Created in 1990 and funded almost exclusively by the AFM-Telethon, Genethon is a major player in gene therapy for rare diseases.
Genethon’s mission is to provide patients with breakthrough treatments for gene therapy. Having played a pioneering role in decoding the human genome (publication of the 1st human genome maps between 1992 and 1996), Genethon is today, with more than 200 scientists, physicians, engineers, regulatory affairs specialists, one of the major international centres for research and preclinical and clinical development of gene therapy for rare diseases.
Notably, Genethon is the sponsor of an international clinical trial in progress concerning Wiskott-Aldrich syndrome (immunodeficiency). Several trials are in preparation for neuromuscular diseases and rare diseases of the eyes, the immune system, the blood, the liver… Genethon also has a bioproduction centre allowing it to manufacture batches of pharmaceutical grade vectors for clinical trials: Genethon Bioprod, which has the largest production capacity of gene therapy treatments in the world.
- Research and development: design of therapeutic approaches and preclinical evaluation of gene therapy products for rare diseases; development of administration methods and optimisation of vectors; development of innovative technologies for bioproduction and control of gene therapy products; evaluation and control of the immune response; search for biomarkers; imaging and histology;
- Clinical trials: large-scale production of batches of drug candidates to GMP standards (Genethon Bioprod) design, development and conduct of clinical trials (neuromuscular diseases, immunodeficiencies, vision disorders…); development of regulatory dossiers.