PFIC3 is caused by loss-of-function variants in ABCB4, the transporter that moves phosphatidylcholine into bile. Without it, bile becomes toxic to the bile ducts — causing itching, jaundice and fibrosis, and for most children, liver transplantation before adulthood.
BiloMedics, a company in process of incorporation, is developing a novel siRNA therapeutic designed to address the underlying cause of PFIC3 — a first-principles approach built on decades of UMCG’s research into bile acid physiology and hepatology.
