[{"orgOrder":0,"company":"CSPC Baike","sponsor":"Beijing Friendship Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Pegfilgrastim","moa":"Granulocyte colony stimulating factor receptor","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"CSPC Baike","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSPC Baike \/ Beijing Friendship Hospital","highestDevelopmentStatusID":"1","companyTruncated":"CSPC Baike \/ Beijing Friendship Hospital"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-102","moa":"Gene","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Shaare Zedek Medical Center","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Taliglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Shaare Zedek Medical Center","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Shaare Zedek Medical Center \/ Pfizer Inc","highestDevelopmentStatusID":"1","companyTruncated":"Shaare Zedek Medical Center \/ Pfizer Inc"},{"orgOrder":0,"company":"Alvotech","sponsor":"Advanz Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ICELAND","productType":"Antibody, Unconjugated","year":"2025","type":"Agreement","leadProduct":"Canakinumab","moa":"Interleukin-1 beta","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Alvotech","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alvotech \/ Advanz Pharma","highestDevelopmentStatusID":"1","companyTruncated":"Alvotech \/ Advanz Pharma"},{"orgOrder":0,"company":"Hope Biosciences","sponsor":"Hope Biosciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"HB-adMSC","moa":"Mesenchymal stem cells","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Hope Biosciences","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hope Biosciences \/ Hope Biosciences","highestDevelopmentStatusID":"1","companyTruncated":"Hope Biosciences \/ Hope Biosciences"},{"orgOrder":0,"company":"University of Primorska","sponsor":"Biovis d.o.o.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SLOVENIA","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Coenzyme Q10","moa":"Mitochondrial electron transport chain","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Primorska","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Primorska \/ Biovis d.o.o.","highestDevelopmentStatusID":"1","companyTruncated":"University of Primorska \/ Biovis d.o.o."},{"orgOrder":0,"company":"University of Illinois, Urbana-Champaign","sponsor":"BIO-CAT","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Protease","moa":"Polypeptides","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Illinois, Urbana-Champaign","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Illinois, Urbana-Champaign \/ BIO-CAT","highestDevelopmentStatusID":"1","companyTruncated":"University of Illinois, Urbana-Champaign \/ BIO-CAT"},{"orgOrder":0,"company":"Meta Healthcare Ltd","sponsor":"Birmingham Women's and Children's NHS Foundation Trust","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Amino Acid","year":"2024","type":"Inapplicable","leadProduct":"L-VALINE","moa":"Propionyl-CoA carboxylase beta chain, mitochondrial; Branched-chain-amino-acid aminotransferase, cytosolic; Valine--tRNA ligase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Meta Healthcare Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Meta Healthcare Ltd \/ Birmingham Women's and Children's NHS Foundation Trust","highestDevelopmentStatusID":"1","companyTruncated":"Meta Healthcare Ltd \/ Birmingham Women's and Children's NHS Foundation Trust"},{"orgOrder":0,"company":"The National Comprehensive Cancer Network","sponsor":"Puma Biotechnology","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Collaboration","leadProduct":"Neratinib","moa":"Receptor protein-tyrosine kinase erbB-4 | Epidermal growth factor receptor erbB1 | Receptor protein-tyrosine kinase erbB-2","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"The National Comprehensive Cancer Network","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"The National Comprehensive Cancer Network \/ Puma Biotechnology","highestDevelopmentStatusID":"1","companyTruncated":"The National Comprehensive Cancer Network \/ Puma Biotechnology"},{"orgOrder":0,"company":"He Huang","sponsor":"Lingyi Biotech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"LY-M001","moa":"SARS-CoV-2 spike protein","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"He Huang","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"He Huang \/ Lingyi Biotech","highestDevelopmentStatusID":"1","companyTruncated":"He Huang \/ Lingyi Biotech"},{"orgOrder":0,"company":"ALPS Biotech CO. LTD","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Antrodia Cinnamomea","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"ALPS Biotech CO. LTD","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ALPS Biotech CO. LTD \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"ALPS Biotech CO. LTD \/ Undisclosed"},{"orgOrder":0,"company":"Western Sydney Local Health District","sponsor":"Westmead Institute for Medical Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"AUSTRALIA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Beetroot","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Western Sydney Local Health District","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Western Sydney Local Health District \/ Westmead Institute for Medical Research","highestDevelopmentStatusID":"1","companyTruncated":"Western Sydney Local Health District \/ Westmead Institute for Medical Research"},{"orgOrder":0,"company":"Bioray Laboratories","sponsor":"Nanfang Hospital, Southern Medical University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BRL-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Bioray Laboratories","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bioray Laboratories \/ Nanfang Hospital, Southern Medical University","highestDevelopmentStatusID":"1","companyTruncated":"Bioray Laboratories \/ Nanfang Hospital, Southern Medical