Genethon, a global leader in gene therapy for rare genetic diseases, has received the green light from the UK and France’s top regulatory bodies EMA and MHRA to begin phase 3 clinical trials for its experimental gene therapy, GNT0004, targeting Duchenne muscular dystrophy (DMD).
This milestone brings Genethon closer to developing a new treatment that could make a significant impact on the global healthcare landscape.
Become a valued research partner with us – https://www.towardshealthcare.com/schedule-meeting
What the Trial Involves
This upcoming phase 3 trial builds on the strong results from earlier phase 1/2 studies, where GNT0004 showed promising outcomes in improving motor function, reducing muscle damage, and increasing microdystrophin expression. Patients in the earlier trials either maintained or showed improved movement, along with a sustained drop in creatine phosphokinase (CPK) a key marker of muscle damage.
Starting in August and September, the phase 3 double-blind trial will be conducted in France and the UK. The treatment involves a single intravenous dose of GNT0004, which contains a modified version of the dystrophin gene (Hmd1 transgene) delivered using an AAV8 vector. A special promoter, Spc5-12, helps the therapy target muscle and heart tissue. The trial will include around 64 boys aged 6 to 10 who still have the ability to walk.
What Genethon Says
“We’re proud to move forward with this pivotal trial,” said Frédéric Revah, CEO of Genethon. “Bringing GNT0004 closer to approval is a meaningful step for the children and families affected by DMD. Our journey began in 2021, and the early results have been extremely encouraging.”
He also noted that the selected dose for this trial is lower than what’s used in other gene therapy trials for DMD yet it’s shown strong results. “The confidence shown by regulators in approving phase 3 trials is a testament to the strength of GNT0004 and the dedication of our team,” Revah added.
Request a customized case study tailored to your business needs and gain deeper insights into healthcare market strategies: sales@towardshealthcare.com
