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March 22, 2018

Regeneron and Alnylam to find new RNAi drugs for NASH disease

Regeneron Pharmaceuticals has formed an alliance with Alnylam Pharmaceuticals to discover RNA interference (RNAi) therapeutics for the treatment of nonalcoholic steatohepatitis (NASH), a chronic liver condition.

Regeneron Pharmaceuticals has formed an alliance with Alnylam Pharmaceuticals to discover RNA interference (RNAi) therapeutics for the treatment of nonalcoholic steatohepatitis (NASH), a chronic liver condition.

The move comes after Regeneron identified a new HSD17B13 gene variant associated with a decreased risk of chronic liver diseases.

RNAi therapeutics are drugs that target and silence any gene involved in the cause or pathway of human disease.

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.

Under the collaboration, Regeneron will undertake research on the hepatocyte-expressed, genetically-validated HSD17B13 target, while Alnylam will use its RNAi therapeutics platform to find compounds directed to this target.

“The prospect of collaborating with a scientific leader like Regeneron on innovative medicines for more prevalent diseases like NASH makes perfect strategic sense.”

Alnylam Pharmaceuticals CEO John Maraganore said: “As we transition Alnylam toward commercialisation in rare diseases, the prospect of collaborating with a scientific leader like Regeneron on innovative medicines for more prevalent diseases like NASH makes perfect strategic sense.

“We believe the exquisite specificity afforded by the RNAi mechanism of action and our industry-leading, proprietary GalNAc-conjugate approach for delivery to the liver is an unparalleled combination for developing an RNAi therapeutic toward genetically-validated targets in NASH.”

The partners are planning another collaboration to jointly research, develop and commercialise the therapeutic product candidates produced by this research alliance.

Regeneron Pharmaceuticals president and chief scientific officer George Yancopoulos said: “Our Regeneron Genetics Center is delivering new targets that will require new approaches beyond our biologics capabilities.

“We’re eager to build on the exciting science delivered by the Regeneron team in the hopes of helping patients with debilitating and life-threatening chronic liver diseases.”

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