AGT‑187 is an investigational enzyme replacement therapy (ERT) for the treatment of Sanfilippo B syndrome, a lysosomal storage disease that arises from a deficiency in the gene encoding for the enzyme α-N-Acetylglucosaminidase (NAGLU), which results in a buildup of complex sugars. Currently, there are no approved therapies for the treatment of Sanfilippo B syndrome.
Unlike recombinant NAGLU, AGT-187 is able to cross the BBB and has shown enzyme activity comparable to recombinant SGSH in animal studies.1
1 Boado RJ, Lu JZ, Hui EK, Lin H, Pardridge WM (2016) Insulin Receptor Antibody-α-N-Acetylglucosaminidase Fusion Protein Penetrates the Primate Blood-Brain Barrier and Reduces Glycosoaminoglycans in Sanfilippo Type B Fibroblasts. Mol Pharm. 13:1385-92.