Programs

GM1 gangliosidosis is a rare and fatal pediatric lysosomal storage disorder (LSD) that progressively destroys neurons in the central nervous system and leads to a host of systemic manifestations. The underlying causes of GM1 gangliosidosis are mutations in the gene for beta-galactosidase (GLB1), a key enzyme responsible for the breakdown of toxic molecules known as gangliosides throughout the body, which results in rapidly progressing neurodegeneration. Despite an understanding of the underlying cause of the disorder and the systemic manifestations of the disease in multiple organs, there are currently no approved treatment options available for these children.

AXO-AAV-GM1 is the only IV-administered gene therapy validated through extensive preclinical studies in naturally occurring animal models. AXO-AAV-GM1’s systemic route of administration has the potential to restore beta-galactosidase function to the entire body, thereby halting disease progression in its tracks. AXO-AAV-GM1 is currently the most advanced GM1 therapy and has the potential to be the first approved treatment for this devastating disease. It is also the only gene therapy in development for both Type 1 and Type 2 forms of GM1 gangliosidosis. The U.S. Food & Drug Administration has granted Orphan Drug Designation and Rare Pediatric Disease Designation for AXO-AAV-GM1 for the treatment of GM1 gangliosidosis.