FDAdhGAA

FDAdhGAA is a gene therapy vector designed to treat Glycogen Storage Disease type II (GSD-II). It is a fully deleted adenovirus (FDAd) vector engineered to express human acid alpha-glucosidase (hGAA) using nonviral regulatory elements, specifically the PEPCK promoter and ApoE enhancer. The therapy aims to correct abnormal glycogen storage in cardiac and skeletal muscles by providing long-term hepatic secretion of hGAA, thereby reversing glycogen accumulation and preserving muscle strength. Preclinical studies in GSD-II mice have shown its ability to achieve long-term efficacy and reduce anti-hGAA antibody responses compared to other gene therapy approaches.