AXO-AAV-GM2

AXO-AAV-GM2 is an investigational gene therapy designed to treat GM2 gangliosidosis, which includes Tay-Sachs disease and Sandhoff disease. These are rare, fatal pediatric neurodegenerative disorders caused by mutations in the HEXA (Tay-Sachs) or HEXB (Sandhoff) genes, leading to deficiency of the β-hexosaminidase A enzyme. AXO-AAV-GM2 delivers functional copies of both the HEXA and HEXB genes using two co-administered adeno-associated virus serotype rh8 (AAVrh8) vectors directly into the central nervous system. This approach aims to restore HexA enzyme activity, slow or halt neurodegeneration, and potentially improve clinical outcomes for affected children. The therapy has received Orphan Drug and Rare Pediatric Disease Designation from the FDA and is currently being evaluated in phase 1/2 clinical trials[1][3][5][7].