Genethon, is a pioneer and a leader in the field of gene therapy for rare genetic diseases. 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 Avexis/Novartis (to which Genethon licensed the founding patents for Zolgensma), Audentes, Gensight Biologics, Orchard Therapeutics, Spark.

Genethon has around 200 scientists and experts in its R&D center in Evry (France), covering R&D, bioprocess development, clinical and regulatory.

Genethon is a non-profit organization created in 1990 by the AFM-Telethon (French Muscular Dystrophy Association) which is its main funding source.