Genethon, located in Evry, France, is a non-profit R&D organization dedicated to the design and development of gene therapy treatments for rare genetic diseases from research to clinical validation. It was created by the AFM-Telethon (French Muscular Dystrophy Association) which is its main funding source. Genethon has multiple ongoing programs at clinical, preclinical and research stage for neuromuscular, blood, immune system, and liver diseases. Several of these programs are pursued by Genethon as sponsor, others have been licensed to leading biotech and pharma players in the gene therapy arena such as Audentes, Avexis/Novartis, Gensight Biologics, Orchard Therapeutics, Spark. Genethon was awarded the “Prix Galien France” in 2012.