AAV-CBhGAA

AAV-CBhGAA is an investigational adeno-associated virus (AAV)-mediated gene therapy that uses an AAV vector to deliver the **acid alpha-glucosidase (GAA) gene** under the control of a CBh promoter. The therapy is designed as a gene replacement strategy for **Pompe disease (glycogen storage disease type II)**, a rare inherited neuromuscular disorder caused by mutations in the GAA gene, leading to GAA enzyme deficiency and lysosomal glycogen accumulation. The CBh promoter is used to drive robust, sustained expression of the therapeutic GAA enzyme in target tissues, aiming at long-term correction of the enzyme deficiency and disease symptoms. This approach falls under **gene therapy** and is typically delivered by **intravenous injection** to target muscle and/or cardiac tissue. The therapy is currently at the **preclinical or early clinical development stage**.

Other names

AAV2-GAAAAV-2-GAAAAV 2-GAAAAV2/2-GAA