LV-IGF2.GAAco

**LV-IGF2.GAAco** is an investigational lentiviral gene therapy for Pompe disease. The therapy uses a lentiviral vector to deliver a construct encoding a codon-optimized acid alpha-glucosidase enzyme (GAA) fused to an insulin-like growth factor 2 (IGF2) peptide. This IGF2 tag is designed to enhance cellular uptake of GAA via the cation-independent mannose 6-phosphate/IGF2 receptor, improving effectiveness in tissues including heart, skeletal muscle, and especially the central nervous system, compared to untagged GAA gene therapy. In preclinical models, LV-IGF2.GAAco has demonstrated near-complete correction of pathological features of Pompe disease at lower vector copy numbers than previous approaches, normalizing glycogen levels and cellular protein homeostasis in affected tissues[1][2][3][5].