ALGS Research & Development Collaboration

Retrophin is participating in a three-way Cooperative Research and Development Agreement (CRADA) with the National Institutes of Health's National Center for Advancing Translational Sciences (NCATS) and patient advocacy foundation Alagille Syndrome Alliance (ALGSA) to collaborate on research efforts aimed at the identification and development of potentially novel therapeutic targets for Alagille syndrome (ALGS). ALGS is a rare and debilitating disease characterized by severe liver and cardiovascular abnormalities.

PATIENT NEED

ALGS is a rare genetic disorder that can affect the liver, heart, skeleton, eyes and kidneys. Symptoms and severity of ALGS can vary greatly from one person to another. Symptoms often develop during the first three months of life and include interrupted bile flow (cholestasis), jaundice, poor weight gain and growth, and severe itching (pruritis). There are no approved therapies for ALGS. Learn more about ALGS.

OUR APPROACH

The program goals are to develop high-throughput screen assays and conduct hit validation to identify potential new compounds that could be advanced in development, with the goal of delivering new and potentially novel therapeutic options to the ALGS patient community.