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    [{"orgOrder":0,"company":"Genethon","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Genethon strengthens its collaboration with Sarepta Therapeutics for the development of the gene therapy product GNT0004 in Duchenne muscular dystrophy","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Musculoskeletal","graph2":"Preclinical"},{"orgOrder":0,"company":"Genethon","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II"}]

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              Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease

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              GNT-0003 is being developing a gene therapy treatment to treat patients with severe forms of CN syndrome by restoring expression of the UGT1A1 enzyme in the patient’s liver.

              Lead Product(s): GNT-003

              Therapeutic Area: Rare Diseases and Disorders Product Name: GNT-003

              Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable January 10, 2023

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              Genethon strengthens its collaboration with Sarepta Therapeutics for the development of the gene therapy product GNT0004 in Duchenne muscular dystrophy

              Details:

              CDMO’s capacity to produce AAV materials at large-scale will support Genethon and Sarepta’s project to take micro-dystrophin to clinical trials this year.

              Lead Product(s): GNT0004

              Therapeutic Area: Musculoskeletal Product Name: Undisclosed

              Highest Development Status: Preclinical Product Type: Undisclosed

              Partner/Sponsor/Collaborator: Sarepta Therapeutics

              Deal Size: Undisclosed Upfront Cash: Undisclosed

              Deal Type: Collaboration January 09, 2020

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