Atamyo Therapeutics and the Dion Foundation for Rare Diseases have partnered to conduct a clinical trial in the United States for ATA-200, a gene therapy for gamma-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5).
ATA-200, an Adeno-Associated Virus (AAV) vector, carries the human gamma-sarcoglycan transgene. The Phase 1b trial will assess the safety, efficacy, and immunogenicity of ATA-200 in children with the rare genetic disease.
LGMD2C/R5 is caused by mutations in the gamma-sarcoglycan gene, leading to progressive muscular weakness and cardiac issues. Approximately 2,000 individuals in Europe are affected. Currently, there is no cure for LGMD2C/R5, with treatment focusing on supportive measures.
The Dion Foundation will fund the US portion of the trial, which has already received regulatory approval in France and Italy. Atamyo is working on filing an Investigational New Drug (IND) application for ATA-200 in the US.
Atamyo, a spin-off from Genethon, is dedicated to developing gene therapies for muscular dystrophies and cardiomyopathies. Genethon CEO Frederic Revah highlights this partnership as a significant milestone in their efforts to bring gene therapies to patients.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
