A Boost in Rare Disease Research Highlights Cardiff Chemists’ Platform Approach

On September 25, 2018, Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in development and commercialization of treatments for orphan disorders, announced its acquisition of Ichorion Therapeutics, Inc. Ichorion, a privately-held biopharmaceutical company based in New York City, focused on developing treatments for inherited metabolic disorders known as Inborn Errors of Metabolism (IEMs) and has supported a multi-year collaboration led by Dr. Fabrizio Pertusati of Cardiff University’s School of Pharmacy and Pharmaceutical Sciences. Dr. Pertusati and his research team have sought to expand the applicability of the ProTide platform, a chemical approach to nucleoside prodrugs that originated at Cardiff with Dr. Pertusati’s mentor and advisor, the late Dr. Chris McGuigan. The Cardiff chemists have already demonstrated the ProTide platform’s utility to combat infectious diseases and cancer, but the collaboration with Ichorion has provided a fresh look at therapeutic approaches for rare metabolic diseases the primarily effect children. These diseases typically have high infant mortality rates and no approved therapies.

“Our collaboration with Ichorion to research and develop new treatments for rare pediatric diseases is critically important to these under-served patient populations” said Dr. Pertusati. “The ProTide approach for IEM therapies is a logical extension of the foundational work that Chris, our lab and colleagues have done over the past 20 years. We’ve witnessed its success in virology and oncology, and now we believe we can leverage the platform to address rare metabolic diseases that primarily affect children with no treatment options.”

Cerecor continues to support advancing a pipeline of therapies for IEMs through its partnership with Cardiff University.

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