AAV5-GAA

AAV5-GAA is an investigational gene therapy consisting of an adeno-associated virus serotype 5 (AAV5) vector encoding the human acid alpha-glucosidase (**GAA**) gene. It is designed to restore GAA enzyme activity in target tissues of patients with **Pompe disease**, a genetic disorder characterized by deficiency of GAA, leading to glycogen accumulation and progressive muscle dysfunction. In animal models, spinal delivery of AAV5-GAA restored GAA enzyme activity in the spinal cord and decreased glycogen accumulation, improving respiratory deficits associated with neuromuscular impairment[4]. The therapy acts by delivering a functional GAA gene to the central nervous system (CNS) or other target tissues, facilitating endogenous production of the enzyme and clearance of pathogenic glycogen.

Other names

AAV5-GAAAAV-5-GAAAAV 5-GAA