Regeneron boosts Intellia CRISPR pact with bleeding disorder focus, cash boost


Regeneron is doubling down on its collaboration with gene-editing biotech Intellia as they expand into work for hemophilia A and B.

Under the pact, Regeneron also nabs rights to develop and sell ex vivo gene-edited products. Intellia gets a $70 million upfront payment, with its partner also throwing in an equity investment of $30 million at $32.42 per share.

This builds on an original pact made back in 2016 in a similar setup, in which Intellia grabbed a $125 million upfront and an equity stake from Regeneron that saw it work with the biotech on up to 10 targets, primarily by studying diseases that can be addressed by editing genes in the liver.

At the time, Regeneron had the option to pick up to five non-liver targets, though Intellia’s work outside the liver was off limits.

Now, the pair is working on what they describe as a potential cure for bleeding diseases hemophilia A and B, tapping into the early work of the first CRISPR/Cas9-mediated targeted transgene insertion in the liver of non-human primates, which in preclinical tests generated normal or higher levels of circulating human factor IX.

Factor IX is a blood-clotting protein that is missing or defective in hemophilia B patients.  

“The Regeneron team works hard to push the boundaries of science and technology, and we believe the precise in vivo gene insertion capabilities jointly developed with Intellia could be a promising therapeutic platform with significant potential in many diseases, including those that have been historically difficult to treat,” said George Yancopoulos, M.D., Ph.D., co-founder, president and chief scientific officer at Regeneron.

“We're pleased to expand our work with Intellia, a like-minded group of scientists focused on maximizing the potential of CRISPR/Cas9 in order to help as many patients as possible.”

Pfizer and Sangamo Therapeutics, BioMarin and Roche/Spark are also looking to create potentially curative therapies for hemophilia, although there have been setbacks in the gene therapy field in the last few years, with safety and efficacy issues hitting a number of biopharmas.

RELATED: BioMarin gene therapy won't need an AdComm as it nabs speedy FDA review

“We're excited to work with Regeneron on what could potentially be a cure for hemophilia A and B in this expansion of our successful collaboration that builds on our leading insertion capabilities,” added Intellia's CEO and president, John Leonard, M.D.

“We believe that our CRISPR/Cas9-based technology addresses the limitations of current replacement and gene therapy approaches, and importantly, may provide a durable, potentially life-long solution to these genetic diseases.”

The deal is now boosted until April 2024, with Regeneron having an option to renew for another two years. Now, Regeneron has rights to discover and develop CRISPR/Cas9-based therapeutic products for an additional five in vivo liver targets, for a total of up to 15 targets.

Intellia was up nearly 25% in early trading on the news this morning.