Genethon, created by the AFM-Telethon, has the mission to make innovative gene therapy treatments available to patients affected with rare genetic diseases. To meet this challenge Genethon has assembled the technical and human resources needed to accelerate the medical application of scientific discoveries arising from fundamental research. Strong translational research programs engage multi-disciplinary teams and are supported by a first rate technological platform and cGMP facility
The pipeline of Genethon includes products currently in international clinical trials and at preclinical stages, for immune deficiencies, muscular dystrophies, ocular and liver diseases. These products are developed either with Genethon as sponsor, or in partnership with private companies and academic institutions.