor":"Claris Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2022","type":"Series B Financing","leadProduct":"KIT2014","moa":"cAMP","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Kither Biotech","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Inhalation","sponsorNew":"Kither Biotech \/ Claris Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Kither Biotech \/ Claris Ventures"},{"orgOrder":0,"company":"Partner Therapeutics","sponsor":"University of Colorado","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Sargramostim","moa":"GM-CSF","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Partner Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Partner Therapeutics \/ University of Colorado","highestDevelopmentStatusID":"4","companyTruncated":"Partner Therapeutics \/ University of Colorado"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Gain Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"ViGeneron","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"VG801","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ViGeneron","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"ViGeneron \/ Regeneron","highestDevelopmentStatusID":"4","companyTruncated":"ViGeneron \/ Regeneron"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"RNA-based Therapeutic","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Erytech Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"RBC-loaded Enzyme","moa":"Enzyme","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Erytech Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Erytech Pharma \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Erytech Pharma \/ Not Applicable"},{"orgOrder":0,"company":"CalciMedica","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","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 \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"CalciMedica \/ Not Applicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"FXS01","moa":"Diacylglycerol kinase kappa","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Lysogene \/ Not Applicable"},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Funding","leadProduct":"Ray-001","moa":"","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":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"OPGx-002","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ Not Applicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Collaboration","leadProduct":"AAV9-mediated Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"CANbridge Pharmaceuticals \/ UMass Chan Medical School","highestDevelopmentStatusID":"4","companyTruncated":"CANbridge Pharmaceuticals \/ UMass Chan Medical School"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"Kennedy Krieger Institute","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Collaboration","leadProduct":"Vafidemstat","moa":"LSD1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Oryzon Genomics \/ Kennedy Krieger Institute","highestDevelopmentStatusID":"4","companyTruncated":"Oryzon Genomics \/ Kennedy Krieger Institute"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"EDIT-103","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Editas Medicine \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Editas Medicine \/ Not Applicable"},{"orgOrder":0,"company":"NeuBase Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"NT-0231.F","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NeuBase Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"NeuBase Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"NeuBase Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"EDIT-103","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Editas Medicine \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Editas Medicine \/ Not Applicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAV9-Based Gene Therapy","moa":"SMN1 gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"CANbridge Pharmaceuticals \/ UMass Chan Medical School","highestDevelopmentStatusID":"4","companyTruncated":"CANbridge Pharmaceuticals \/ UMass Chan Medical School"},{"orgOrder":0,"company":"Otonomy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"OTO-825","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Otonomy","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Otonomy \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Otonomy \/ Not Applicable"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"GT-02329","moa":"GCase level","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Gain Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"SOM Biotech","sponsor":"University of Minnesota","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SOM Biotech","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"SOM Biotech \/ University of Minnesota","highestDevelopmentStatusID":"4","companyTruncated":"SOM Biotech \/ University of Minnesota"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"LNP-encapsulated mRNA Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Aerosol For Inhalation","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAV9-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"CANbridge Pharmaceuticals \/ UMass Chan Medical School","highestDevelopmentStatusID":"4","companyTruncated":"CANbridge Pharmaceuticals \/ UMass Chan Medical School"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"JAG101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jaguar Gene Therapy","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Jaguar Gene Therapy \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Jaguar Gene Therapy \/ Not Applicable"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"GPH102","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Graphite Bio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Graphite Bio \/ Not Applicable"},{"orgOrder":0,"company":"Codexis","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"CDX-6316","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Codexis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Codexis \/ Takeda","highestDevelopmentStatusID":"4","companyTruncated":"Codexis \/ Takeda"},{"orgOrder":0,"company":"Capsida","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"CAP-001","moa":"IDS","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Capsida","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Capsida \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Capsida \/ Not Applicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"LNP-based mRNA Therapeutics","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","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":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Code Bio","sponsor":"Northpond Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Series A Financing","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Code Bio","amount2":0.080000000000000002,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"Code Bio \/ Northpond Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Code Bio \/ Northpond Ventures"},{"orgOrder":0,"company":"Ashvattha Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Hydroxyl