Duchenne Muscular Dystrophy: Start of the pivotal phase of Genethon's gene therapy trial

Building on the positive results of the dose escalation phase, which demonstrated the safety and efficacy of the second dose tested, Genethon has just obtained authorization from the European Medicines Agency (EMA) (with France (ANSM) as the reporting member state) and the United Kingdom (MHRA) to start the pivotal phase of the trial.

The first patients will be enrolled in August and September in the United Kingdom and France for this pivotal phase, conducted as a double-blind trial, which will involve 64 boys aged 6 to 10 with Duchenne muscular dystrophy who have retained their ability to walk. 

Genethon CEO Frederic Revah observed, “This marks a decisive step forward for our gene therapy program for DMD, which began in 2021 and has demonstrated extremely promising results in the first children treated in the Phase1/2 portion of our Phase1/2/3 study.” 
Dr. Revah added, “In addition to the very positive results in patients treated in the early phases, one of the strengths of our product is the dose selected for the pivotal phase, which is lower than those used in other gene therapy trials for DMD. Approvals of our Phase 3 trials reflect the regulatory authorities’ confidence in GNT0004 as well as the work accomplished by our teams.”

AFM-Téléthon, committed to fighting this complex neuromuscular disease, which is the most common neuromuscular disease in children and the reason for its creation, is delighted with the start of the pivotal phase in Europe.

Sacha, 9, is one of the children who received the treatment developed by Généthon as part of the first phase of the trial using microdystrophin, which aims to restore the production of the missing protein in the muscles. Today, Sacha runs, climbs, jumps... movements he would have been unable to do without treatment! Discover his story (English subtitles available).