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Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"PYC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"PYC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"SparingVision","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ SparingVision","highestDevelopmentStatusID":"5","companyTruncated":"Agtc \/ SparingVision"},{"orgOrder":0,"company":"DTx Pharma","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"DTx Pharma","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"DTx Pharma \/ RA Capital Management","highestDevelopmentStatusID":"5","companyTruncated":"DTx Pharma \/ RA Capital Management"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"VP-001","moa":"PRPF31","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"PYC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"PYC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"VP-001","moa":"PRPF31","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"PYC Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"PYC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"PYC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"NMD PHARMA \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"NMD PHARMA \/ Inapplicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Rimtuzalcap","moa":"Calcium-sensitive potassium ion channel PAM","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"ViGeneron","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"VG801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"ViGeneron","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ViGeneron \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"ViGeneron \/ Inapplicable"},{"orgOrder":0,"company":"Ascidian Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Large Molecule","year":"2024","type":"Inapplicable","leadProduct":"ACDN-01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Ascidian Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ascidian Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Ascidian Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Ascidian Therapeutics","sponsor":"Apple Tree Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Ascidian Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ascidian Therapeutics \/ Apple Tree Partners","highestDevelopmentStatusID":"5","companyTruncated":"Ascidian Therapeutics \/ Apple Tree Partners"},{"orgOrder":0,"company":"Mammoth Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"MB-111","moa":"APOC-3","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Mammoth Biosciences","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"Mammoth Biosciences \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Mammoth Biosciences \/ Inapplicable"},{"orgOrder":0,"company":"Ensoma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"EN-374","moa":"CYBB","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Ensoma","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ensoma \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Ensoma \/ Inapplicable"}]
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