Dendrimer Based Therapeutics","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Ashvattha Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ashvattha Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Ashvattha Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Lenire Biosciences","sponsor":"University College London","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"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":"","sponsorNew":"Lenire Biosciences \/ University College London","highestDevelopmentStatusID":"4","companyTruncated":"Lenire Biosciences \/ University College London"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"FRAXA Research Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","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":"Liquid","sponsorNew":"Anavex Life Sciences \/ FRAXA Research Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Anavex Life Sciences \/ FRAXA Research Foundation"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"CY6463","moa":"sGC","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cyclerion Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cyclerion Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Cyclerion Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"CMT Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"HDAC6","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Oryzon Genomics \/ CMT Research Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Oryzon Genomics \/ CMT Research Foundation"},{"orgOrder":0,"company":"Oxford Biomedica","sponsor":"Orchard Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"OTL-203","moa":"IDUA gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oxford Biomedica","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Oxford Biomedica \/ Orchard Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Oxford Biomedica \/ Orchard Therapeutics"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"HMI-204","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Homology Medicines \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Homology Medicines \/ Not Applicable"},{"orgOrder":0,"company":"Verve Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"VERVE-201","moa":"ANGPTL3 gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Verve Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Verve Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Verve Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"BV-101","moa":"CYP46A1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"AskBio \/ Bayer","highestDevelopmentStatusID":"4","companyTruncated":"AskBio \/ Bayer"},{"orgOrder":0,"company":"Attralus","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"AT-02","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Attralus \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Attralus \/ Not Applicable"},{"orgOrder":0,"company":"iECURE","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"GTP-506A","moa":"PCSK9 gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"iECURE \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"iECURE \/ Not Applicable"},{"orgOrder":0,"company":"VectorY","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAV Vector","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"VectorY","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"VectorY \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"VectorY \/ Not Applicable"},{"orgOrder":0,"company":"Castle Creek Biosciences","sponsor":"Paragon Biosciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"LV-FAH","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Castle Creek Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Castle Creek Biosciences \/ Paragon Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"Castle Creek Biosciences \/ Paragon Biosciences"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Cure AP-4","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Charles River Laboratories, Inc \/ Cure AP-4","highestDevelopmentStatusID":"4","companyTruncated":"Charles River Laboratories, Inc \/ Cure AP-4"},{"orgOrder":0,"company":"iECURE","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"GTP-506A","moa":"PCSK9 gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"iECURE","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"iECURE \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"iECURE \/ Not Applicable"},{"orgOrder":0,"company":"Attralus","sponsor":"University of Tennessee Graduate School of Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"AT-04","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Attralus \/ University of Tennessee Graduate School of Medicine","highestDevelopmentStatusID":"4","companyTruncated":"Attralus \/ University of Tennessee Graduate School of Medicine"},{"orgOrder":0,"company":"Attralus","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"AT-02","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Attralus \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Attralus \/ Not Applicable"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Vaccine","year":"2022","type":"Not Applicable","leadProduct":"mRNA-3139","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Moderna Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Moderna Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AmideBio","sponsor":"NIDDK","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2022","type":"Funding","leadProduct":"Glucagon","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AmideBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AmideBio \/ NIDDK","highestDevelopmentStatusID":"4","companyTruncated":"AmideBio \/ NIDDK"},{"orgOrder":0,"company":"Cellectis","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"IDUA gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cellectis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Cellectis \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Cellectis \/ Not Applicable"},{"orgOrder":0,"company":"Chameleon Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"AAV8 IgG","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Chameleon Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Chameleon Biosciences \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Chameleon Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"EDIT-103","moa":"AAV5 vectors gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Editas Medicine \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Editas Medicine \/ Not Applicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAV9-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"CANbridge Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"CANbridge Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAV-SLB101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Solid Biosciences \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Solid Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Oregon State University","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"mRNA Loaded Lipid Nanoparticle","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oregon State