[{"orgOrder":0,"company":"Memorial Sloan Kettering Cancer Center","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Bortezomib","moa":"26S proteasome","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Memorial Sloan Kettering Cancer Center","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Memorial Sloan Kettering Cancer Center \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Memorial Sloan Kettering Cancer Center \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Sutphin Drugs","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Nitisinone","moa":"4-hydroxyphenylpyruvate dioxygenase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sutphin Drugs","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sutphin Drugs \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Sutphin Drugs \/ Undisclosed"},{"orgOrder":0,"company":"Versiti","sponsor":"Medical College of Wisconsin | La Jolla Institute for Allergy & Immunology | University of Illinois, Chicago | Oregon Health and Science University | Dana-Farber Cancer Institute","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Regadenoson Monohydrate","moa":"Adenosine A2a receptor","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Versiti","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Versiti \/ Medical College of Wisconsin | La Jolla Institute for Allergy & Immunology | University of Illinois, Chicago | Oregon Health and Science University | Dana-Farber Cancer Institute","highestDevelopmentStatusID":"1","companyTruncated":"Versiti \/ Medical College of Wisconsin | La Jolla Institute for Allergy & Immunology | University of Illinois, Chicago | Oregon Health and Science University | Dana-Farber Cancer Institute"},{"orgOrder":0,"company":"Lucas Otieno Tina, MD MSc","sponsor":"GSK | Strathmore University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Lofexidine","moa":"Adrenergic receptor alpha-2","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Lucas Otieno Tina, MD MSc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Lucas Otieno Tina, MD MSc \/ GSK | Strathmore University","highestDevelopmentStatusID":"1","companyTruncated":"Lucas Otieno Tina, MD MSc \/ GSK | Strathmore University"},{"orgOrder":0,"company":"University College Cork","sponsor":"Queen's University, Belfast | Universit\u00e9 Paris Cit\u00e9 | University of Dundee | University of Washington | University Hospital Heidelberg | Teagasc | Clininfo | GABO:mi | Papworth Hospital NHS Foundation Trust | KU Leuven | Assistance Publique \u2013 H\u00f4pitaux de ","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Ceftazidime","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University College Cork","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University College Cork \/ Queen's University, Belfast | Universit\u00e9 Paris Cit\u00e9 | University of Dundee | University of Washington | University Hospital Heidelberg | Teagasc | Clininfo | GABO:mi | Papworth Hospital NHS Foundation Trust | KU Leuven | Assistance Publique \u2013 H\u00f4pitaux de ","highestDevelopmentStatusID":"1","companyTruncated":"University College Cork \/ Queen's University, Belfast | Universit\u00e9 Paris Cit\u00e9 | University of Dundee | University of Washington | University Hospital Heidelberg | Teagasc | Clininfo | GABO:mi | Papworth Hospital NHS Foundation Trust | KU Leuven | Assistance Publique \u2013 H\u00f4pitaux de "},{"orgOrder":0,"company":"Unity Health Toronto","sponsor":"Sunnybrook Health Sciences Centre | University of Toronto","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Antibiotic","year":"2010","type":"Inapplicable","leadProduct":"Meropenem","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Unity Health Toronto","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Unity Health Toronto \/ Sunnybrook Health Sciences Centre | University of Toronto","highestDevelopmentStatusID":"1","companyTruncated":"Unity Health Toronto \/ Sunnybrook Health Sciences Centre | University of Toronto"},{"orgOrder":0,"company":"National Taiwan University Hospital","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"TAIWAN","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Eliglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"National Taiwan University Hospital","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"National Taiwan University Hospital \/ Sanofi","highestDevelopmentStatusID":"1","companyTruncated":"National Taiwan University Hospital \/ Sanofi"},{"orgOrder":0,"company":"National Taiwan University Hospital","sponsor":"Johnson & Johnson","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"TAIWAN","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"National Taiwan University Hospital","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"National Taiwan University Hospital \/ Johnson & Johnson","highestDevelopmentStatusID":"1","companyTruncated":"National Taiwan University Hospital \/ Johnson & Johnson"},{"orgOrder":0,"company":"Unity Health Toronto","sponsor":"The Ottawa Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Thrombin","moa":"Coagulation factor XI; Coagulation factor XIII A chain; Coagulation factor XIII B chain; Fibrinogen alpha chain; Fibrinogen beta chain; Fibrinogen gamma chain; Coagulation factor V; Coagulation factor VIII; Proteinase-activated receptor 1; Proteinase-activated receptor 4","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Unity Health Toronto","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"Unity Health Toronto \/ The Ottawa Hospital","highestDevelopmentStatusID":"1","companyTruncated":"Unity Health Toronto \/ The Ottawa Hospital"},{"orgOrder":0,"company":"Yale University","sponsor":"Bausch Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Trientine Hydrochloride","moa":"Copper","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Yale University","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Yale University \/ Bausch Health","highestDevelopmentStatusID":"1","companyTruncated":"Yale University \/ Bausch Health"},{"orgOrder":0,"company":"University of California, San Diego","sponsor":"Amgen Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Amino