AGT‑184 is an investigational enzyme replacement therapy (ERT) for the treatment of Sanfilippo A syndrome, a lysosomal storage disease that arises from a deficiency in the gene encoding for the enzyme N-sulphoglucosamine sulphohydrolase (SGSH), which results in a buildup of complex sugars. Currently, there are no approved therapies for the treatment of Sanfilippo A syndrome.
Unlike recombinant SGSH, AGT-184 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, Pardridge WM (2014) Insulin Receptor Antibody-Sulfamidase Fusion Protein Penetrates the Primate Blood-Brain Barrier and Reduces Glycosoaminoglycans in Sanfilippo Type A Cells. Mol Pharm 11:2928-34. 2014