Genethon Launches Phase 3 Trial in Europe for Gene Therapy to Treat Duchenne Muscular Dystrophy

Genethon's New Trial in Europe for Duchenne Muscular Dystrophy

Announcement

Genethon, a global prominent leader in research and development in gene therapy for rare genetic diseases, received approval from regulatory authorities, EMA and MHRA, for crucial phase 3 clinical trials in the UK and France of its gene therapy ‘GNT0004’ for Duchenne muscular dystrophy (DMD). The new phase 3 clinical trial will take Genethon near to the new drug development that will lead to an addition in the global healthcare market.

Phase 3 clinical trial

The authorizations for the phase 3 trial in Europe are based on the positive results of phase ½ studies that highlighted good tolerance and efficacy of GNT0004 regarding microdystrophin expression, motor function, and creatine phosphokinase (CPK) diminisher. The patients stabilized or improved interaction to the clinical trial of motor functions, and a valuable persistent reduction in CPK is a crucial marker of muscle damage. The phase 3 double blind trials will start in August and September in France and the UK using one intravenous injection of GNT0004. The injection consists of an enhanced Hmd1 transgene, a short (3*10 square 13 vg/kg microdystrophin). But the functional version of the gene encoding dystrophin in an AAV8 vector was induced with transient immunological prophylactic treatment. The vector is introduced to highlight itself in the heart and muscle tissue. The big thanks to the Spc5-12 promoter sequence mainly for these tissues. Around 64 boys aged between 6 to 10 years with DMD that has retained their walking action will be fit for enrollment.

Views and Statements

CEO of Genethon, Frederic Revah, expressed, “We are glad to be able to consistently work through these trials, and the dedication to introduce GNT0004 to the market for young patients and their families seeking a solution is the biggest honor for us. This symbolizes our valuable step towards our gene therapy program for DMD that began in 2021 and achieved commendable results in the first children treated in the phase 1/2 portion of our phase 1/2/3 study.”

Revah further added, “Witnessing the positive results in patients treated during the early phases is one of the powers of our product in the dose selected for the crucial phase. The dose is less than that used in different gene therapy trials for DMD. The phase 3 trials approvals complement the regulatory authorities' confidence and trust in GNT0004 and the accomplishment of our team's work.”