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Disease","country":"GERMANY","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"BAY 3401016","moa":"SEMA3A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Macupatide","moa":"GIPR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eli Lilly \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eli Lilly \/ Undisclosed"},{"orgOrder":0,"company":"The Emmes Company, LLC","sponsor":"Rachel G. Greenberg, MD, MB, MHS","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Guanfacine","moa":"Adrenergic receptor alpha-2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"The Emmes Company, LLC","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"The Emmes Company, LLC \/ Rachel G. Greenberg, MD, MB, MHS","highestDevelopmentStatusID":"8","companyTruncated":"The Emmes Company, LLC \/ Rachel G. Greenberg, MD, MB, MHS"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Scott R. Plotkin, MD, PhD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Brigatinib","moa":"ALK tyrosine kinase receptor | Epidermal growth factor receptor erbB1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Takeda Pharmaceutical \/ Scott R. Plotkin, MD, PhD","highestDevelopmentStatusID":"8","companyTruncated":"Takeda Pharmaceutical \/ Scott R. Plotkin, MD, PhD"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Region Skane","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Trametinib","moa":"Dual specificity mitogen-activated protein kinase kinase; MEK1\/2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Region Skane","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Region Skane"},{"orgOrder":0,"company":"Healx","sponsor":"Craig Erickson","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Gaboxadol","moa":"GABA-A receptor; agonist GABA site","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Healx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Healx \/ Craig Erickson","highestDevelopmentStatusID":"8","companyTruncated":"Healx \/ Craig Erickson"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Jerry Vockley","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Acer Therapeutics \/ Jerry Vockley","highestDevelopmentStatusID":"8","companyTruncated":"Acer Therapeutics \/ Jerry Vockley"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Jerry Vockley","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zevra Therapeutics \/ Jerry Vockley","highestDevelopmentStatusID":"8","companyTruncated":"Zevra Therapeutics \/ Jerry Vockley"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Michael Campos, MD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Alpha 1-Antitrypsin","moa":"Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Michael Campos, MD","highestDevelopmentStatusID":"8","companyTruncated":"CSL Behring \/ Michael Campos, MD"},{"orgOrder":0,"company":"Dompe Farmaceutici","sponsor":"Ospedale San Raffaele","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Recombinant Human Nerve Growth Factor","moa":"Neurotrophic tyrosine kinase receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Dompe Farmaceutici","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Dompe Farmaceutici \/ Ospedale San Raffaele","highestDevelopmentStatusID":"8","companyTruncated":"Dompe Farmaceutici \/ Ospedale San Raffaele"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"John Mark Sloan","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Idelalisib","moa":"PI3-kinase p110-delta subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gilead Sciences \/ John Mark Sloan","highestDevelopmentStatusID":"8","companyTruncated":"Gilead Sciences \/ John Mark Sloan"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Romain Lazor","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Nintedanib Esylate","moa":"Platelet-derived growth factor receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Romain Lazor","highestDevelopmentStatusID":"8","companyTruncated":"Boehringer Ingelheim GmbH \/ Romain Lazor"},{"orgOrder":0,"company":"Genentech","sponsor":"Jesse Roman","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Pirfenidone","moa":"TNF","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Genentech \/ Jesse Roman","highestDevelopmentStatusID":"8","companyTruncated":"Genentech \/ Jesse Roman"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Jerry Vockley","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Jerry Vockley","highestDevelopmentStatusID":"8","companyTruncated":"Ultragenyx Pharmaceutical \/ Jerry Vockley"},{"orgOrder":0,"company":"Incyte Corporation","sponsor":"Aaron Logan","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Ruxolitinib Phosphate","moa":"Tyrosine-protein kinase JAK1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Incyte Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Incyte Corporation \/ Aaron Logan","highestDevelopmentStatusID":"8","companyTruncated":"Incyte Corporation \/ Aaron Logan"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"David Williams","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Autologous CD34+ Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ David Williams","highestDevelopmentStatusID":"8","companyTruncated":"Bluebird Bio \/ David Williams"}]
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