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Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Ligand Pharmaceuticals","sponsor":"Palvella Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2020","type":"Agreement","leadProduct":"Sirolimus","moa":"FK506-binding protein 1A","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ligand Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical","sponsorNew":"Ligand Pharmaceuticals \/ Palvella Therapeutics","highestDevelopmentStatusID":"9","companyTruncated":"Ligand Pharmaceuticals \/ Palvella Therapeutics"},{"orgOrder":0,"company":"Electra Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"ELA026","moa":"SIRP-alpha","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Electra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Electra Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Electra Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Glycomine \/ Inapplicable"},{"orgOrder":0,"company":"Bambino Ges\u00f9 Hospital and Research Institute","sponsor":"University of Exeter | Radboud University Medical Center | Consorzio per Valutazioni Biologiche e Farmacologiche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Posaconazole","moa":"Cytochrome P450 51","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Bambino Ges\u00f9 Hospital and Research Institute","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bambino Ges\u00f9 Hospital and Research Institute \/ University of Exeter | Radboud University Medical Center | Consorzio per Valutazioni Biologiche e Farmacologiche","highestDevelopmentStatusID":"9","companyTruncated":"Bambino Ges\u00f9 Hospital and Research Institute \/ University of Exeter | Radboud University Medical Center | Consorzio per Valutazioni Biologiche e Farmacologiche"},{"orgOrder":0,"company":"Assistance Publique \u2013 H\u00f4pitaux de Paris","sponsor":"INSERM U1163","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Famotidine","moa":"Histamine H2 receptor","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Assistance Publique \u2013 H\u00f4pitaux de Paris","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ INSERM U1163","highestDevelopmentStatusID":"9","companyTruncated":"Assistance Publique \u2013 H\u00f4pitaux de Paris \/ INSERM U1163"},{"orgOrder":0,"company":"OnKure Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"REN001","moa":"PARP delta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"OnKure Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"OnKure Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"OnKure Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Indiana University","sponsor":"Global Health Uganda | Makerere University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Zinc Sulphate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Indiana University","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Indiana University \/ Global Health Uganda | Makerere University","highestDevelopmentStatusID":"9","companyTruncated":"Indiana University \/ Global Health Uganda | Makerere University"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Govorestat","moa":"enzyme inhibitors: aldose-reductase inhibitors","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Applied Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Applied Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Camurus","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Peptide","year":"2022","type":"Inapplicable","leadProduct":"CAM2029","moa":"SST receptor","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Camurus","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Camurus \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Camurus \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"TAK-672","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"rAAV2tYF-GRK1-hRPGRco","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beacon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Generium","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"GNR-038","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Generium \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Generium \/ Inapplicable"},{"orgOrder":0,"company":"Generium","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"GNR-055","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Lyophilized Powder","sponsorNew":"Generium \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Generium \/ Inapplicable"},{"orgOrder":0,"company":"PT Prodia StemCell Indonesia","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"Umbilical Cord Mesenchymal Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"PT Prodia StemCell Indonesia","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PT Prodia StemCell Indonesia \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"PT Prodia StemCell Indonesia \/ Inapplicable"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AMO Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2024","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"PT Prodia StemCell Indonesia","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Umbilical Cord Mesenchymal Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"PT Prodia StemCell Indonesia","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PT Prodia StemCell Indonesia \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"PT Prodia StemCell Indonesia \/ Inapplicable"},{"orgOrder":0,"company":"Electra Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"ELA026","moa":"SIRP-alpha","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Electra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Electra Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Electra Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Electra Therapeutics","sponsor":"Westlake Village BioPartners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2022","type":"Series B Financing","leadProduct":"ELA026","moa":"SIRP-alpha","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Electra Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Electra Therapeutics \/ Westlake Village BioPartners","highestDevelopmentStatusID":"9","companyTruncated":"Electra Therapeutics \/ Westlake Village BioPartners"},{"orgOrder":0,"company":"Generium","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"GNR-038","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Generium \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Generium \/ Inapplicable"}]

Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : The STAGED-PKD study was stopped for futility following an independent analysis of the annualized rate of change in total kidney volume (TKV) in patients receiving venglustat compared to placebo.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 06, 2021

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          May 14, 2018

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : The proceeds will advance GLM101, a first-in-class mannose-1-phosphate replacement therapy, is in development for phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG).

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          April 16, 2025

                          Lead Product(s) : GLM101

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : CTI Life Sciences Fund

                          Deal Size : $115.0 million

                          Deal Type : Series C Financing

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                          04

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : DNL310 (tividenofusp alfa) is composed of the iduronate 2-sulfatase enzyme fused to Denali’s proprietary ETV, designed to deliver IDS, addressing symptoms of Hunter syndrome.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          April 02, 2025

                          Lead Product(s) : Tividenofusp Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          March 24, 2025

                          Lead Product(s) : GLM101

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : RGX-202 is a cell and gene therapy drug that works by transferring the micro-dystrophin gene, It is infused intravenously for the treatment of Duchenne Muscular Dystrophy.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 19, 2025

                          Lead Product(s) : RGX-202

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 12, 2025

                          Lead Product(s) : Infigratinib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 12, 2025

                          Lead Product(s) : SKY-0515

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          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 : Undisclosed

                          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

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                          10

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : DNL310 (tividenofusp alfa), a fusion protein composed of IDS fused to Denali’s proprietary Enzyme transport vehicle. It is being evaluated in the treatment of Mucopolysaccharidosis Type II.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          February 06, 2025

                          Lead Product(s) : Tividenofusp Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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