Regeneron, Alnylam to take on NASH in discovery deal

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The pair will apply Alnylam's gene-silencing tech to a gene variant associated with a lower risk of chronic liver disease. (Gerd Altmann)

Regeneron and RNA biotech Alnylam are teaming up to identify RNAi treatments for the chronic liver disease, nonalcoholic steatohepatitis (NASH), and potentially for other diseases too.

The collaboration will build on new findings from Regeneron identifying a link between a variant of the HSD17B13 gene and a lower risk of chronic liver disease. Alnylam will bring its gene-silencing RNAi technology to the table, while Regeneron will contribute research on the target. They will apply Alnylam’s tech to the gene in question to imitate the loss-of-function variant that staves off the progression of NASH in some people.

If the partnership turns up any potential therapies, the duo will pull the trigger on a separate, 50-50 collaboration to research, co-develop and commercialize them, the companies said in a statement.

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Any new treatment would plug a gap—the cause of NASH is unclear and it has no cure. Instead, physicians treat underlying conditions such as obesity and Type 2 diabetes. About 3% to 12% of adults in the United States have NASH, Regeneron said, and this number is only going to rise due to increasing rates of obesity.

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"NASH is a major cause of death in this country, with no current treatment options. 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,” said Regeneron’s Chief Scientific Officer George Yancopoulos.

After spending 15 years in R&D mode, Alnylam is slated to transition to the commercial stage midyear, with the rollout of its hereditary ATTR amyloidosis drug, patisiran.

"As we transition Alnylam toward commercialization 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," said John Maraganore, Alnylam CEO. "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."

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