Risorius

Risorius Therapeutics is a preclinical-stage biotechnology company dedicated to developing transformative treatments for neurodevelopmental disorders, with a primary focus on Angelman Syndrome. The company's core technology centers on small molecule inhibitors designed to unsilence the paternal copy of the UBE3A gene, compensating for the loss of the maternal allele. By restoring functional UBE3A protein levels in the brain, Risorius aims to address the underlying genetic cause of the disorder rather than just managing symptoms. The company was founded based on pioneering research from the University of North Carolina at Chapel Hill and is supported by venture capital investment.