U Penn receives $10M from Clayco Foundation for research on rare genetic disease RVCL

The University of Pennsylvania has received $10 million from the Clayco Foundation to develop a potential treatment for retinal vasculopathy with cerebral leukoencephalopathy (RVCL), a rare genetic disease that presently has no cure. The award will fund preclinical safety studies of a small-molecule degrader designed to eliminate a faulty protein that drives disease progression. Researchers aim to complete animal testing within a year and advance the effective candidate toward human trials. Fewer than 50 families worldwide are known to have RVCL, which can cause blindness, organ damage, disability, and premature death.