University","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Oregon State University \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Oregon State University \/ Not Applicable"},{"orgOrder":0,"company":"Tavanta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"TAVT-135","moa":"Chloride ion","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Tavanta Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Tavanta Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Tavanta Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Hansa Biopharma","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","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":"Infusion","sponsorNew":"Hansa Biopharma \/ Sarepta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Hansa Biopharma \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Protagonist Therapeutics","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2022","type":"Not Applicable","leadProduct":"Rusfertide Analog","moa":"Hepcidin","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Protagonist Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Protagonist Therapeutics \/ Janssen","highestDevelopmentStatusID":"4","companyTruncated":"Protagonist Therapeutics \/ Janssen"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"EXG-001","moa":"ZSCAN4 gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Elixirgen Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"PGN-EDO53","moa":"Exon 53 skipping oligonucleotide","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"PepGen \/ Not Applicable"},{"orgOrder":0,"company":"Tenaya Therapeutics","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"TN-201","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Tenaya Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"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":"","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"TN-201","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Tenaya Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"Tenaya Therapeutics \/ Morgan Stanley","highestDevelopmentStatusID":"4","companyTruncated":"Tenaya Therapeutics \/ Morgan Stanley"},{"orgOrder":0,"company":"Vyant Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vyant Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Vyant Bio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Vyant Bio \/ Not Applicable"},{"orgOrder":0,"company":"AavantiBio","sponsor":"Solid Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Merger","leadProduct":"AVB-202","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AavantiBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"AavantiBio \/ Solid Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"AavantiBio \/ Solid Biosciences"},{"orgOrder":0,"company":"AavantiBio","sponsor":"Solid Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Merger","leadProduct":"AVB-202","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AavantiBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"AavantiBio \/ Solid Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"AavantiBio \/ Solid Biosciences"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Collaboration","leadProduct":"EEV-conjugated Phosphorodiamidate Morpholino Oligomer","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Entrada Therapeutics","amount2":0.73999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.73999999999999999,"dosageForm":"","sponsorNew":"Entrada Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Entrada Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"IVERIC bio","sponsor":"Opus Genetics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Acquisition","leadProduct":"AAv-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"IVERIC bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"IVERIC bio \/ Opus Genetics","highestDevelopmentStatusID":"4","companyTruncated":"IVERIC bio \/ Opus Genetics"},{"orgOrder":0,"company":"Cellectis","sponsor":"Primera Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Collaboration","leadProduct":"mtDNA-based Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cellectis","amount2":0.75,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.75,"dosageForm":"","sponsorNew":"Cellectis \/ Primera Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Cellectis \/ Primera Therapeutics"},{"orgOrder":0,"company":"Metagenomi","sponsor":"Novo Hold\u00adings","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Series B Financing","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0.28000000000000003,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.28000000000000003,"dosageForm":"","sponsorNew":"Metagenomi \/ Novo Hold\u00adings","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ Novo Hold\u00adings"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Engineered Stem Cell Antibody Paired Evasion","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Beam Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Beam Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Licensing Agreement","leadProduct":"AT845","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Selecta Biosciences","amount2":0.34999999999999998,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.34999999999999998,"dosageForm":"","sponsorNew":"Selecta Biosciences \/ Astellas Pharma","highestDevelopmentStatusID":"4","companyTruncated":"Selecta Biosciences \/ Astellas Pharma"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"The Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Funding","leadProduct":"mRNA-based Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Aerosol (PMDI)","sponsorNew":"ReCode Therapeutics \/ The Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ The Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"ENTR-601-45","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Entrada Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Entrada Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Herophilus","sponsor":"Rett Syndrome Research Trust","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Funding","leadProduct":"HRP-12975","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Herophilus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Herophilus \/ Rett Syndrome Research Trust","highestDevelopmentStatusID":"4","companyTruncated":"Herophilus \/ Rett Syndrome Research Trust"},{"orgOrder":0,"company":"INmune Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"INmune Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"INmune Bio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"INmune Bio \/ Not Applicable"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Acadia Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2022","type":"Collaboration","leadProduct":"RNA-based Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Stoke Therapeutics","amount2":0.96999999999999997,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.96999999999999997,"dosageForm":"","sponsorNew":"Stoke Therapeutics \/ Acadia Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Stoke Therapeutics \/ Acadia Pharmaceuticals"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Agreement","leadProduct":"EEV-conjugated