Regeneron and Intellia Therapeutics form pact to develop CRISPR/Cas therapeutics

New York-based Regeneron Pharmaceuticals and Intellia Therapeutics have signed a licensing and collaboration agreement to advance CRISPR/Cas gene-editing technology for in vivo therapeutic development. As well as developing new therapies, the companies will focus on technology development of the CRISPR/Cas platform.

Under the terms of the six-year agreement, Regeneron has the exclusive right to develop CRISPR-based products against up to 10 targets, focused primarily on therapies for diseases that may be treated by editing genes in the liver. Of the 10 targets, Regeneron can select up to five non-liver targets. Non-liver targets from Intellia's ongoing and planned research, as well as targets included in another Intellia collaboration, are excluded from this collaboration.

Cambridge, MA-based Intellia will receive a US$75m upfront payment and is eligible for significant milestone and royalty payments. The two firms have agreed to co-develop and co-commercialise a certain number of targets that are generated during the collaboration. Transthyretin amyloidosis is the first target to be jointly developed and potentially commercialised. Regeneron has also agreed to invest up to US$50m in Intellia's next equity financing.

'Our human genetics research with the Regeneron Genetics Centre is already identifying important genetic targets, building on our long-standing expertise in genetic engineering,' said George Yancopoulos, Chief Scientific Officer of Regeneron and President of Regeneron Laboratories. 'We believe combining these capabilities with Intellia's technology holds real promise for serious diseases that have been historically difficult to address, and expands our ability to help patients where antibody-based therapies may not be the optimal solution.'

CRISPR/Cas9 is a gene-editing technology that can cut DNA in precise locations, providing the opportunity to selectively knock out, repair or insert specific genetic sequences. It has potential application across multiple therapeutic areas including autoimmune diseases, metabolic and blood disorders, cancer and rare and genetic-based diseases.