AAV-TCAP gene therapy

Cure Rare Disease is developing an adeno-associated virus (AAV) vector-based gene replacement therapy for the treatment of Limb-Girdle Muscular Dystrophy type 2G (LGMD2G). LGMD2G is an ultra-rare, autosomal recessive muscular dystrophy caused by mutations in the *TCAP* gene, which encodes the telethonin protein. Telethonin is a critical component of the muscle sarcomere, interacting with titin to maintain structural integrity and stability. The therapy utilizes an AAV vector to deliver a functional copy of the *TCAP* gene to muscle cells, aiming to restore telethonin expression and stabilize muscle structure to prevent progressive atrophy. The program is currently in the preclinical stage of development.

Other names

Cure Rare Disease TCAP gene therapytelethonin gene therapyTCAP gene replacement therapy