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.4
4 Urayama, A.; Grubb, J. H.; Sly, W. S.; Banks, W. A. Mannose 6-phosphate receptor-mediated transport of sulfamidase across the blood−brain barrier in the newborn mouse. Mol. Ther. 2008, 16, 1261−1266.