Our therapies

The teams at Genethon develop gene therapy products for rare diseases, in collaboration with academic and industrial teams in France and abroad. Several products derived from R&D at Genethon or to which Genethon contributed as part of a collaboration, are currently in the clinical trial stage. These trials are sponsored by Genethon or by partners in Europe or the United States. These therapies involve neuromuscular pathologies, immune system diseases, ocular and metabolic diseases. To date, 10 products derived from research conducted at Genethon are in clinical trials and 8 others should be in clinical trial within the next five years.