University"},{"orgOrder":0,"company":"Bioray Laboratories","sponsor":"First Affiliated Hospital of Guangxi Medical University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BRL-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Bioray Laboratories","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bioray Laboratories \/ First Affiliated Hospital of Guangxi Medical University","highestDevelopmentStatusID":"1","companyTruncated":"Bioray Laboratories \/ First Affiliated Hospital of Guangxi Medical University"},{"orgOrder":0,"company":"Bioray Laboratories","sponsor":"Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"BRL-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Bioray Laboratories","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bioray Laboratories \/ Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College","highestDevelopmentStatusID":"1","companyTruncated":"Bioray Laboratories \/ Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College"},{"orgOrder":0,"company":"Maastricht University Medical Center","sponsor":"DSM Food Specialties","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Canola Protein","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Maastricht University Medical Center","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Maastricht University Medical Center \/ DSM Food Specialties","highestDevelopmentStatusID":"1","companyTruncated":"Maastricht University Medical Center \/ DSM Food Specialties"},{"orgOrder":0,"company":"Children's Hospital of Fudan University","sponsor":"Hedu Biotechnology","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"CBT102-A","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Children's Hospital of Fudan University","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Children's Hospital of Fudan University \/ Hedu Biotechnology","highestDevelopmentStatusID":"1","companyTruncated":"Children's Hospital of Fudan University \/ Hedu Biotechnology"},{"orgOrder":0,"company":"Institute of Hematology & Blood Diseases Hospital, China","sponsor":"Connoya Biomedical Technology","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"CM-336","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Institute of Hematology & Blood Diseases Hospital, China","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Institute of Hematology & Blood Diseases Hospital, China \/ Keymed Biosciences","highestDevelopmentStatusID":"1","companyTruncated":"Institute of Hematology & Blood Diseases Hospital, China \/ Keymed Biosciences"},{"orgOrder":0,"company":"Frontera Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FT-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Frontera Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Frontera Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Frontera Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Genecombio Ltd.","sponsor":"The Children's Hospital of Zhejiang University School of Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"GCB-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genecombio Ltd.","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genecombio Ltd. \/ The Children's Hospital of Zhejiang University School of Medicine","highestDevelopmentStatusID":"1","companyTruncated":"Genecombio Ltd. \/ The Children's Hospital of Zhejiang University School of Medicine"},{"orgOrder":0,"company":"Leiden University Medical Center","sponsor":"ZonMw | European Commission","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Gene Therapy","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Leiden University Medical Center","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Leiden University Medical Center \/ ZonMw | European Commission","highestDevelopmentStatusID":"1","companyTruncated":"Leiden University Medical Center \/ ZonMw | European Commission"},{"orgOrder":0,"company":"GlycosBio, Inc.","sponsor":"Baylor College of Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Dietary Supplement","year":"2021","type":"Inapplicable","leadProduct":"Glycosbio Nutritional Supplement","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"GlycosBio, Inc.","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GlycosBio, Inc. \/ Baylor College of Medicine","highestDevelopmentStatusID":"1","companyTruncated":"GlycosBio, Inc. \/ Baylor College of Medicine"},{"orgOrder":0,"company":"Ajinomoto Company","sponsor":"Birmingham Children's Hospital | Great Ormond Street Hospital for Children NHS Foundation Trust","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Glytactin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Ajinomoto Company","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ajinomoto Company \/ Birmingham Children's Hospital | Great Ormond Street Hospital for Children NHS Foundation Trust","highestDevelopmentStatusID":"1","companyTruncated":"Ajinomoto Company \/ Birmingham Children's Hospital | Great Ormond Street Hospital for Children NHS Foundation Trust"},{"orgOrder":0,"company":"Nutricia Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Dietary Supplement","year":"2025","type":"Inapplicable","leadProduct":"GMP Intervention Product","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Nutricia Inc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nutricia Inc \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Nutricia Inc \/ Undisclosed"},{"orgOrder":0,"company":"Dr. Sch\u00e4r AG \/ SPA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Dietary Supplement","year":"2020","type":"Inapplicable","leadProduct":"GMPOWER","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dr. Sch\u00e4r AG \/ SPA","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dr. Sch\u00e4r AG \/ SPA \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Dr. Sch\u00e4r AG \/ SPA \/ Undisclosed"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Mitsubishi UFJ Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell & Gene