Penn Medicine receives $10M to accelerate experimental drug treatment for rare genetic disease

The Clayco Foundation has announced a $10 million grant to the University of Pennsylvania’s Perelman School of Medicine to advance preclinical safety trials of an experimental therapy for retinal vasculopathy with cerebral leukoencephalopathy, a rare, fatal genetic disease with no approved treatment. Foundation chair Shawn Clark’s mother and grandfather died from RVCL. The funding will accelerate development of a novel therapy to help the body eliminate the abnormal proteins that drive the disease, positioning the therapy for future clinical trials. The grant addresses a critical funding gap for rare disease research and aims to speed progress toward a potential first treatment for the roughly 200 people worldwide living with RVCL.