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Duchenne Muscular Dystrophy : First child dosed in Clinical Trial of Investigational Gene therapy

A first patient suffering from Duchenne muscular dystrophy received a first dose of the investigational gene therapy GNT0004 in an international phase I/II/III multicener trial for which Genethon is the sponsor. More information on the gene therapy clinical trial lead by Genethon.

A Genethon team has succeeded in inhibiting the immune response linked to AAV

A research team from Genethon, in collaboration with teams from CNRS/Inserm and from the biotechnology company Spark Therapeutics, announced in Nature Medicine that it has succeeded in inhibiting the immune response induced by AAV antibodies present as a result of natural immunity or following gene therapy, thanks to the IdeS enzyme. This result opens up … [Read more]

Treatment of X-CGD : positive results of the gene therapy initiated by Genethon

Genethon, AFM-Telethon’s laboratory, at the origin of the gene therapy for this rare immunodeficiency disorder, welcomes the very encouraging results announced by Orchard Therapeutics, a biopharmaceutical company. February 25, 2019 : Orchard Therapeutics presents clinical proof-of-concept data for OTL-102, a gene therapy for the treatment of X-CGD. Patients with this rare immune inherited immunodeficiency disorder … [Read more]

Interview with Frédéric Revah, CEO of Généthon

Frédéric Révah, CEO of Généthon and president of Yposkesi, explains the role of Généthon in the discovery of innovative therapies since the 1990s and the recent launch of YposKesi, its innovative manufacturing platform. Read the interview.

Co-administration of AAV Vectors with SVP-Rapamycin enables vector re-administration in pre-clinical gene therapy

Généthon, founded by the AFM-Téléthon, and Selecta Biosciences, a clinical-stage biopharmaceutical company, today announced that Nature Communications has published their jointly authored paper entitled “Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration”. A world first ! The pre-clinical study led by Genethon demonstrated that co-administration of synthetic vaccine particles encapsulating … [Read more]

Clinical trial to test a gene therapy treatment for a rare liver disease

Généthon starts a European phase I/II clinical trial to test a treatment for Crigler-Najjar Syndrome, a rare liver disease. This European trial will include 17 patients over 10 years old, (the age when the liver reaches maturity); it aims to assess the tolerance of the product, determine the optimal dose, and assess the therapeutic efficacy … [Read more]

Audentes Therapeutics announces dosing of first patient, in a phase 1/2 clinical trial for the treatment of X-linked myotubular myopathy

Audentes Therapeutics, a biotechnology company focused on developing and commercializing gene therapy products, today announced it has commenced dosing of patients in a Phase 1 / 2 clinical trial of AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM). This trial is a multicenter, multinational, open-label, ascending dose study to evaluate the safety and preliminary … [Read more]

Spark Therapeutics and Genethon announce collaboration

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, for the development and commercialization of an adeno-associated viral (AAV) gene therapy targeting the liver to address a rare genetic disease. Spark Therapeutics has the option to develop … [Read more]

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 vector, the researchers managed to restore muscle strength and … [Read more]

Sarepta Therapeutics and Genethon Announce Gene Therapy Research Collaboration for Duchenne Muscular Disease (DMD) Therapies

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]