Genethon announces dosing of first patient at pediatric clinical trial platform for neuromuscular diseases

A first participant was dosed at I-Motion, the pediatric clinical trial platform for neuromuscular disease at Trousseau Hospital in Paris, as part of the gene therapy study in Duchenne muscular dystrophy (DMD) conducted by Genethon.

A young boy suffering from Duchenne muscular dystrophy received a first dose of the experimental gene therapy GNT 0004 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 multicentre study sponsored by Genethon. The trial was approved in France by the French National Agency for the Safety of Medicines and Health Products (ANSM) and in the UK by the UK Regulatory Authority for Medicines and Healthcare Products.

Administering this first patient is a step that is highly symbolic of Genethon. This trial is the result of 30 years of groundbreaking research by Genethon. It embodies the quality of the research carried out 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 today become drug candidates that could change the future for patients suffering from Duchenne muscular dystrophy. “

Frédéric Revah, CEO

“There remains a tremendous unmet need for treatments to help people with DMD. We have high hopes for this new candidate,” said Prof. Muntoni of the Dubowitz Neuromuscular Center (UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital (UCL)). London)., UK)), the lead investigator for the trial. There is no cure for Duchenne muscular dystrophy, and developing an effective treatment is extremely challenging. Duchenne muscular dystrophy was a cornerstone of the fight led by AFM-Téléthon and Genethon.

The gene therapy (GNT 0004) is based on an adeno-associated virus (AAV) capsid and an optimized gene, a shortened version of the gene coding for dystrophin, the protein absent in patients with Duchenne muscular dystrophy. This microdystrophin, associated with a vector designed to be expressed in muscle tissue, was developed by Genethon in collaboration with the teams of Prof. Dickson (University of London, Royal Holloway) and the Institute of Myology (Paris). It is now being co-developed with Sarepta Therapeutics in the clinical phase.

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