Acid","year":"2011","type":"Inapplicable","leadProduct":"Cysteamine Bitartrate","moa":"Cystine","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of California, San Diego","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of California, San Diego \/ Amgen Inc","highestDevelopmentStatusID":"1","companyTruncated":"University of California, San Diego \/ Amgen Inc"},{"orgOrder":0,"company":"Masonic Cancer Center, University of Minnesota","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2014","type":"Inapplicable","leadProduct":"Galsulfase","moa":"Dermatan sulfate","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Masonic Cancer Center, University of Minnesota","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Masonic Cancer Center, University of Minnesota \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Masonic Cancer Center, University of Minnesota \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2015","type":"Inapplicable","leadProduct":"Drisapersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"City of Hope","sponsor":"Charta Foundation | Grifols International | CSL Behring | Biotest","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2010","type":"Inapplicable","leadProduct":"Coagulation Factor VIII","moa":"F8*","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"City of Hope","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"City of Hope \/ Charta Foundation | Grifols International | CSL Behring | Biotest","highestDevelopmentStatusID":"1","companyTruncated":"City of Hope \/ Charta Foundation | Grifols International | CSL Behring | Biotest"},{"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":"Cameroon Baptist Convention Health","sponsor":"Carolinas Medical Center | Muhimbili National Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CAMEROON","productType":"Controlled Substance","year":"2015","type":"Inapplicable","leadProduct":"Ketamine Hydrochloride","moa":"Glutamate [NMDA] receptor","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Cameroon Baptist Convention Health","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cameroon Baptist Convention Health \/ Carolinas Medical Center | Muhimbili National Hospital","highestDevelopmentStatusID":"1","companyTruncated":"Cameroon Baptist Convention Health \/ Carolinas Medical Center | Muhimbili National Hospital"},{"orgOrder":0,"company":"Rambam Health Care Campus","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Probiotic","year":"2013","type":"Inapplicable","leadProduct":"Probiotic","moa":"Immune","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Rambam Health Care Campus","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Rambam Health Care Campus \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Rambam Health Care Campus \/ Undisclosed"},{"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 Toronto","sponsor":"Ferring Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Coenzyme Q10","moa":"Mitochondrial electron transport chain","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Toronto","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Toronto \/ Ferring Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"University of Toronto \/ Ferring Pharmaceuticals"},{"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 Texas Health Science Center at Houston","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Texas Health Science Center at Houston","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Texas Health Science Center at Houston \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"University of Texas Health Science Center at Houston \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Linda Randolph","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Linda Randolph","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Linda Randolph \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Linda Randolph \/ BioMarin Pharmaceutical"},{"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":"Eiger BioPharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Protein farnesyltransferase","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Eiger BioPharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Eiger BioPharmaceuticals \/ Undisclosed"},{"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":"United BioSource","sponsor":"AveXis, Inc.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"AVXS-101","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"United BioSource","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"United BioSource \/ AveXis, Inc.","highestDevelopmentStatusID":"1","companyTruncated":"United BioSource \/ AveXis, Inc."},{"orgOrder":0,"company":"Gabrail Cancer Center Research","sponsor":"Children's Healthcare of Atlanta","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Alefacept","moa":"T-cell surface antigen CD2","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Gabrail Cancer Center Research","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Insert","sponsorNew":"Gabrail Cancer Center Research \/ Children's Healthcare of Atlanta","highestDevelopmentStatusID":"1","companyTruncated":"Gabrail Cancer Center Research \/ Children's Healthcare of Atlanta"},{"orgOrder":0,"company":"Sumitomo Pharma Switzerland","sponsor":"National Institutes of Health | National Institute of Allergy and Infectious Diseases | Gansu Provincial Maternal and Child Health Care Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2010","type":"Inapplicable","leadProduct":"Cultured Thymus Tissue","moa":"Thymus tissue","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sumitomo Pharma Switzerland","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"Sumitomo Pharma Switzerland \/ National Institutes of Health | National Institute of Allergy and Infectious Diseases | Gansu Provincial Maternal and Child Health Care Hospital","highestDevelopmentStatusID":"1","companyTruncated":"Sumitomo Pharma Switzerland \/ National Institutes of Health | National Institute of Allergy and Infectious Diseases | Gansu Provincial Maternal and Child Health Care