ATA-001

ATA-001 (also known as GNT-015-SGCG) is an investigational gene therapy designed for the treatment of limb-girdle muscular dystrophy type R5 (LGMD R5), formerly known as LGMD2C. This condition is a rare, progressive autosomal recessive muscle-wasting disease caused by mutations in the *SGCG* gene, which encodes the gamma-sarcoglycan protein. ATA-001 utilizes a non-replicating adeno-associated virus serotype 8 (AAV8) vector to deliver a functional copy of the human gamma-sarcoglycan gene specifically to muscle cells. By restoring the expression of the gamma-sarcoglycan protein, the therapy aims to stabilize the dystrophin-associated glycoprotein complex (DAGC) at the sarcolemma, thereby preventing muscle fiber degeneration and improving motor function. The drug was originally developed by Genethon and is being advanced clinically by Atamyo Therapeutics.

Other names

adeno-associated virus serotype 8 expressing human gamma-sarcoglycan geneAAV8-hSGCGAAV-8-hSGCGAAV 8-hSGCGrAAV8-hSGCGrAAV-8-hSGCGrAAV 8-hSGCG