Valanafusp-Alpha: The Development Path of a Brain Penetrating Molecular Enzyme Replacement Therapy for the Treatment of Neurological & Somatic Symptoms pf MPS I

Time: 1:10 pm
day: Day One


• The trojan horse platform of antibody-enzyme fusion proteins to transport therapeutics across the blood-brain-barrier
• Discovery, validation and preclinical development of Valanafusp alpha, aka AGT-181
• Phase II clinical proof of concept of Valanafusp alpha and its effect on pediatric patients with severe untransplanted MPS I
• Regulatory pathway and clinical endpoints discussed with FDA and EMA for a pivotal trial with Valanafusp alpha