Genethon
As a pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a non-profit laboratory that was established by AFM-Telethon. A first gene therapy, for spinal muscular atrophy, to which Genethon contributed was approved worldwide. With more than 200 scientists and professional staff, Genethon is pursuing its aim to develop innovative therapies which change the lives of patients suffering from rare genetic diseases. Thirteen products stemming from Genethon’s R&D or from collaborations are at clinical trial stages for diseases of the liver, blood, immune system, muscles, and eyes. Seven other products could enter clinical trials over the next five years.