Genethon doses first patient in clinical trial of gene therapy for DMD

The first dose of the investigational gene therapy GNT 0004 was given for Dunchenne Muscular Dystrophy (DMD) at I-Motion, the paediatric clinical trial platform for neuromuscular diseases in Paris.

The young boy was the first patient in an international phase I/II/III multicentre trial, sponsored by Genethon. 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.

Genethon CEO Frédéric Revah said: “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”.

“There remains a tremendous unmet need for treatments to help individuals affected by DMD. We put high hopes in this novel candidate” said Professor Muntoni from the Dubowitz Neuromuscular Centre of UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, the principal investigator for the trial.

There is currently no cure for DMD and the development of an effective treatment is challenging. The gene therapy is based on an adeno-associated virus (AAV) capsid and an optimised gene, a shortened version of the gene coding for dystrophin, the protein that is absent in patients with DMD. This micro-dystrophin is associated with a vector designed to be expressed in muscle tissues.

This phase I/II/III gene therapy trial is followed by the randomised efficacy part of the trial, to assess the product’s efficacy versus placebo. The cross-over is planned after the one year following the treatment with placebo to allow all participants to potentially benefit from the treatment. The trial uses a single intravenous injection of GNT 0004. The trial aims to enrol boys aged six to ten suffering from DMD who are still able to walk.

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