AAVhu68.vIGF2.hGAA

AAVhu68.vIGF2.hGAA is an investigational gene therapy consisting of an **adeno-associated virus serotype hu68 (AAVhu68)** vector that encodes a **human acid alpha-glucosidase (hGAA)** transgene engineered with a **variant insulin-like growth factor 2 peptide tag (vIGF2)**. The vIGF2 tag enhances selective binding to the IGF2/cation-independent mannose-6-phosphate receptor (CI-MPR) to promote uptake in target tissues (notably muscle and neurons), while reducing off-target binding and minimizing hypoglycemia risk. The strategy is to deliver the vector systemically or via combined intravenous and intrathecal routes for treating the neuromuscular and CNS components of **Pompe disease**. Preclinical studies in non-human primates have shown immune-mediated toxicities, including myocarditis, primarily attributed to T-cell responses directed at the hGAA transgene[1][3][5].

Other names

AAVhu68-vIGF2-hGAAAAVhu-68-vIGF2-hGAAAAVhu 68-vIGF2-hGAA