Following an initial research and development collaboration on an innovative gene therapy product using micro-dystrophin, Genethon and Sarepta Therapeutics are now started a co-development clinical program for Duchenne muscular dystrophy.
Gene therapy combining an AAV-type viral vector and a shortened version of the dystrophin gene (microdystrophin) is an innovative technology developed by Genethon researchers in collaboration with the team of Prof. George Dickson (University of London). This innovative technology has demonstrated significant efficacy in preclinical works with high expression of the protein and significant restoration of muscle function with stabilization of clinical symptoms. Under the terms of the licensing agreement, Genethon will be responsible for commercializing the product GNT0004 in Europe (excluding the UK) and Sarepta will be responsible in the rest of the world.
“This cutting-edge technology is the result of years of research and development based on the expertise of Genethon researchers, pioneers in the field of gene therapy for rare diseases. Combining this promising approach, which targets the majority of Duchenne patients, with the know-how of our partner Sarepta is a new opportunity for patients. We have just started a clinical study of pre-inclusion of patients (“baseline” study) to allow us to precisely evaluate the efficacy of the product. This is a very concrete step towards the injection of the first patient with the product in the coming months,” says Frédéric Revah, CEO of Genethon.
The production of clinical batches and, eventually, commercial batches of this gene therapy, based on innovative suspension-based production methods, has been entrusted to YposKesi, one of the largest European sites for GMP vector production, with 160 bio-production experts and a dedicated site of 5,000 m².