AGT‑183 is an investigational enzyme replacement therapy (ERT) for the treatment of metachromatic leukodystrophy (MLD), a lysosomal storage disease that arises from a deficiency of the enzyme arylsulfatase (ASA).

Although recombinant ASA has been developed, it is not effective against MLD because it does not cross the BBB. AGT-183, a form of human ASA, has shown the ability to cross the BBB and demonstrated comparable enzyme activity to recombinant ASA in animal studies.1


1 Boado MJ, et al. Pharmacokinetics and brain uptake in the rhesus monkey of a fusion protein of arylsulfatase A and a monoclonal antibody against the human insulin receptor. Biotechnol Bioeng. 2013; 110(5): 1456–1465.