Date: 2018-03-21

Type of information: Research agreement

Compound: RNAi therapeutics

Company: Alnylam Pharmaceuticals (USA - MA) Regeneron Pharmaceuticals (USA - NY)

Therapeutic area: Hepatic diseases - Liver diseases

Type agreement: R&D - research

Action mechanism:

  • RNAi. RNAi (RNA interference) is a natural cellular process of gene silencing. Small interfering RNA (siRNA), the molecules that mediate RNAi  function by potently silencing messenger RNA (mRNA) that encode for disease-causing proteins. This approach could potentially mimic the naturally occurring loss-of-function genetic variation in HSD17B13 seen in people who are protected from NASH disease progression.

Disease: NASH (non-alcoholic steatohepatitis) and potentially other related diseases


  • • On March 21, 2018, Regeneron Pharmaceuticals and Alnylam Pharmaceuticals announced a collaboration to identify RNAi therapeutics for the chronic liver disease nonalcoholic steatohepatitis (NASH) and potentially other related diseases. The discovery collaboration is based on a new Regeneron publication in the New England Journal of Medicine identifying for the first time a variant in the HSD17B13 gene that is associated with reduced risk of chronic liver diseases.   Under the discovery collaboration, Regeneron will contribute research on the hepatocyte-expressed, genetically-validated HSD17B13 target, and Alnylam will leverage its RNAi therapeutics platform to identify compounds directed to this target. Regeneron and Alnylam intend to enter into a separate, fifty-fifty collaboration to further research, co-develop and commercialize any therapeutic product candidates that emerge from these discovery efforts.
  • This is Regeneron and Alnylam's second recent collaboration founded on genetic exploration. In January 2018, Alnylam, alongside multiple other leading life sciences companies, joined Regeneron's pre-competitive consortium to sequence 500,000 individuals in the UK Biobank health resource and subsequently make the data publicly available to the global research community.

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