Atamyo OK’d for Gene Therapy Trial

FDA has approved an Atamyo Therapeutics IND for a Phase 1b/2b clinical trial involving ATA-200, a one-time gene therapy for treating γ-sarcoglycan-related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5). The serious and debilitating condition is said to affect children and leads to loss of ambulation before adulthood. The company says its open-label, dose-escalation study (NCT05973630) will evaluate safety, pharmacodynamics, efficacy, and immunogenicity in children receiving intravenous ATA-200, a single-dose adeno-associated virus vector carrying the human γ-sarcoglycan transgene.

LGMD-2C/R5 is described as a rare genetic disease caused by mutations in the gene that produces γ-sarcoglycan, a transmembrane protein that is involved in the connection between muscle fibers and their environment. About 2,000 people are affected by the disorder in the U.S. and Europe.