HDAd.GAA

HDAd.GAA is a helper-dependent adenoviral (HDAd) vector-based gene therapy candidate designed for the treatment of Pompe disease (glycogen storage disease type II). It functions by delivering a functional copy of the GAA gene, which encodes the enzyme acid α-glucosidase, responsible for breaking down glycogen in lysosomes. The vector is used to transduce cells, such as hematopoietic stem and progenitor cells (HSPCs) or fibroblasts, to induce the expression and secretion of the GAA enzyme. This approach aims to achieve systemic glycogen clearance and ameliorate symptoms in multiple tissues, including the heart and central nervous system. In research settings, it serves as a benchmark for engineered versions like HDAd.CD63-GAA, which utilizes extracellular vesicles for enhanced delivery.

Other names

helper-dependent adenoviral vector expressing acid alpha-glucosidase