Therapy","year":"2020","type":"Collaboration","leadProduct":"GtCCR4","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Daiichi Sankyo \/ Mitsubishi UFJ Capital","highestDevelopmentStatusID":"1","companyTruncated":"Daiichi Sankyo \/ Mitsubishi UFJ Capital"},{"orgOrder":0,"company":"HuidaGene Therapeutics","sponsor":"Peking University First Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"HG204","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"HuidaGene Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"HuidaGene Therapeutics \/ Peking University First Hospital","highestDevelopmentStatusID":"1","companyTruncated":"HuidaGene Therapeutics \/ Peking University First Hospital"},{"orgOrder":0,"company":"HuidaGene Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"HG302","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"HuidaGene Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"HuidaGene Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"HuidaGene Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"MED Institute Inc.","sponsor":"Signature Biologics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Human Umbilical Cord Mesenchymal Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"MED Institute Inc.","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MED Institute Inc. \/ Signature Biologics","highestDevelopmentStatusID":"1","companyTruncated":"MED Institute Inc. \/ Signature Biologics"},{"orgOrder":0,"company":"Dr. Sch\u00e4r AG \/ SPA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Dietary Supplement","year":"2020","type":"Inapplicable","leadProduct":"Lipano","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dr. Sch\u00e4r AG \/ SPA","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dr. Sch\u00e4r AG \/ SPA \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Dr. Sch\u00e4r AG \/ SPA \/ Undisclosed"},{"orgOrder":0,"company":"Nutricia Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Low Calorie Protein Substitute","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Nutricia Inc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Nutricia Inc \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Nutricia Inc \/ Undisclosed"},{"orgOrder":0,"company":"Shanghai General Hospital","sponsor":"Innostellar Biotherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"LX103","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Shanghai General Hospital","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Shanghai General Hospital \/ Innostellar Biotherapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Shanghai General Hospital \/ Innostellar Biotherapeutics"},{"orgOrder":0,"company":"Hunterian Medicine","sponsor":"Inscripta","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Licensing Agreement","leadProduct":"MAD7 Nuclease","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Hunterian Medicine","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hunterian Medicine \/ Inscripta","highestDevelopmentStatusID":"1","companyTruncated":"Hunterian Medicine \/ Inscripta"},{"orgOrder":0,"company":"MitoChem Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"MC16","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"MitoChem Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MitoChem Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"MitoChem Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Genevant Sciences","sponsor":"2Seventy Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Cell & Gene Therapy","year":"2022","type":"Licensing Agreement","leadProduct":"MegaTAL mRNA","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genevant Sciences","amount2":0.040000000000000001,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Genevant Sciences \/ 2Seventy Bio","highestDevelopmentStatusID":"1","companyTruncated":"Genevant Sciences \/ 2Seventy Bio"},{"orgOrder":0,"company":"2Seventy Bio","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Collaboration","leadProduct":"MegaTAL mRNA","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"2Seventy Bio","amount2":0.040000000000000001,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Undisclosed","sponsorNew":"2Seventy Bio \/ Novo Nordisk","highestDevelopmentStatusID":"1","companyTruncated":"2Seventy Bio \/ Novo Nordisk"},{"orgOrder":0,"company":"SR-Tiget","sponsor":"Genespire","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2020","type":"Collaboration","leadProduct":"Microrna-Regulated Lentiviral Vectors","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"SR-Tiget","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SR-Tiget \/ Genespire","highestDevelopmentStatusID":"1","companyTruncated":"SR-Tiget \/ Genespire"},{"orgOrder":0,"company":"Dr. Sch\u00e4r AG \/ SPA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Dietary Supplement","year":"2020","type":"Inapplicable","leadProduct":"Motion","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dr. Sch\u00e4r AG \/ SPA","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dr. Sch\u00e4r AG \/ SPA \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Dr. Sch\u00e4r AG \/ SPA \/ Undisclosed"},{"orgOrder":0,"company":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine","sponsor":"Mabwell","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"MWAV201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine \/ Mabwell","highestDevelopmentStatusID":"1","companyTruncated":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine \/ Mabwell"},{"orgOrder":0,"company":"Myogem Health Company, S.L.","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Dietary Supplement","year":"2020","type":"Inapplicable","leadProduct":"Myodm","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Myogem Health Company, S.L.","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Myogem Health Company, S.L. \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Myogem Health Company, S.L. \/ Undisclosed"},{"orgOrder":0,"company":"AgeX