Hospital"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"Inotersen","moa":"Transthyretin","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Jerry Vockley","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Jerry Vockley","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jerry Vockley \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Jerry Vockley \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Washington University School of Medicine","sponsor":"Incyte Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2012","type":"Inapplicable","leadProduct":"Ruxolitinib Phosphate","moa":"Tyrosine-protein kinase JAK1","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Washington University School of Medicine","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Washington University School of Medicine \/ Incyte Corporation","highestDevelopmentStatusID":"1","companyTruncated":"Washington University School of Medicine \/ Incyte Corporation"},{"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":"Axcella Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Dietary Supplement","year":"2019","type":"Inapplicable","leadProduct":"AXA4010","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Axcella Health","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Axcella Health \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Axcella Health \/ 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":"Vitaflo International, Ltd","sponsor":"Arla Foods","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Protein","year":"2019","type":"Inapplicable","leadProduct":"CGMP Protein Substitute","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 \/ Arla Foods","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Arla Foods"},{"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":"Ricardo Maselli","sponsor":"Catalyst Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"Diaminopyridine","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Ricardo Maselli","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ricardo Maselli \/ Catalyst Pharmaceuticals","highestDevelopmentStatusID":"1","companyTruncated":"Ricardo Maselli \/ Catalyst Pharmaceuticals"},{"orgOrder":0,"company":"University of British Columbia","sponsor":"Vitaflo International, Ltd","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Free Amino Acid","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of British Columbia","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of British Columbia \/ Vitaflo International, Ltd","highestDevelopmentStatusID":"1","companyTruncated":"University of British Columbia \/ Vitaflo International, Ltd"},{"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":"City of Hope","sponsor":"Charta Foundation | Grifols International | CSL Behring | Biotest","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"FVIII Concentrates","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"City of Hope","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"City of Hope \/ Charta Foundation | Grifols International | CSL Behring | Biotest","highestDevelopmentStatusID":"1","companyTruncated":"City of Hope \/ Charta Foundation | Grifols International | CSL Behring | Biotest"},{"orgOrder":0,"company":"Children's University Hospital, Ireland","sponsor":"Health Research Board, Ireland | Medical Research Charities Group Ireland | University College Dublin","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Carbohydrate","year":"2014","type":"Inapplicable","leadProduct":"Galactose","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Children's University Hospital, Ireland","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Children's University Hospital, Ireland \/ Health Research Board, Ireland | Medical Research Charities Group Ireland | University College Dublin","highestDevelopmentStatusID":"1","companyTruncated":"Children's University Hospital, Ireland \/ Health Research Board, Ireland | Medical Research Charities Group Ireland | University College Dublin"},{"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":"Nutricia Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Peptide, Unconjugated","year":"2016","type":"Inapplicable","leadProduct":"Glycomacropeptide-Based Protein Substitute","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":"Vitaflo International, Ltd","sponsor":"University College London Hospitals | University of Florida | Great Ormond Street Hospital for Children NHS Foundation Trust | University Medical Center Groningen | H\u00f4pital Necker-Enfants Malades","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"Glycosade","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 \/ University College London Hospitals | University of Florida | Great Ormond Street Hospital for Children NHS Foundation Trust | University Medical Center Groningen | H\u00f4pital Necker-Enfants Malades","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ University College London Hospitals | University of Florida | Great Ormond Street Hospital for Children NHS Foundation Trust | University Medical Center Groningen | H\u00f4pital Necker-Enfants Malades"},{"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":"University of Virginia","sponsor":"Xemed LLC","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Helium-3","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Virginia","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Virginia \/ Xemed LLC","highestDevelopmentStatusID":"1","companyTruncated":"University of Virginia \/ Xemed LLC"},{"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":"Nestle Health Sciences SA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Dietary Supplement","year":"2016","type":"Inapplicable","leadProduct":"Modified Lipid Formulation","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Nestle Health Sciences SA","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Nestle Health Sciences SA \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Nestle Health Sciences SA \/ 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":"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":"Erempharma","sponsor":"Hospices