Phosphorodiamidate Morpholino Oligomer","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Entrada Therapeutics","amount2":0.73999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.73999999999999999,"dosageForm":"","sponsorNew":"Entrada Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Entrada Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Unravel Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"RVL001","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Unravel Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Unravel Biosciences \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Unravel Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"IPS Heart","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"GIVI-MPC","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"IPS Heart","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"IPS Heart \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"IPS Heart \/ Not Applicable"},{"orgOrder":0,"company":"Xinvento","sponsor":"Rhythm Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2023","type":"Acquisition","leadProduct":"Setmelanotide Acetate","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Xinvento","amount2":0.20999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20999999999999999,"dosageForm":"Injectable\/Injection","sponsorNew":"Xinvento \/ Rhythm Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Xinvento \/ Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"Nanite","sponsor":"CF Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Funding","leadProduct":"m-RNA Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Nanite","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Nanite \/ CF Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Nanite \/ CF Foundation"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"ORY-4001","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Oryzon Genomics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Oryzon Genomics \/ Not Applicable"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"ABO-504","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Abeona Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Abeona Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Intergalactic Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"IG-002","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intergalactic Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Intergalactic Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Intergalactic Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Eurostars","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Funding","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Gain Therapeutics \/ Eurostars","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Eurostars"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Zagociguat","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cyclerion Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Cyclerion Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Cyclerion Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Anagram Therapeutics","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Funding","leadProduct":"Enzyme-based Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Anagram Therapeutics","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Tablet","sponsorNew":"Anagram Therapeutics \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Anagram Therapeutics \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Peter Hecht","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Agreement","leadProduct":"Zagociguat","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cyclerion Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"","sponsorNew":"Cyclerion Therapeutics \/ Peter Hecht","highestDevelopmentStatusID":"4","companyTruncated":"Cyclerion Therapeutics \/ Peter Hecht"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"PCSK9-specific ARCUS Nuclease Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Precision BioSciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Precision BioSciences \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Precision BioSciences \/ Not Applicable"},{"orgOrder":0,"company":"Rumi Scientific","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rumi Scientific","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Rumi Scientific \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Rumi Scientific \/ Not Applicable"},{"orgOrder":0,"company":"Amplo Biotechnology","sponsor":"NIH-NIAMS","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Funding","leadProduct":"AAV-ColQ-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Amplo Biotechnology","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Amplo Biotechnology \/ NIH-NIAMS","highestDevelopmentStatusID":"4","companyTruncated":"Amplo Biotechnology \/ NIH-NIAMS"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"VCA-894A","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vanda Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Vanda Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Vanda Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Kate Therapeutics","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Licensing Agreement","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Kate Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Kate Therapeutics \/ Astellas Pharma","highestDevelopmentStatusID":"4","companyTruncated":"Kate Therapeutics \/ Astellas Pharma"},{"orgOrder":0,"company":"IPS Heart","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"ISX9-CPC","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"IPS Heart","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"IPS Heart \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"IPS Heart \/ Not Applicable"},{"orgOrder":0,"company":"SparingVision","sponsor":"Tenpoint Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Divestment","leadProduct":"SPVN20","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"SparingVision \/ Tenpoint Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"SparingVision \/ Tenpoint Therapeutics"},{"orgOrder":0,"company":"Solve GNE","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"GA-002","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Solve GNE","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Solve GNE \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Solve GNE \/ Undisclosed"},{"orgOrder":0,"company":"Can-Fite BioPharma","sponsor":"Fondazione Telethon","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Agreement","leadProduct":"Piclidenoson","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Can-Fite BioPharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Can-Fite BioPharma \/ Fondazione Telethon","highestDevelopmentStatusID":"4","companyTruncated":"Can-Fite BioPharma \/ Fondazione Telethon"},{"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":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Elastin Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Elastin Biosciences \/ Longaevus