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AveXis Enters into Licensing Agreement with Genethon

AveXis, Inc. (NASDAQ:AVXS) and Genethon today announced they have entered into an exclusive, worldwide license agreement for in vivo gene therapy delivery of AAV9 vector into the central nervous system (CNS) for the treatment of spinal muscular atrophy (SMA).

Genethon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler-Najjar Syndrome

Genethon, a laboratory created by AFM-Telethon, starts a European phase I/II clinical trial to test a treatment for Crigler-Najjar Syndrome, a rare liver disease. The trial, sponsored by Généthon, will include 17 patients in four centres in Europe, and will assess safety and therapeutic efficacy of the gene therapy product developed by Genethon. Read the … [Read more]

First promising results towards the treatment of myotubular myopathy

Genethon welcomes interim data of a phase I / II clinical trial with a gene therapy product developed in its laboratories. At the 12-week timepoint, the first 3 children treated show early signs of efficacy. Read the press release

A gene therapy alliance in X-linked chronic granulomatous disease with Orchard Therapeutics

Orchard Therapeutics (“Orchard”), a clinical-stage biotechnology company dedicated to transforming the lives of patients with rare disorders through innovative gene therapies announces today a strategic alliance with Généthon, to develop gene therapy for X-linked chronic granulomatous disease (“X-CGD”). Généthon is a non-profit research and development organization whose mission is to develop gene therapies for orphan … [Read more]

Audentes Therapeutics announces dosing of first patient in ASPIRO for the treatment of X-Linked Myotubular Myopathy

Preliminary data from ASPIRO expected to be available in the fourth quarter of 2017 Audentes Therapeutics, abiotechnology company focused on developing and commercializing gene therapy products for patients  living with serious, life-threatening  rare diseases, today announced it has commenced dosing of patients in ASPIRO, a Phase 1/2 clinical trial of AT132 for the treatment of … [Read more]

Spark Therapeutics Enters into Licensing Agreement with Genethon

Spark Therapeutics, a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, today announced it has entered into a licensing agreement with Genethon, a non-profit research and development organization dedicated to the development of gene therapies for orphan genetic diseases from research to clinical validation, for the development and commercialization of … [Read more]

Restore muscle strength in Duchenne muscular dystrophy through microdystrophin

Gene therapy:  Microdystrophin restores muscle strength in Duchenne muscular dystrophy Researchers from Genethon, the AFM-Téléthon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal  Holloway) demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy. Indeed, after injecting microdystrophin (a “shortened” version of the dystrophin gene) via a drug … [Read more]

New partnership for the treatment of Duchenne muscular disease (DMD)

Sarepta Therapeutics, a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, and Genethon, have signed a gene therapy research collaboration to jointly develop treatments for Duchenne muscular dystrophy (DMD). Genethon’s micro-dystrophin gene therapy approach can target the majority of patients with DMD. … [Read more]

New step towards the treatment of myotubular myopathy: gene therapy restores strength and prolongs lives in affected dogs

Evry (France), 5 April, 2017. A team of researchers in France, led by Dr. Ana Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, achieved a new step towards the treatment of myotubular myopathy by gene therapy. The researchers demonstrated the efficacy of administration of a therapeutic … [Read more]

Creation of YposKesi

AFM-Téléthon and the SPI fund, managed by Bpifrance under the Programme d’Investissement d’Avenir, are creating YposKesi, the first French industrial pharmaceutical company dedicated to producing gene and cell therapy drugs for rare  diseases.