AB-1009

AB-1009 is an investigational adeno-associated virus (AAV) gene therapy being developed for the treatment of Pompe disease, a rare and progressive genetic metabolic disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). The therapy is designed to deliver a functional copy of the *GAA* gene to the patient's cells, allowing for the endogenous production of the enzyme needed to break down lysosomal glycogen in cardiac and skeletal muscles. Developed by AskBio (a subsidiary of Bayer), AB-1009 incorporates licensed technology from Genethon, including specific patented components such as liver-specific promoters or expression cassettes intended to optimize transgene expression and potentially induce immune tolerance to the replacement enzyme.