AAVMYO3-GAA

AAVMYO3-GAA is a **muscle-targeted gene therapy** designed for the treatment of **Pompe disease**. It employs an engineered adeno-associated virus (AAV) serotype called **AAVMYO3** to deliver a codon-optimized **human acid alpha-glucosidase (GAA)** gene, controlled by a muscle-specific promoter and enhancer elements. This approach enables highly efficient and specific transduction of skeletal and cardiac muscle tissues while detargeting the liver, minimizing the risk of hepatic toxicity. Systemic administration in animal models restores GAA expression in muscle, normalizes glycogen metabolism, corrects muscle pathology, improves muscle mass and strength, and prevents cardiac enlargement. AAVMYO3-GAA achieves increased GAA activity in target tissues to levels exceeding wild-type controls, with minimal off-target expression.