Technologies","highestDevelopmentStatusID":"4","companyTruncated":"Elastin Biosciences \/ Longaevus Technologies"},{"orgOrder":0,"company":"Actio Biosciences","sponsor":"Canaan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Series A Financing","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Actio Biosciences","amount2":0.059999999999999998,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"","sponsorNew":"Actio Biosciences \/ Canaan","highestDevelopmentStatusID":"4","companyTruncated":"Actio Biosciences \/ Canaan"},{"orgOrder":0,"company":"AlveoGene","sponsor":"Oxford Science Enterprises","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"AVG-001","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AlveoGene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"AlveoGene \/ Oxford Science Enterprises","highestDevelopmentStatusID":"4","companyTruncated":"AlveoGene \/ Oxford Science Enterprises"},{"orgOrder":0,"company":"Axovia Therapeutics","sponsor":"ALSA Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Acquisition","leadProduct":"AXV101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Axovia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Axovia Therapeutics \/ ALSA Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Axovia Therapeutics \/ ALSA Ventures"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Tideglusib","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AMO Pharma \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"AMO Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Capsida","sponsor":"Kate Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Capsida","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Capsida \/ Kate Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Capsida \/ Kate Therapeutics"},{"orgOrder":0,"company":"Rejuvenate Biomed","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"RJB-0402","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rejuvenate Biomed","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Rejuvenate Biomed \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Rejuvenate Biomed \/ Not Applicable"},{"orgOrder":0,"company":"Rezolute","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"RZ358","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Rezolute \/ Not Applicable"},{"orgOrder":0,"company":"Generation Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"iqDNA","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Generation Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Generation Bio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Generation Bio \/ Not Applicable"},{"orgOrder":0,"company":"Harness Therapeutics","sponsor":"Epidarex Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Harness Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"","sponsorNew":"Harness Therapeutics \/ Epidarex Capital","highestDevelopmentStatusID":"4","companyTruncated":"Harness Therapeutics \/ Epidarex Capital"},{"orgOrder":0,"company":"MitoRx Therapeutics","sponsor":"Wren Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"MitoRx Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"","sponsorNew":"MitoRx Therapeutics \/ Wren Capital","highestDevelopmentStatusID":"4","companyTruncated":"MitoRx Therapeutics \/ Wren Capital"},{"orgOrder":0,"company":"Edity Therapeutics","sponsor":"Dr. Reddy's Laboratories","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Edity Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Edity Therapeutics \/ Dr. Reddy's Laboratories","highestDevelopmentStatusID":"4","companyTruncated":"Edity Therapeutics \/ Dr. Reddy's Laboratories"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Collaboration","leadProduct":"Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Voyager Therapeutics, Inc","amount2":1.3,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":1.3,"dosageForm":"","sponsorNew":"Voyager Therapeutics, Inc \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"4","companyTruncated":"Voyager Therapeutics, Inc \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Andelyn Biosciences","sponsor":"Armatus Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Partnership","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Andelyn Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Andelyn Biosciences \/ Armatus Bio","highestDevelopmentStatusID":"4","companyTruncated":"Andelyn Biosciences \/ Armatus Bio"},{"orgOrder":0,"company":"Sail Biomedicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"eRNA-based Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Sail Biomedicines","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Sail Biomedicines \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Sail Biomedicines \/ Not Applicable"},{"orgOrder":0,"company":"Fujifilm Cellular Dynamics","sponsor":"BlueRock Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Licensing Agreement","leadProduct":"OpCT-001","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Fujifilm Cellular Dynamics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Fujifilm Cellular Dynamics \/ BlueRock Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Fujifilm Cellular Dynamics \/ BlueRock Therapeutics"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Novel Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Recombinant Human Heparan N-sulfatase","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"GC Biopharma \/ Novel Pharma","highestDevelopmentStatusID":"4","companyTruncated":"GC Biopharma \/ Novel Pharma"},{"orgOrder":0,"company":"Prime Medicine","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Prime Medicine","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"","sponsorNew":"Prime Medicine \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Prime Medicine \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"KRRO-110","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Korro Bio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Korro Bio \/ Not Applicable"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"French Government","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"VTX-806","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"","sponsorNew":"Vivet Therapeutics \/ French Government","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ French Government"},{"orgOrder":0,"company":"Metagenomi","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Public Offering","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0.089999999999999997,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"","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":"","productType":"Cell and Gene therapy","year":"2024","type":"Public Offering","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0.089999999999999997,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"","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":"","productType":"Cell and Gene therapy","year":"2024","type":"Public Offering","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0.089999999999999997,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"","sponsorNew":"Metagenomi \/ J.P. Morgan","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ J.P. Morgan"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Neurocrine Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Collaboration","leadProduct":"VY-FXN01","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Voyager Therapeutics, Inc","amount2":1.8700000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":1.8700000000000001,"dosageForm":"Injection","sponsorNew":"Voyager Therapeutics, Inc \/ Neurocrine Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"Voyager Therapeutics, Inc \/ Neurocrine Biosciences"},{"orgOrder":0,"company":"Cyprium Therapeutics","sponsor":"National Institute of Neurological Disorders and Stroke","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"AAV-ATP7A","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cyprium Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Cyprium Therapeutics \/ National Institute of Neurological Disorders and Stroke","highestDevelopmentStatusID":"4","companyTruncated":"Cyprium Therapeutics \/ National Institute of Neurological Disorders and Stroke"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Termination","leadProduct":"CRISPR-based Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intellia Therapeutics","amount2":0.41999999999999998,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.41999999999999998,"dosageForm":"","sponsorNew":"Intellia Therapeutics \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Intellia Therapeutics \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Genflow Biosciences","sponsor":"EXO Biologics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Collaboration","leadProduct":"GF-3001","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Genflow Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Genflow Biosciences \/ EXO Biologics","highestDevelopmentStatusID":"4","companyTruncated":"Genflow Biosciences \/ EXO Biologics"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"GT-00513","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Gain Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Gain Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Gain Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"BE-101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Be Biopharma \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Be Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"MaxCyte","sponsor":"Be Biopharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Agreement","leadProduct":"B Cell-based Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"MaxCyte","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"MaxCyte \/ Be Biopharma","highestDevelopmentStatusID":"4","companyTruncated":"MaxCyte \/ Be Biopharma"},{"orgOrder":0,"company":"Diagonal Therapeutics","sponsor":"BVF Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Financing","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Diagonal Therapeutics","amount2":0.13,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"","sponsorNew":"Diagonal Therapeutics \/ BVF Partners","highestDevelopmentStatusID":"4","companyTruncated":"Diagonal Therapeutics \/ BVF Partners"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"KRRO-110","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Korro Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Korro Bio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Korro Bio \/ Not Applicable"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Axovia Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Collaboration","leadProduct":"AXV101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Charles River Laboratories, Inc \/ Axovia Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Charles River Laboratories, Inc \/ Axovia Therapeutics"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Prevail Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Termination","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Precision BioSciences","amount2":0.56000000000000005,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.56000000000000005,"dosageForm":"","sponsorNew":"Precision BioSciences \/ Prevail Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Precision BioSciences \/ Prevail Therapeutics"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Deep Track Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Private Placement","leadProduct":"KRRO-110","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Korro Bio","amount2":0.070000000000000007,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.070000000000000007,"dosageForm":"","sponsorNew":"Korro Bio \/ Deep Track Capital","highestDevelopmentStatusID":"4","companyTruncated":"Korro Bio \/ Deep Track Capital"},{"orgOrder":0,"company":"hC Bioscience","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"HCB-101","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"hC Bioscience","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"hC Bioscience \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"hC Bioscience \/ Not Applicable"},{"orgOrder":0,"company":"Metagenomi","sponsor":"Moderna Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Termination","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Metagenomi","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Metagenomi \/ Moderna Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ Moderna Therapeutics"},{"orgOrder":0,"company":"Latus Bio","sponsor":"8VC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Series A Financing","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Latus Bio","amount2":0.050000000000000003,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"","sponsorNew":"Latus Bio \/ 8VC","highestDevelopmentStatusID":"4","companyTruncated":"Latus Bio \/ 8VC"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Be Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Be Biopharma \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Be Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"Silence Therapeutics","sponsor":"Hansoh Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Collaboration","leadProduct":"siRNA-based Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Silence Therapeutics","amount2":1.3200000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":1.3200000000000001,"dosageForm":"","sponsorNew":"Silence Therapeutics \/ Hansoh Pharma","highestDevelopmentStatusID":"4","companyTruncated":"Silence Therapeutics \/ Hansoh Pharma"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Foundation Fighting Blindness","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"OPGx-RHO","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Opus Genetics \/ Foundation Fighting Blindness","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ Foundation Fighting Blindness"},{"orgOrder":0,"company":"2Seventy Bio","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Divestment","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"2Seventy Bio","amount2":0.040000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"","sponsorNew":"2Seventy Bio \/ Novo Nordisk","highestDevelopmentStatusID":"4","companyTruncated":"2Seventy Bio \/ Novo Nordisk"}]

