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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]

SMA: European marketing authorisation for SpinrazaTM (nusinersen)

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]

92,740,769 euros

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 !!!!

Prize4Life Announces $1 Million ALS Treatment Prize Winner

Prize4Life, a nonprofit organization whose mission is to accelerate the discovery of treatments and a cure for ALS (Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s disease), today announced the winner of its $1M Avi Kremer ALS Treatment Prize. The winners are the team of Drs. Martine Barkats and Maria-Grazia Biferi, from the Institute of … [Read more]

Call for proposals on remyelination projects

ARSEP Foundation and AFM-Téléthon will provide significant support (€ 300,000) for 2 to 3 scientific projects of high caliber for one year. Applications will be evaluated on their international competiveness and the originality of the proposed work. Research projects from basic science to clinical research will be considered, provided they have primarily focused on the … [Read more]