AAV9-DES-IGFIIcoGAA

AAV9-DES-IGFIIcoGAA is an investigational **gene therapy** vector designed to treat Pompe disease by delivering a codon-optimized human acid α-glucosidase (GAA) gene fused to an insulin-like growth factor II (IGFII) glycosylation-independent lysosomal targeting (GILT) tag, under the control of the human desmin (DES) promoter, via an adeno-associated virus serotype 9 (AAV9) vector. The IGFII GILT tag enhances cellular uptake and lysosomal delivery of the GAA enzyme through high-affinity binding to mannose-6-phosphate/IGFII receptors, thus improving cross-correction in muscle and neuronal tissue. This approach increases GAA expression and activity—clearing glycogen accumulation and improving muscle and hypoglossal motor neuron pathology in Pompe disease models—more effectively than untargeted GAA constructs. Intramuscular and intralingual administration have both been evaluated in preclinical studies, with ongoing clinical evaluation for late-onset Pompe disease[1][7].

Other names

AAV9-DES-IGF2coGAAAAV-9-DES-IGF2coGAAAAV 9-DES-IGF2coGAAAAV9-DES-GILT-GAAAAV-9-DES-GILT-GAAAAV 9-DES-GILT-GAA