AGT-182

AGT-182 is an investigational enzyme replacement therapy (ERT) designed for the treatment of Hunter syndrome (mucopolysaccharidosis type II, MPS II), a rare, severe lysosomal storage disorder. The drug is engineered as a fusion protein combining the iduronate 2-sulfatase (IDS) enzyme with a human insulin receptor monoclonal antibody. This design enables the therapeutic enzyme to cross the blood-brain barrier by targeting insulin receptors on endothelial cells, potentially addressing both somatic and neurological symptoms of MPS II. AGT-182 has completed Phase 1 clinical trials and has received orphan drug designation from the FDA for this indication[1][2][3][4][5].

Other names

Recombinant human insulin receptor monoclonal antibody-fused iduronate 2-sulfatase