Researchers are conducting a clinical trial of AGT-182, an investigational treatment for people with Hunter Syndrome aka mucopolysaccharidosis II or MPS 2.
AGT-182 is an enzyme replacement therapy (ERT) designed to treat both the body-related (somatic) and central nervous system (CNS) symptoms and complications of Hunter syndrome.
Currently approved treatments for Hunter syndrome (or MPS 2) are unable to treat the brain (CNS) because of the enzyme’s inability to cross the blood-brain barrier (BBB). The BBB is a filter that protects the brain from toxins but allows vital nutrients like insulin to cross from the blood into the brain. As a result, available therapies for MPS disease do not address many of the severe and progressive neurological complications of the Hunter Syndrome. AGT-182 is designed to cross the BBB in the same way insulin does.