Genethon receives the 2012 French Galien Prize, in the category “medicines destined for rare diseases – cell therapy and gene therapy.” This is the first time that the Galien, one of the most prestigious scientific awards in the world – eight Nobel laureates are members of the jury – awards a laboratory created and developed by a patient association.
“This award confirms the undisputed role of Genethon as world leader in the field of gene therapy. It recognises our major contribution to medical progress in general and more particularly to rare diseases,” said Frédéric Revah, Managing Director of Genethon.
Created in 1990 by the AFM-Telethon, and 90% financed by Telethon donations, Genethon develops gene therapy treatments for rare diseases, from their design through to production and through their preclinical and clinical development.
Major advances
“I would like to dedicate this award to Telethon donors and partners who have supported Genethon since its creation and have enabled its development,” added Laurence Tiennot-Herment, President of the AFM-Telethon and Genethon. “Without them this tremendous adventure would not have been possible. Thanks to their unfailing support, we have addressed the most audacious challenges, from the first human genome maps to the production of gene therapy treatments today.”
Since the 1990s, Genethon has been renowned for accomplishing the first maps of the human genome. A major discovery that was immediately made available to the international scientific community. The 200 experts at Genethon are also responsible for major breakthroughs such as AAV exon skipping, the first gene therapy trial for a neuromuscular disease (gamma-sarcoglycanopathy), the first multicentre, transatlantic (France/England/USA), gene therapy trial for an immunodeficiency.
Focus on production
Genethon has set a new challenge: produce gene therapy treatments for rare diseases. In 2011, Genethon-Bioprod was created, the world’s largest centre for the bioproduction of clinical grade gene therapy products. The centre is required to conduct human trials.