    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

                            Axplora

                            Germany
                            Medlab Asia & Asia Health
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothAxplora- The partner of choice for complex APIs.

                            Lead Product(s) : OTOF-GT,Aav Vectors

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Sensorion

                            Deal Size : Undisclosed

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Sensorion and Novasep Announce Signature of a Gene Therapy Product Development and Manufacturing Agreement

                            Details : Novasep will be in charge of developing and manufacturing the two AAV vectors designed for the Sensorion OTOF-GT project and will supply Drug Product batches to support preclinical and clinical studies.

                            Brand Name : OTOF-GT

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            October 27, 2020

                            Lead Product(s) : OTOF-GT,Aav Vectors

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Sensorion

                            Deal Size : Undisclosed

                            Deal Type : Agreement

                            Axplora CB

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            02

                            Caszyme

                            Lithuania arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Caszyme

                            Lithuania arrow
                            Medlab Asia & Asia Health
                            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) : Cell and Gene Therapy

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Integra Therapeutics

                            Deal Size : $42.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Caszyme And Integra Ink Deal for Novel CRISPR Cas12l Nucleases

                            Details : The agreement aims for the use of Caszyme's novel Cas12l nucleases to develop safer and more efficient gene and cell therapies.

                            Brand Name : Undisclosed

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            November 04, 2024

                            Lead Product(s) : Cell and Gene Therapy

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Integra Therapeutics

                            Deal Size : $42.0 million

                            Deal Type : Licensing Agreement

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            03

                            Axovia Therapeutics

                            United Kingdom arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Axovia Therapeutics

                            United Kingdom arrow
                            Medlab Asia & Asia Health
                            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) : AXV-101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : A Race Against Blindness

                            Deal Size : $1.0 million

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            $1M Grant Supports Axovia's Bardet-Biedl Syndrome Clinical Trial

                            Details : The funding will be used to support the company’s lead program, AXV-101, an adeno-associated virus (AAV9)-based gene therapy targeting retinal dystrophy associated with Bardet-Biedl Syndrome.

