AAV-LSPGAApA

AAV-LSPGAApA is an adeno-associated virus (AAV) vector-based gene therapy candidate designed to deliver a functional copy of the **GAA gene** for the treatment of **Pompe disease** (glycogen storage disease type II). Pompe disease is caused by mutations in the acid alpha-glucosidase (GAA) gene, leading to GAA enzyme deficiency, excessive glycogen accumulation in muscle and other tissues, and resulting in progressive muscle weakness and cardiorespiratory dysfunction. AAV-LSPGAApA uses a liver-specific promoter (LSP) to drive GAA gene expression, aiming to induce sustained production of the GAA enzyme, potentially offering a one-time therapeutic alternative to chronic enzyme replacement therapy (ERT). The therapy is delivered using an AAV vector, most likely an AAV serotype designed for efficient liver transduction. The primary developer appears to be Asklepios BioPharmaceuticals (AskBio). The treatment is intended for systemic administration, most likely via intravenous route, to achieve systemic distribution and liver-targeted gene delivery, supporting continuous endogenous GAA production.