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]
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, 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]
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]
SpinrazaTM (nusinersen), the first and only treatment for spinal muscular atrophy (SMA), is now available in Europe. Biogen has just announced that it has obtained marketing authorisation for SpinrazaTM (nusinersen) in Europe. This is the first drug for the treatment of SMA. Now, every European country will have to evaluate the value of this drug … [Read more]
New step towards the treatment of myotubular myopathy: gene therapy restores strength and prolongs lives in affected dogs
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 vector by a single intravenous … [Read more]
The 30th Telethon edition reaches a final collection of 92,740,769 euros On 2nd and 3rd December 2016, the Telethon enthralled the public throughout France. The final collection figure now allows us to pursue the objective we wet ourselves 30 years ago : to find a cure ! Thank you !!!!