A young boy suffering from Duchenne muscular dystrophy received a first dose of the investigational gene therapy GNT0004 at I-Motion, the pediatric clinical trial platform for neuromuscular diseases in Paris. He was the first patient in an international phase I/II/III multicenter trial for which Genethon is the sponsor. The trial has been approved in France by the French National Agency for Medicines and Health Products Safety (ANSM) and in the UK by British Medicines & Healthcare products Regulatory Agency.
The gene therapy is based on an optimized gene, a shortened version of the gene coding for dystrophin, the protein that is absent in patients with Duchenne muscular dystrophy. This micro-dystrophin, associated with a vector designed to be expressed in muscle tissues, was developed by Genethon, in partnership with the teams of Prof. Dickson (University of London, Royal Holloway) and the Institute of Myology (Paris). It is now developed jointly in the clinical phase with Sarepta Therapeutics.
“Dosing this first patient is a step that is profoundly symbolic for Genethon. This trial is the culmination of 30 years of pioneering research by Genethon. It embodies the quality of the research conducted in our laboratories, in collaboration with high-performing international teams. Duchenne is a very challenging disease and while we are cautious, we are hopeful and proud that the technologies developed at Genethon are today becoming drug candidates that could change the future for patients suffering from Duchenne muscular dystrophy” explains CEO Frédéric Revah.
“There remains a tremendous unmet need for treatments to help individuals affected by DMD. We put high hopes in this novel candidate ” says Prof. Muntoni from the Dubowitz Neuromuscular Center (UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital (London, UK)), the principal investigator for the trial, which will include patients from France, UK, USA and Israël.
Duchenne muscular dystrophy is a rare progressive genetic disease that affects all the muscles in the body, including the heart muscle, and affects 1 in 3,500 boys. There is no cure for Duchenne muscular dystrophy, and the development of an effective treatment is exceptionally challenging.