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(SGSH)||Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Lysogene \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Undisclosed"},{"orgOrder":0,"company":"Lysogene","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase 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Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"RO7204239","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"F. 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Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"PT Prodia StemCell Indonesia","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDONESIA","productType":"Cell & 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 \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"PT Prodia StemCell Indonesia \/ 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Bio","highestDevelopmentStatusID":"9","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Ultevursen","moa":"usherin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Laboratoires Thea","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Laboratoires Thea \/ Sepul Bio","highestDevelopmentStatusID":"9","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"QR-421a","moa":"Usherin messenger RNA (USH2A 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Berry-Kravis","highestDevelopmentStatusID":"9","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Elizabeth Berry-Kravis"}]

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

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                          01

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II/ Phase III

                          Sponsor : Undisclosed

                          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 : Miscellaneous

                          Upfront Cash : Inapplicable

                          June 01, 2021

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Venglustat is a Other Small Molecule drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Polycystic Kidney, Autosomal Dominant.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          May 14, 2018

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          SupplySide West
                          Not Confirmed
                          SupplySide West
                          Not Confirmed

                          Details : Atumelnant is a Small Molecule drug candidate drug candidate, which is currently being evaluated in Phase II/ Phase III clinical studies for the treatment of Adrenal Hyperplasia, Congenital.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          September 08, 2025

                          Lead Product(s) : Atumelnant

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          SupplySide West
                          Not Confirmed
                          SupplySide West
                          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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          SupplySide West
                          Not Confirmed
                          SupplySide West
                          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 : Miscellaneous

                          Upfront Cash : Inapplicable

                          March 19, 2025

                          Lead Product(s) : RGX-202

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          SupplySide West
                          Not Confirmed
                          SupplySide West
                          Not Confirmed

                          Details : Infigratinib is a Other Small Molecule drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Hypochondroplasia.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          March 12, 2025

                          Lead Product(s) : Infigratinib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          SupplySide West
                          Not Confirmed
                          SupplySide West
                          Not Confirmed

                          Details : SKY-0515 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Huntington Disease.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          March 12, 2025

                          Lead Product(s) : SKY-0515

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          SupplySide West
                          Not Confirmed
                          SupplySide West
                          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 : Miscellaneous

                          Upfront Cash : Inapplicable

                          February 19, 2025

                          Lead Product(s) : Vatiquinone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          SupplySide West
                          Not Confirmed
                          SupplySide West
                          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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          SupplySide West
                          Not Confirmed
                          SupplySide West
                          Not Confirmed

                          Details : AGTC-501 (laruparetigene zovaparvovec) is an XLRP GTPase regulator cell & gene therapy candidate, which is being evauated for the treatment of patients suffering from X-linked retinitis Pigmentosa.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          January 28, 2025

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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