valanafusp alpha

**Valanafusp alpha (AGT-181)** is a recombinant fusion protein developed by ArmaGen Technologies as an enzyme replacement therapy (ERT) for mucopolysaccharidosis type I (MPSI, Hurler syndrome). It consists of human α-L-iduronidase (IDUA) fused to a monoclonal antibody (IgG) targeting the human insulin receptor (HIR), enabling receptor-mediated transcytosis across the blood-brain barrier (BBB) to address both peripheral and central nervous system manifestations of MPSI, which standard IDUA ERT (laronidase) cannot reach. Administered intravenously, it has shown stabilization of cognitive function (Development Quotient), cortical grey matter volume, urinary glycosaminoglycans, hepatosplenomegaly, and shoulder range of motion in pediatric patients with severe MPSI in a phase 1-2 trial, with a linear pharmacokinetic profile comparable to laronidase but faster clearance in children.[1][3][4]

Other names

valanafusp alfacTfRMAb-IDUA murine fusion proteinIgG-alpha-L-iduronidase fusion protein