ATMY-003

ATMY-003 is an investigational adeno-associated virus (AAV) vector-based gene therapy designed for the treatment of Limb-Girdle Muscular Dystrophy Type 2D (LGMD2D), also known as LGMD R3 alpha-sarcoglycan-related. Developed by Atamyo Therapeutics, a spin-off from the non-profit research organization Genethon, the therapy utilizes an AAV9 vector to deliver a functional copy of the human SGCA gene to muscle cells. LGMD2D is caused by mutations in the SGCA gene, which leads to a deficiency in the alpha-sarcoglycan protein, a critical component of the sarcoglycan complex that maintains the structural integrity of muscle cell membranes. By restoring alpha-sarcoglycan expression in skeletal and cardiac muscles, ATMY-003 aims to stabilize muscle fibers, prevent progressive muscle wasting, and improve motor function in patients with this rare genetic disorder.

Other names

AAV9-hSGCAAAV-9-hSGCAAAV 9-hSGCA