[{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Agreement","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Royalty Pharma","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Royalty Pharma"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Spinal Muscular Atrophy Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Partnership","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Spinal Muscular Atrophy Foundation","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Spinal Muscular Atrophy Foundation"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Acquisition","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Royalty Pharma","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Royalty Pharma"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Collaboration","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Licensing Agreement","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Deflazacort","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Eladocagene Exuparvovec","moa":"AADC","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intraputaminal Infusion","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Utreloxastat","moa":"15-LOX","graph1":"Neurology","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Divestment","leadProduct":"Eladocagene Exuparvovec","moa":"AADC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0.14999999999999999,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14999999999999999,"dosageForm":"Intraputaminal Infusion","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Eladocagene Exuparvovec","moa":"AADC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Eladocagene Exuparvovec","moa":"AADC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"ClearPoint Neuro","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Eladocagene Exuparvovec","moa":"AADC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"PTC Therapeutics \/ ClearPoint Neuro","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ ClearPoint Neuro"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Eladocagene Exuparvovec","moa":"AADC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Eladocagene Exuparvovec","moa":"AADC","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intraperitoneal Infusion","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Eladocagene Exuparvovec","moa":"AADC","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intraperitoneal Infusion","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Eladocagene Exuparvovec","moa":"AADC","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intraperitoneal Infusion","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Emvododstat","moa":"DHODH","graph1":"Infections and Infectious Diseases","graph2":"Phase II\/ Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Infectious Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Emvododstat","moa":"DHODH","graph1":"Infections and Infectious Diseases","graph2":"Phase II\/ Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Infectious Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Emvododstat","moa":"DHODH","graph1":"Infections and Infectious Diseases","graph2":"Phase II\/ Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Infectious Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Ataluren","moa":"80s ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"15-LOX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"15-LOX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"15-LOX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"15-LOX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Licensing Agreement","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Unesbulin","moa":"Tubulin","graph1":"Oncology","graph2":"Phase I","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Unesbulin","moa":"Tubulin","graph1":"Oncology","graph2":"Phase I","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Large Molecule","year":"2022","type":"Inapplicable","leadProduct":"Volanesorsen","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"15-LOX","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Blackstone Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Collaboration","leadProduct":"Ataluren","moa":"80s ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"PTC Therapeutics \/ Blackstone Life Sciences","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Blackstone Life Sciences"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Ataluren","moa":"80s ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Ataluren","moa":"80s ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Ataluren","moa":"80s ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Ataluren","moa":"80s ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Ataluren","moa":"80s ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Ataluren","moa":"80s ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Ataluren","moa":"80s ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Ataluren","moa":"80s ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"NICE","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Ataluren","moa":"80s ribosome","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"PTC Therapeutics \/ NICE","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ NICE"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Ataluren","moa":"80s ribosome","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Utreloxastat","moa":"15-LOX","graph1":"Neurology","graph2":"Phase I","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Powder","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Powder","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Powder","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Powder","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Powder","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"}]

Find Clinical Drug Pipeline Developments & Deals by PTC Therapeutics

Menu
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

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Sephience (sepiapterin) is an oral formulation of sepiapterin, has a dual mechanism of action to increase activity of the phenylalanine hydroxylase enzyme. It is being evaluated for Phenylketonuria.

                          Product Name : Sephience

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          April 25, 2025

                          Lead Product(s) : Sepiapterin

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          02

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : PTC923 (sepiapterin) is an oral formulation, that has a dual moa to increase the activity of the PAH enzyme. It is being evaluated for the treatment of pediatric and adult phenylketonuria patients.

                          Product Name : PTC923

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 20, 2025

                          Lead Product(s) : Sepiapterin

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          03

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : PTC743 (vatiquinone) is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), which is being developed for the treatment of Friedreich ataxia.

                          Product Name : PTC743

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 19, 2025

                          Lead Product(s) : Vatiquinone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          04

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : PTC743 (vatiquinone) is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), which is being developed for the treatment of Friedreich ataxia.

                          Product Name : PTC743

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          December 19, 2024

                          Lead Product(s) : Vatiquinone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          05

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : PTC was granted PRV along with the FDA approval of Kebilidi (eladocagene exuparvovec) for the treatment of children and adults with AADC deficiency, including the full spectrum of disease severity.

                          Product Name : Kebilidi

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          November 27, 2024

                          Lead Product(s) : Eladocagene Exuparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : $150.0 million

                          Deal Type : Divestment

                          blank

                          06

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : PTC857 (utreloxastat) is being evaluated in the mid-stage clinical trial studies for the treatment of amyotrophic lateral sclerosis.

                          Product Name : PTC857

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          November 26, 2024

                          Lead Product(s) : Utreloxastat

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          07

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Kebilidi (eladocagene exuparvovec) is a gene therapy medicinal product that expresses the human aromatic L-amino acid decarboxylase enzyme (hAADC).

                          Product Name : Upstaza

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          November 14, 2024

                          Lead Product(s) : Eladocagene Exuparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          08

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Upstaza (eladocagene exuparvovec) is a gene therapy indicated for the treatment of aromatic L-amino acid decarboxylase enzyme (hAADC) deficiency in children and adults.

                          Product Name : Upstaza

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          November 13, 2024

                          Lead Product(s) : Eladocagene Exuparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          09

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Translarna (ataluren) is a dystrophin expression stimulator small molecule drug candidate, being evaluated for duchenne muscular dystrophy from a nonsense mutated dystrophin gene in males.

                          Product Name : Translarna

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          October 30, 2024

                          Lead Product(s) : Ataluren

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          10

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Translarna (ataluren) is a dystrophin expression stimulator small molecule drug candidate, indicated for the treatment of nonsense mutated duchenne muscular dystrophy , in patients aged 2 or above.

                          Product Name : Translarna

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          October 18, 2024

                          Lead Product(s) : Ataluren

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank