AAVrh10-CAG-hGAA

AAVrh10-CAG-hGAA is an experimental gene therapy vector based on the recombinant adeno-associated virus serotype rh10 (AAVrh10) engineered to deliver the human acid alpha-glucosidase (hGAA) gene under the control of the CAG promoter. It is primarily investigated for the treatment of Pompe disease (glycogen storage disease type II). The aim is to address the deficiency of the GAA enzyme in affected tissues, thereby reducing glycogen accumulation and improving muscle and central nervous system (CNS) function. In preclinical models (notably GAA knockout mice), a single intrathecal administration of AAVrh10-CAG-hGAA resulted in substantial reductions of glycogen storage throughout the CNS, improvement in motor coordination and muscle strength, and decreased cardiomyopathy. This vector is being explored for its ability to enable widespread and durable GAA gene expression in the CNS with the potential for clinical application in Pompe disease[3][4].