STX-GAA

STX-GAA is a preclinical-stage, muscle-targeting extracellular vesicle (EV) therapeutic being developed by Capricor Therapeutics for the treatment of Pompe disease. Utilizing Capricor's proprietary Stealth-X (STX) exosome platform, the candidate is engineered with a muscle-targeting moiety on the EV surface and carries functional acid alpha-glucosidase (GAA) protein. This targeted delivery system is designed to improve uptake in skeletal and cardiac muscle, allowing for therapeutic efficacy at lower doses compared to conventional enzyme replacement therapies (ERTs). In preclinical models of Pompe disease (GAA-knockout mice), systemic administration of STX-GAA demonstrated enhanced muscle delivery and partially restored lysosomal GAA activity.