Regeneron, Alnylam partner to turn discovery into new drugs for NASH
- Regeneron Pharmaceuticals Inc. on Thursday unveiled a new research collaboration with RNA specialist Alnylam Pharmaceuticals Inc. aimed at developing new drugs for a form of liver disease called non-alcoholic steatohepatitis (NASH).
- Combing through data from tens of thousands of electronic health records, researchers at Regeneron's Genetics Center uncovered a genetic variant associated with a reduced risk of chronic liver disease — a discovery the biotech hopes to turn into therapeutics using Alnylam's RNA interference technology.
- In addition to the initial research to find active compounds, the two companies also entered into an agreement to equally split R&D costs for any drug candidates identified through the discovery collaboration.
Alnylam has pioneered the development of therapeutics based on a cellular process known as RNA interference, using small strands of RNA to effectively "silence" genes that encode for disease-causing proteins. After 15 years of research, the biotech stands on the verge of winning approval for its first drug and has built a pipeline of other promising RNAi-based treatments.
It's that expertise that Regeneron hopes to tap to discover a compound capable of hitting the genetic target identified by scientists at the company's wholly owned genetics research lab.
Regeneron has made a major investment in its Genetics Center, aiming to increase its discovery output through greater emphasis on how genetic data can inform R&D. The Center recently celebrated the milestone of sequencing more than 250,000 exomes and has evaluated data from 180,000 de-identified electronic health records since its start in 2014.
Through a human genetic study known as DiscovEHR, researchers there identified a variant in a gene called HSD17B13 that was linked to lower serum levels of two liver enzymes. Subsequent work found people with two broken copies of that gene had reduced risk of both alcoholic and non-alcholic liver disease and cirrhosis.
Regeneron and Alnylam believe an RNAi therapeutic could be ideally suited to replicating that natural loss-of-function variant by interfering with the expression of HSD17B13. To be sure, the collaboration will start quite a ways from that goal, with only a potential target identified.
Yet the partnership speaks to both the interest in RNAi's applications outside of rare disease and to the increasing attention pharmaceutical firms are paying to NASH.
"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 Alnylam CEO John Maraganore in a statement.
While little known until recently, NASH is a more severe form of non-alcoholic fatty liver disease and can progress to cirrhosis. Estimates of the disease's prevalence vary widely: Regneron and Alnylam cite data indicating between 3% and 12% of adults in the U.S. have some form of NASH.
No drugs are currently approved for non-viral forms of chronic liver disease, so companies ranging from large drugmakers like Gilead Sciences Inc. and Allergan plc to smaller biotechs like Intercept Pharmceuticals Inc. and Galectin Therapeutics Inc. are racing to develop new drugs.
- Regeneron Pharmaceuticals Inc. Statement
- New England Journal of Medicine A protein-truncating HSD17B13 variant and protection from chronic liver disease
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