Genethon announces dosing of first patient on pediatric neuromuscular disease platform

The first participant was dosed with I-Motion, a platform for pediatric clinical trials of neuromuscular diseases located at the Trousseau Hospital in Paris, as part of a gene therapy trial of Duchenne muscular dystrophy (DMD) conducted by Genethon.

A boy suffering from Duchenne muscular dystrophy received the first dose of the GNT 0004 gene therapy tested at I-Motion, a pediatric platform for clinical trials of neuromuscular diseases in Paris. He was the first patient in the international phase I / II / III of the multicenter trial sponsored by Genethon. The experiment was approved in France by the French National Agency for the Safety of Medicines and Health Products (ANSM), and in the UK by the British Regulatory Agency for Medicines and Health Products.

Dosing this first patient is a step that is deeply symbolic for Genethon. This trial is the culmination of 30 years of Genethon’s pioneering research. It embodies the quality of research conducted in our laboratories, in collaboration with international high-performance teams. Duchenne is a very challenging disease and although we are cautious, we hope and are proud that the technologies developed at Genethon today are becoming candidates for drugs that could change the future for patients suffering from Duchenne muscular dystrophy. “

Frédéric Revah, CEO

“There remains a huge unmet need for treatments to help people affected by DMD. We have high hopes for this new candidate,” says Prof. Muntoni of the Neuromuscular Center Dubowitz (Institute of Child Health UCL Great Ormond Street and Great Ormond Street Hospital (London, UK)), chief trial investigator. There is no cure for Duchenne muscular dystrophy, and the development of an effective treatment is extremely demanding. Duchenne’s muscular dystrophy was the cornerstone of the struggle waged by AFM-Téléthon and Genethon.

Gene therapy (GNT 0004) is based on the aden-associated virus capsid (AAV) and the optimized gene, an abbreviated version of the gene encoding dystrophin, a protein not found in patients with Duchenne muscular dystrophy. This micro-dystrophin, associated with a vector designed to be expressed in muscle tissue, 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 being developed together in the clinical phase with Sarepta Therapeutics.