AAV9-DES-coGAA

AAV9-DES-coGAA is an investigational **gene therapy** product in which a recombinant adeno-associated virus serotype 9 (AAV9) vector delivers a human codon-optimized acid alpha-glucosidase (coGAA) gene, driven by a human desmin (DES) promoter, to target cardiac and skeletal muscle tissue. The therapy is being developed for the treatment of Pompe disease, a lysosomal storage disorder caused by GAA deficiency that leads to glycogen accumulation in muscles. The vector promotes expression of functional GAA enzyme in affected tissues, aiming to correct the underlying enzyme deficiency, reduce pathology, and improve muscle function[1][2][3][4]. Immune modulation strategies (use of rituximab and sirolimus) are typically used in trials to counter immune responses that can arise due to vector or transgene exposure[1][3]. Clinical studies focus on both safety and efficacy, with development status currently at the phase I trial stage for late-onset Pompe disease[3][4].

Other names

rAAV9-DES-hGAArAAV-9-DES-hGAArAAV 9-DES-hGAA