                            Brand Name : AXV-101

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            October 30, 2024

                            Lead Product(s) : AXV-101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : A Race Against Blindness

                            Deal Size : $1.0 million

                            Deal Type : Funding

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            04

                            Papillon Therapeutics

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Papillon Therapeutics

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            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) : PPL-002

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Papillon Therapeutics Receives Orphan Drug Designation For PPL-002 in Danon Disease

                            Details : PPL-002 is an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, being evaluated for the treatment of Danon disease.

                            Brand Name : PPL-002

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            October 01, 2024

                            Lead Product(s) : PPL-002

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            05

                            Vivet Therapeutics

                            France arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Vivet Therapeutics

                            France arrow
                            Medlab Asia & Asia Health
                            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) : VTX-806

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            European Commission Grants Orphan Drug Designation for Vivet's Gene Therapy for CTX

                            Details : VTX-806, an Adeno-associated viral (AAV) vector encoding human CYP27A1 is being investigating for the treatment of Cerebrotendinous Xanthomatosis (CTX).

                            Brand Name : VTX-806

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            September 05, 2024

                            Lead Product(s) : VTX-806

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            06

                            Metagenomi

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Metagenomi

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            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) : MGX-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Metagenomi Presents Preclinical Data For Durable Factor VIII Activity in Hemophilia A

                            Details : MGX-001, a gene-editing therapy being developed by Metagenomi for hemophilia A, achieved long term persistence of FVIII activity levels in patients.

                            Brand Name : MGX-001

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            September 03, 2024

                            Lead Product(s) : MGX-001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            07

                            Ractigen Therapeutics

                            China arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Ractigen Therapeutics

                            China arrow
                            Medlab Asia & Asia Health
                            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) : RAG-18

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Ractigen Receives FDA Orphan Drug Designation for RAG-18 in DMD and BMD

                            Details : RAG-18 is a first of its kind saRNA candidate designed to specifically target and activate UTRN gene expression. It is being evaluated for duchenne muscular dystrophy & becker muscular dystrophy.

                            Brand Name : RAG-18

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            August 22, 2024

                            Lead Product(s) : RAG-18

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            08

                            GondolaBio

                            Country arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            GondolaBio

                            Country arrow
                            Medlab Asia & Asia Health
                            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 : BridgeBio Pharma

                            Deal Size : $300.0 million

                            Deal Type : Demerger

                            DEALS_DEV arrow-down

                            BridgeBio launches another offshoot with $300M and early genetic disease programs

                            Details : GondolaBio will conduct R&D activities for early-stage clinical or preclinical programs across erythropoietic protoporphyria, alpha-1 antitrypsin deficiency and tuberous sclerosis complex.

                            Brand Name : Undisclosed

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            August 21, 2024

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : BridgeBio Pharma

                            Deal Size : $300.0 million

                            Deal Type : Demerger

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            09

                            Release Therapeutics

                            Switzerland arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Release Therapeutics

                            Switzerland arrow
                            Medlab Asia & Asia Health
                            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 : Undisclosed

                            Deal Size : $3.8 million

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            Release Therapeutics Secures CHF 3.3 Million in Seed Funding

                            Details : The financing is intended to support the initial preclinical stages to advance the clinical development of the company's lead product for treating patients with metachromatic leukodystrophy.

                            Brand Name : Undisclosed

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            August 06, 2024

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : $3.8 million

                            Deal Type : Financing

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            10

                            Meta Pharmaceuticals

                            Hong Kong arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Meta Pharmaceuticals

                            Hong Kong arrow
                            Medlab Asia & Asia Health
                            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) : META-001-PH

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            META Receives Rare Pediatric Disease Designation for META-001-PH

                            Details : META-001-PH is a groundbreaking oral small molecule drug developed by META for the treatment of primary hyperoxaluria.

                            Brand Name : META-001-PH

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            August 05, 2024

                            Lead Product(s) : META-001-PH

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth