AGT-184

AGT-184 is an investigational enzyme replacement therapy (ERT) developed by ArmaGen for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo Syndrome A. It is a recombinant fusion protein consisting of a human monoclonal antibody against the insulin receptor fused to the lysosomal enzyme N-sulfoglucosamine sulfohydrolase (SGSH). The antibody domain acts as a molecular "Trojan horse," binding to the endogenous insulin receptor on the blood-brain barrier to facilitate receptor-mediated transcytosis of the fusion protein into the central nervous system. Once in the brain, the SGSH enzyme payload degrades heparan sulfate, addressing the underlying enzyme deficiency that leads to progressive neurodegeneration and intellectual disability in MPS IIIA patients.

Other names

HIRMAb-SGSHIgG-SGSH fusion proteinanti-insulin receptor antibody-SGSH fusion protein