Civils de Lyon | University of Lyon","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"Nebcinal Tobi","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Erempharma","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Erempharma \/ Hospices Civils de Lyon | University of Lyon","highestDevelopmentStatusID":"1","companyTruncated":"Erempharma \/ Hospices Civils de Lyon | University of Lyon"},{"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":"Nutricia Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Phenylalanine-Free 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":"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":"Birmingham Women's and Children's NHS Foundation Trust","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2017","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 \/ 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":"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":"Oregon Health and Science University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2018","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 \/ Oregon Health and Science University","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Oregon Health and Science University"},{"orgOrder":0,"company":"Vitaflo International, Ltd","sponsor":"Kreiskliniken Reutlingen | Johannes Gutenberg University Mainz","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2018","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 \/ Kreiskliniken Reutlingen | Johannes Gutenberg University Mainz","highestDevelopmentStatusID":"1","companyTruncated":"Vitaflo International, Ltd \/ Kreiskliniken Reutlingen | Johannes Gutenberg University Mainz"},{"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":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Dietary Supplement","year":"2017","type":"Inapplicable","leadProduct":"PKU Start","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":"Bellicum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"Rivogenlecleucel","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Bellicum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bellicum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Bellicum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Laboratorios Ordesa","sponsor":"Quantum Experimental | Peruvian Clinical Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"Symbiotic Formula","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Laboratorios Ordesa","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Laboratorios Ordesa \/ Quantum Experimental | Peruvian Clinical Research","highestDevelopmentStatusID":"1","companyTruncated":"Laboratorios Ordesa \/ Quantum Experimental | Peruvian Clinical Research"},{"orgOrder":0,"company":"Nutricia Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Dietary Supplement","year":"2017","type":"Inapplicable","leadProduct":"Synergy","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Nutricia Inc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"Nutricia Inc \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Nutricia Inc \/ Undisclosed"},{"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":"2019","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":"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":"Virginia Commonwealth University","sponsor":"CSL Behring","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2010","type":"Inapplicable","leadProduct":"Vitamin D","moa":"Vitamin D receptor","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Virginia Commonwealth University","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Virginia Commonwealth University \/ CSL Behring","highestDevelopmentStatusID":"1","companyTruncated":"Virginia Commonwealth University \/ CSL Behring"},{"orgOrder":0,"company":"University of Florida","sponsor":"Acorda Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Fampridine","moa":"Voltage-gated potassium channel","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University of Florida","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of Florida \/ Acorda Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"University of Florida \/ Acorda Therapeutics"},{"orgOrder":0,"company":"University Hospital, Lille","sponsor":"Imagine Institute | Octapharma | CSL Behring | LFB Group | Air Liquide Group | The Binding Site Ltd","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"University Hospital, Lille","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University Hospital, Lille \/ Imagine Institute | Octapharma | CSL Behring | LFB Group | Air Liquide Group | The Binding Site Ltd","highestDevelopmentStatusID":"1","companyTruncated":"University Hospital, Lille \/ Imagine Institute | Octapharma | CSL Behring | LFB Group | Air Liquide Group | The Binding Site Ltd"},{"orgOrder":0,"company":"Assistance Publique \u2013 H\u00f4pitaux de Paris","sponsor":"Vaincre la Mucoviscidose","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2018","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Assistance Publique \u2013 H\u00f4pitaux de Paris","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Vaincre la Mucoviscidose","highestDevelopmentStatusID":"1","companyTruncated":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ Vaincre la Mucoviscidose"},{"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":"Genelex Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genelex Corporation","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genelex Corporation \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Genelex Corporation \/ Undisclosed"},{"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"},{"orgOrder":0,"company":"Adventia Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Dietary Supplement","year":"2025","type":"Inapplicable","leadProduct":"Eicosapentaenoic Acid|Docosahexaenoic Acid","moa":"Lipid|PPAR-gamma","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Adventia Pharma","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oil","sponsorNew":"Adventia Pharma \/ Undisclosed","highestDevelopmentStatusID":"1","companyTruncated":"Adventia Pharma \/ Undisclosed"}]