Therapeutics","sponsor":"University of California San Diego","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Collaboration","leadProduct":"Neural Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"AgeX Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AgeX Therapeutics \/ University of California San Diego","highestDevelopmentStatusID":"1","companyTruncated":"AgeX Therapeutics \/ University of California San Diego"},{"orgOrder":0,"company":"LGD","sponsor":"CEN Biotech | Assistance Publique Hopitaux De Marseille | Etablissement Fran\u00e7ais du Sang","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Dietary Supplement","year":"2023","type":"Inapplicable","leadProduct":"NUV001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"LGD","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"LGD \/ CEN Biotech | Assistance Publique Hopitaux De Marseille | Etablissement Fran\u00e7ais du Sang","highestDevelopmentStatusID":"1","companyTruncated":"LGD \/ CEN Biotech | Assistance Publique Hopitaux De Marseille | Etablissement Fran\u00e7ais du Sang"},{"orgOrder":0,"company":"LGD","sponsor":"Prorelix Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Dietary Supplement","year":"2023","type":"Inapplicable","leadProduct":"NUV001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"LGD","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"LGD \/ Prorelix Research","highestDevelopmentStatusID":"1","companyTruncated":"LGD \/ Prorelix Research"},{"orgOrder":0,"company":"Emendo Biotherapeutics","sponsor":"AnGes","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Series B Financing","leadProduct":"OMNI Nuclease","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Emendo Biotherapeutics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Emendo Biotherapeutics \/ AnGes","highestDevelopmentStatusID":"1","companyTruncated":"Emendo Biotherapeutics \/ AnGes"},{"orgOrder":0,"company":"GALEN LTD","sponsor":"Birmingham Women's and Children's NHS Foundation Trust","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2024","type":"Inapplicable","leadProduct":"PKU Easy Microtabs Plus","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"GALEN LTD","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"GALEN LTD \/ Birmingham Women's and Children's NHS Foundation Trust","highestDevelopmentStatusID":"1","companyTruncated":"GALEN LTD \/ Birmingham Women's and Children's NHS Foundation Trust"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2021","type":"Inapplicable","leadProduct":"PKU Explore","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2021","type":"Inapplicable","leadProduct":"PKU Express Plus","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Undisclosed"},{"orgOrder":0,"company":"APR Applied Pharma Research","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"PKU Golike","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"APR Applied Pharma Research","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"APR Applied Pharma Research \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"APR Applied Pharma Research \/ Undisclosed"},{"orgOrder":0,"company":"APR Applied Pharma Research","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"PKU Golike","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"APR Applied Pharma Research","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"APR Applied Pharma Research \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"APR Applied Pharma Research \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2020","type":"Inapplicable","leadProduct":"PKU Sphere","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2021","type":"Inapplicable","leadProduct":"PKU Sphere","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Birmingham Women's and Children's NHS Foundation Trust","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2023","type":"Inapplicable","leadProduct":"PKU UP","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vitaflo International, Ltd \/ Birmingham Women's and Children's NHS Foundation Trust","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Birmingham Women's and Children's NHS Foundation Trust"},{"orgOrder":0,"company":"Nutricia Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"PKU\/TYR GMP Protein","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Nutricia Inc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nutricia Inc \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Nutricia Inc \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"TeamedOn","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"rAAV2tYF-CB-hRS1","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ TeamedOn","highestDevelopmentStatusID":"1","companyTruncated":"Agtc \/ TeamedOn"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"rAAVrh8-HexA\/HexB","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sio Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Sio Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Virica Biotech","sponsor":"Innovation, Science and Economic Development Canada","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Cell & Gene Therapy","year":"2022","type":"Collaboration","leadProduct":"Re-engineered AAV-LPL","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Virica Biotech","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Virica Biotech \/ Innovation, Science and Economic Development Canada","highestDevelopmentStatusID":"1","companyTruncated":"Virica Biotech \/ Innovation, Science and Economic Development Canada"},{"orgOrder":0,"company":"Cornell University","sponsor":"reVision Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Licensing Agreement","leadProduct":"REV-0100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Cornell University","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cornell University \/ reVision Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Cornell University \/ reVision