Genethon is dedicated to design and develop a gene therapy products for rare diseases. On Monday 30th of November it received the authorisation from the ANSM, the French National Agency for Medicines and Health Products Safety, to start a clinical trial for the treatment of Duchenne muscular dystrophy with product GNT 004. This multicentre international clinical trial starts in France.
This trial’s goal is to assess the safety and efficacy of an innovative gene therapy designed at Généthon. It combines an AAV-type viral vector with a shortened version of the dystrophin gene, “microdystrophin”.
About the technology
The innovative technology used for this clinical trial is GNT 0004, a gene therapy combining an AAV-type viral vector and a shortened version of the dystrophin gene, microdystrophin, designed by researchers and experts at Généthon, in collaboration with Prof. George Dickson’s team (University of London). Following demonstrated safety and efficacy in preclinical animal studies (Nature Communications, July 2017), Généthon and Sarepta Therapeutics, the leader in precision gene medicine for rare diseases, announced a collaboration to complete preclinical development, then to co-develop the clinical programme.