The results of the gene therapy trial (GNT0004) conducted by Généthon for Duchenne muscular dystrophy were presented on 17 May at the 2025 conference of the American Society of Gene & Cell Therapy (ASGCT). They demonstrate stabilisation of motor function and a significant and sustained reduction in CPK levels in patients treated at the effective dose.
The gene therapy product was developed by Généthon, in collaboration with teams led by Professor Dickson (University of London, Royal Holloway) and the Institute of Myology. The trial combined phases I/II/III and was conducted in France (I-Motion, Paris) and the United Kingdom in boys aged 6 to 10 with Duchenne muscular dystrophy who were able to walk.
Following these positive results, Genethon has just obtained authorisation from the European Medicines Agency (EMA), with France (ANSM) as the rapporteur 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 double-blind phase, which will involve 64 boys aged 6 to 10 with Duchenne muscular dystrophy who have retained their ability to walk.
Read Genethon press release Genethon to Launch Pivotal Trial in Europe of GNT0004 a Low-Dose Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
