The international gene therapy trial for Duchenne Muscular Dystrophy (DMD) using GNT0004 has unveiled promising findings at the Myology 2024 conference. The trial, led by Professor Francesco Muntoni, combines phases I to III with a dose escalation stage, followed by a pivotal phase.
The trial has enrolled ambulant boys aged 6 to 10 with DMD and has received approval from French and UK authorities. Five patients have been administered GNT0004, demonstrating good tolerability and immunological response.
Initial efficacy results for the patient receiving the higher dose (3×10^13 vg/kg) after one year showed clinical improvement, including an inflection in the North Star Ambulatory Assessment score. Other functional assessments also displayed positive trends.
The safety and efficacy data have enabled Genethon, the trial’s sponsor, to prepare the pivotal European phase with the European Medicines Agency (EMA).
GNT0004 is an AAV8 gene therapy containing a shortened and functional version of the DMD gene that encodes dystrophin, the deficient protein in DMD. It targets key tissues like skeletal and cardiac muscles through a single intravenous injection.
DMD is a rare genetic disease that affects muscle function. Its absence leads to muscle degeneration and complications such as cardiomyopathy and loss of mobility. The trial’s progress represents a significant step towards potential therapeutic options for DMD.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
