ATA-100 (Atamyo Therapeutics)

ATA-100 (rAAV8-hSGCG) is a recombinant adeno-associated virus serotype 8 (AAV8) vector-based gene therapy developed by Atamyo Therapeutics for the treatment of limb-girdle muscular dystrophy type R5 (LGMD R5), also known as gamma-sarcoglycanopathy. LGMD R5 is a rare, progressive genetic muscle-wasting disease caused by mutations in the SGCG gene, which encodes the gamma-sarcoglycan protein. This protein is a critical component of the dystrophin-associated glycoprotein complex (DAGC) that maintains the structural integrity of muscle cell membranes. ATA-100 delivers a functional copy of the human SGCG gene specifically to skeletal and cardiac muscle cells using a muscle-specific promoter. By restoring gamma-sarcoglycan expression, the therapy aims to stabilize the muscle cell membrane, prevent further muscle degeneration, and improve motor function in affected patients.

Other names

AAV8-hSGCGAAV-8-hSGCGAAV 8-hSGCGgamma-sarcoglycan gene therapy