Généthon

Généthon is a not-for-profit research centre that was created by the French Muscular Dystrophy Association (AFM) and is now funded almost exclusively by donations from France's annual Telethon. Its goal is to deliver gene therapies to patients with rare diseases in general and neuromuscular diseases in particular. With over 200 scientists, physicians, engineers and regulatory affairs specialists and after having played a pioneering role in the global effort to decipher the human genome, Généthon is now a world-leading centre for preclinical and clinical research and development in the field of gene therapy. Généthon also has a biomanufacturing platform for clinical-grade vectors and is involved in building the world's largest facility for pre-industrial pilot production.