Therapeutics"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"TAK-577","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Corporacion Parc Tauli","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Tridocosahexaenoine","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Corporacion Parc Tauli","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Corporacion Parc Tauli \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Corporacion Parc Tauli \/ Undisclosed"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2021","type":"Inapplicable","leadProduct":"TYR sphere","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vitaflo International, Ltd","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Vitaflo International, Ltd \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Undisclosed"},{"orgOrder":0,"company":"Applied Science & Performance Institute","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Plant Extract\/Herbal","year":"2025","type":"Inapplicable","leadProduct":"CAS 501-94-0","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Applied Science & Performance Institute","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oil","sponsorNew":"Applied Science & Performance Institute \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Applied Science & Performance Institute \/ Undisclosed"},{"orgOrder":0,"company":"Biotrial","sponsor":"European Union | Nantes University Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Biotrial","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biotrial \/ European Union | Nantes University Hospital","highestDevelopmentStatusID":"1","companyTruncated":"Biotrial \/ European Union | Nantes University Hospital"},{"orgOrder":0,"company":"University Hospital, Montpellier","sponsor":"Institut de Recherche en Infectiologie de Montpellier (CNRS) | A*STAR","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University Hospital, Montpellier","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University Hospital, Montpellier \/ Institut de Recherche en Infectiologie de Montpellier (CNRS) | A*STAR","highestDevelopmentStatusID":"1","companyTruncated":"University Hospital, Montpellier \/ Institut de Recherche en Infectiologie de Montpellier (CNRS) | A*STAR"},{"orgOrder":0,"company":"Centre Hospitalier de Saint-Denis","sponsor":"INSERM U955","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Centre Hospitalier de Saint-Denis","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Centre Hospitalier de Saint-Denis \/ INSERM U955","highestDevelopmentStatusID":"1","companyTruncated":"Centre Hospitalier de Saint-Denis \/ INSERM U955"},{"orgOrder":0,"company":"University of Primorska","sponsor":"Biostile d.o.o. | QFarm s.r.l.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SLOVENIA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Primorska","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Primorska \/ Biostile d.o.o. | QFarm s.r.l.","highestDevelopmentStatusID":"1","companyTruncated":"University of Primorska \/ Biostile d.o.o. | QFarm s.r.l."},{"orgOrder":0,"company":"Genespire","sponsor":"Sofinnova Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genespire","amount2":0.02,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Injection","sponsorNew":"Genespire \/ Sofinnova Partners","highestDevelopmentStatusID":"1","companyTruncated":"Genespire \/ Sofinnova Partners"},{"orgOrder":0,"company":"CYTOO","sponsor":"Astellas Gene Therapies","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"CYTOO","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CYTOO \/ Astellas Gene Therapies","highestDevelopmentStatusID":"1","companyTruncated":"CYTOO \/ Astellas Gene Therapies"},{"orgOrder":0,"company":"Sirion Biotech","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sirion Biotech","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sirion Biotech \/ Sanofi","highestDevelopmentStatusID":"1","companyTruncated":"Sirion Biotech \/ Sanofi"},{"orgOrder":0,"company":"Genespire","sponsor":"Sofinnova Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2020","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genespire","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genespire \/ Sofinnova Partners","highestDevelopmentStatusID":"1","companyTruncated":"Genespire \/ Sofinnova Partners"},{"orgOrder":0,"company":"Anjarium Biosciences","sponsor":"Abingworth","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Anjarium Biosciences","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Anjarium Biosciences \/ Abingworth","highestDevelopmentStatusID":"1","companyTruncated":"Anjarium Biosciences \/ Abingworth"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Ysios Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"SpliceBio","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"SpliceBio \/ Ysios Capital","highestDevelopmentStatusID":"1","companyTruncated":"SpliceBio \/ Ysios Capital"},{"orgOrder":0,"company":"Brigham and Women's Hospital","sponsor":"Baylor College of Medicine | Boston Children's Hospital | Broad Institute of MIT and Harvard | Dartmouth-Hitchcock Medical Center | Harvard Pilgrim Health Care | Howard University | HudsonAlpha Institute for Biotechnology | Massachusetts General Hospital ","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Brigham and Women's Hospital","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Brigham and Women's Hospital \/ Baylor College of Medicine | Boston Children's Hospital | Broad Institute of MIT and Harvard | Dartmouth-Hitchcock Medical Center | Harvard Pilgrim Health Care | Howard University | HudsonAlpha Institute for Biotechnology | Massachusetts General Hospital ","highestDevelopmentStatusID":"1","companyTruncated":"Brigham and Women's Hospital \/ Baylor College of Medicine | Boston Children's Hospital | Broad Institute of MIT and Harvard | Dartmouth-Hitchcock Medical Center | Harvard Pilgrim Health Care | Howard University | HudsonAlpha Institute for