Open Drug Discovery to Treat Primary Sclerosing Cholangitis

Primary Sclerosing Cholangitis (PSC) is a rare liver disease with no approved treatments and a significant unmet medical need. The limited patient population makes investment in drug development for PSC challenging. This project seeks to address this by taking a collaborative open science approach to advance new treatments for this rare disease, which currently has only a limited number of active pharmaceutical programs.The investigators have developed the first transcriptomic map of the PSC liver, providing an unprecedented view of the disease mechanisms at the cellular level. This work has enabled the identification of known targets and their corresponding drugs, highlighting SYK inhibition as an avenue to target immune-mediated fibrotic pathways within the PSC liver. This project will test the ability of SYK inhibitors to modulate fibrogenic processes in human explanted PSC tissue in comparison to healthy human liver.