Charles Vite, DVM, PhD, is Associate Professor of Neurology and Neurosurgery at the School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA. Dr. Vite has a long-standing interest in neurodegeneration in feline and canine models of genetic disease, with specific focus on NPC disease. He maintains a unique breeding colony for production of cats affected with NPC disease, the only large animal model for NPC disease. Dr. Vite’s demonstration that intrathecal HPBCD rescued the neuropathology in NPC1 cat model is largely responsible for selection of dosing and route of administration for the HPBCD trial. He is currently focused on gene therapy for treatment of NPC disease, where the feline model will again prove pivotal in generating preclinical data appropriate and necessary to move this therapy towards clinical trial.
SOAR-NPC is a group of family foundations and scientists, with an extensive network of collaborators.