Biotechnology | Massachusetts General Hospital "},{"orgOrder":0,"company":"University of Massachusetts Medical School","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Massachusetts Medical School","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Massachusetts Medical School \/ CANbridge Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"University of Massachusetts Medical School \/ CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Public Offering","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Arcturus Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arcturus Therapeutics \/ Guggenheim Securities","highestDevelopmentStatusID":"1","companyTruncated":"Arcturus Therapeutics \/ Guggenheim Securities"},{"orgOrder":0,"company":"National Institutes of Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"National Institutes of Health","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Institutes of Health \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"National Institutes of Health \/ Undisclosed"},{"orgOrder":0,"company":"Codexis","sponsor":"Crosswalk Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Codexis","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Codexis \/ Crosswalk Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Codexis \/ Crosswalk Therapeutics"},{"orgOrder":0,"company":"AavantiBio","sponsor":"University of Florida","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"AavantiBio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AavantiBio \/ University of Florida","highestDevelopmentStatusID":"1","companyTruncated":"AavantiBio \/ University of Florida"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Personalis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Personalis","highestDevelopmentStatusID":"1","companyTruncated":"Sarepta Therapeutics \/ Personalis"},{"orgOrder":0,"company":"Duchenne research","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Duchenne research","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Duchenne research \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Duchenne research \/ Undisclosed"},{"orgOrder":0,"company":"MaxCyte","sponsor":"Curamys","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"MaxCyte","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MaxCyte \/ Curamys","highestDevelopmentStatusID":"1","companyTruncated":"MaxCyte \/ Curamys"},{"orgOrder":0,"company":"PepGen","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"PepGen","amount2":0.050000000000000003,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"PepGen \/ RA Capital Management","highestDevelopmentStatusID":"1","companyTruncated":"PepGen \/ RA Capital Management"},{"orgOrder":0,"company":"Dewpoint Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dewpoint Therapeutics","amount2":0.23999999999999999,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Dewpoint Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"1","companyTruncated":"Dewpoint Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"AavantiBio","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"AavantiBio","amount2":0.11,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"AavantiBio \/ Sarepta Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"AavantiBio \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Entos Pharmaceuticals","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Entos Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Entos Pharmaceuticals \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Entos Pharmaceuticals \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Mammoth Biosciences","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Mammoth Biosciences","amount2":0.68999999999999995,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.68999999999999995,"dosageForm":"Undisclosed","sponsorNew":"Mammoth Biosciences \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"Mammoth Biosciences \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"hC Bioscience","sponsor":"Arch Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"hC Bioscience","amount2":0.02,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Undisclosed","sponsorNew":"hC Bioscience \/ Arch Ventures","highestDevelopmentStatusID":"1","companyTruncated":"hC Bioscience \/ Arch Ventures"},{"orgOrder":0,"company":"Vigene Bioscience","sponsor":"ASC Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Vigene Bioscience","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vigene Bioscience \/ ASC Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Vigene Bioscience \/ ASC Therapeutics"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Affinia Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Affinia Therapeutics \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"Affinia Therapeutics \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Cureduchenne Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dyne Therapeutics \/ Cureduchenne Ventures","highestDevelopmentStatusID":"1","companyTruncated":"Dyne Therapeutics \/ Cureduchenne Ventures"},{"orgOrder":0,"company":"Replay Bio","sponsor":"KKR","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Replay Bio","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Replay Bio \/ KKR","highestDevelopmentStatusID":"1","companyTruncated":"Replay Bio \/ KKR"},{"orgOrder":0,"company":"Dr Jorg Taubel","sponsor":"Richmond Pharmacology | National Amyloidosis Centre","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dr Jorg Taubel","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Dr Jorg Taubel \/ Richmond Pharmacology | National Amyloidosis Centre","highestDevelopmentStatusID":"1","companyTruncated":"Dr Jorg Taubel \/ Richmond Pharmacology | National Amyloidosis Centre"}]
Find Novel Drugs for Genetic Disease under Development
Filters
×
FILTER:
Company Name
Year
DEALS // DEV.
Country
Sponsor
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Reset All
