Drug discovery and genetics

A loss-of-function genetic variant that protects against chronic liver disease was reported in the The New England Journal of Medicine on 22nd March 2018. This finding adds to a series of previously established protective variants and appears to have stimulated efforts to advance treatment options within chronic liver disease — Regeneron Pharmaceuticals, Inc. have subsequently announced a collaboration with Alnylam Pharmaceuticals, Inc. to develop RNAi therapeutics for non-alcoholic steatohepatitis.

Such reports tend to generate excitement. To understand why requires analysis of the underlying genetics, biology, drug discovery challenges and opportunities within clinical medicine. My goal is to provide context to this intersection between science, medicine and business.

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