Genethon’s Duchenne Muscular Dystrophy Gene Therapy GNT0004 Produces Expression of Microdystrophin in Phase 1/2/3 Trial

Genethon reported positive results from its AAV-microdystrophin treatment trial in Europe.  The three participants receiving the study’s higher dose (3×1013vg/kg) showed that an average of 54% of muscle fibers were producing microdystrophin, and individuals demonstrated a reduced level of creatine kinase, a marker of muscle damage.  Genethon said it is planning a pivotal trial in Europe. 

Genethon’s GNT0004, an investigational recombinant adeno-associated virus (AAV) vector-based gene therapy intended to treated Duchenne muscular dystrophy (DMD), has demonstrated the ability to produce expression of microdystrophin, among other signs of efficacy, in a phase 1/2/3 clinical trial.1,2 The data were presented by Francesco Muntoni, MD, the principal investigator of the trial and the chair of paediatric neurology at University College London Great Ormond Street Institute of Child Health, at the Myology 2024 International Scientific Congress, held April 22 to 25, in Paris, France. READ FULL NEWS RELEASE HERE

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