Find Novel Drugs for Genetic Disease under 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...

                          Therapeutic Area by Lead Product

                          Study Phase by Lead Product

                          Company by Lead Product

                          Top Deals by Deal Size (USD bn)

                          01

                          Details : Fosigotifator is a Other Small Molecule drug candidate, which is currently being evaluated in clinical studies for the treatment of Leukoencephalopathies.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          September 19, 2024

                          Lead Product(s) : Fosigotifator

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Abbvie Company Banner

                          02

                          Adventia Pharma

                          Country arrow
                          OTS 2025
                          Not Confirmed

                          Adventia Pharma

                          Country arrow
                          OTS 2025
                          Not Confirmed

                          Details : Eicosapentaenoic Acid|Docosahexaenoic Acid is a Dietary Supplement drug candidate, which is currently being evaluated in Undisclosed clinical studies for the treatment of Frail Elderly Syndrome.

                          Product Name : Undisclosed

                          Product Type : Dietary Supplement

                          Upfront Cash : Inapplicable

                          September 19, 2025

                          Lead Product(s) : Eicosapentaenoic Acid|Docosahexaenoic Acid

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          03

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : Advanz secures exclusive rights from Alvotech to commercialize three biosimilar candidates, Ilaris (canakinumab), Kesimpta (ofatumumab) and an undiclosed biosimilar in Europe.

                          Product Name : Undisclosed

                          Product Type : Antibody, Unconjugated

                          Upfront Cash : Undisclosed

                          May 28, 2025

                          Lead Product(s) : Canakinumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Advanz Pharma

                          Deal Size : Undisclosed

                          Deal Type : Agreement

                          blank

                          04

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : TAK-881 is a drug candidate, which is currently being evaluated in clinical studies for the treatment of Immunologic Deficiency Syndromes.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          May 02, 2025

                          Lead Product(s) : TAK-881

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          05

                          Nutricia Inc

                          Country arrow
                          OTS 2025
                          Not Confirmed

                          Nutricia Inc

                          Country arrow
                          OTS 2025
                          Not Confirmed

                          Details : GMP Intervention Product is a Dietary Supplement drug candidate, which is currently being evaluated in clinical studies for the treatment of Phenylketonurias.

                          Product Name : Undisclosed

                          Product Type : Dietary Supplement

                          Upfront Cash : Inapplicable

                          April 23, 2025

                          Lead Product(s) : GMP Intervention Product

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          06

                          Applied Science & Performance Institute

                          Country arrow
                          OTS 2025
                          Not Confirmed

                          Applied Science & Performance Institute

                          Country arrow
                          OTS 2025
                          Not Confirmed

                          Details : Tyrosol is a Plant Extract/Herbal drug candidate, which is currently being evaluated in clinical studies for the treatment of Creatine Deficiency Syndromes.

                          Product Name : Undisclosed

                          Product Type : Plant Extract/Herbal

                          Upfront Cash : Inapplicable

                          March 19, 2025

                          Lead Product(s) : CAS 501-94-0

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          07

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : Apitegromab is a Antibody drug candidate, which is currently being evaluated in clinical studies for the treatment of Muscular Atrophy, Spinal.

                          Product Name : Undisclosed

                          Product Type : Antibody, Unconjugated

                          Upfront Cash : Inapplicable

                          March 14, 2025

                          Lead Product(s) : Apitegromab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          08

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : Plozasiran is a Oligonucleotide drug candidate, which is currently being evaluated in clinical studies for the treatment of Hyperlipoproteinemia Type I.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          January 28, 2025

                          Lead Product(s) : Plozasiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          09

                          Genecombio Ltd.

                          Country arrow
                          OTS 2025
                          Not Confirmed

                          Genecombio Ltd.

                          Country arrow
                          OTS 2025
                          Not Confirmed

                          Lead Product(s) : GCB-001

                          Therapeutic Area : Genetic Disease

                          Study Phase : Undisclosed

                          Sponsor : The Children's Hospital of Zhejiang University School of Medicine

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : GCB-001 is a drug candidate, which is currently being evaluated in clinical studies for the treatment of Spinal Muscular Atrophies of Childhood.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          January 13, 2025

                          Lead Product(s) : GCB-001

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : The Children's Hospital of Zhejiang University School of Medicine

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          10

                          Centre Hospitalier de Saint-Denis

                          Country arrow
                          OTS 2025
                          Not Confirmed

                          Centre Hospitalier de Saint-Denis

                          Country arrow
                          OTS 2025
                          Not Confirmed

                          Details : This drug candidate is currently being evaluated in clinical studies for the treatment of Anemia, Sickle Cell.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          December 16, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : INSERM U955

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank