AX-552

AX-552 is an investigational enzyme replacement therapy (ERT) being developed by Allievex Corp. for the treatment of GM1 gangliosidosis, a severe and progressive neurodegenerative lysosomal storage disease. This condition is caused by autosomal recessive mutations in the GLB1 gene, leading to a deficiency in lysosomal β-galactosidase (β-gal) and the accumulation of galactose-containing substrates. AX-552 aims to replace the deficient heparan sulfate sulfatase and β-galactosidase enzymes, thereby addressing the underlying enzymatic defect and reducing substrate accumulation. Preclinical studies have shown that a single intracerebroventricularly (ICV) administered dose of recombinant human β-gal (rhβ-gal), which is the active component of AX-552, resulted in broad biodistribution to critical brain regions in a mouse model of GM1 gangliosidosis, leading to reduced ganglioside and oligosaccharide substrates and reversal of neuropathology.