ATMY-002

ATMY-002 is an adeno-associated virus (AAV) vector-based gene therapy designed for the treatment of Limb-Girdle Muscular Dystrophy Type 2C (LGMD2C), also known as LGMD R5 (gamma-sarcoglycanopathy). LGMD2C is a rare, progressive autosomal recessive genetic disorder caused by mutations in the *SGCG* gene, which encodes the gamma-sarcoglycan protein. This protein is a vital component of the sarcoglycan complex within the dystrophin-associated glycoprotein complex, which maintains the structural integrity of muscle cell membranes. ATMY-002 utilizes an AAV9 capsid to deliver a functional human *SGCG* transgene under the control of a muscle-specific promoter, aiming to restore gamma-sarcoglycan expression in skeletal and cardiac muscles. The therapy was originally developed by researchers at Genethon and is currently being advanced by Atamyo Therapeutics.