GNT0004

GNT0004 is an investigational gene therapy designed to treat Duchenne Muscular Dystrophy (DMD). It uses an adeno-associated virus serotype 8 (AAV8) vector to deliver a shortened but functional microdystrophin gene (hMD1 transgene) into muscle and heart tissue. The therapy aims to restore dystrophin protein expression, which is deficient in DMD patients, thereby improving muscle function and reducing muscle damage. Administered as a single intravenous infusion, GNT0004 has demonstrated good safety and tolerability with transient immunological prophylactic treatment. Clinical trials have shown significant microdystrophin expression in muscle fibers, reduction of creatine phosphokinase (CPK) levels—a biomarker of muscle damage—and stabilization or improvement in motor function measured by the North Star Ambulatory Assessment (NSAA). The dose used in pivotal trials is lower than doses used in other gene therapies for DMD. Developed by Genethon with academic partners including University of London and Institut de Myologie, the ongoing Phase 1/2/3 trial includes ambulant boys aged 6 to 10 years[1][4][5][7][8].

Other names

AAV8-microdystrophinAAV-8-microdystrophinAAV 8-microdystrophinGenethon micro-dystrophin