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CRISPR for the liver
TARRYTOWN, N.Y. & CAMBRIDGE, Mass.—With a focus on the liver as the starting point, Regeneron Pharmaceuticals Inc. and Intellia Therapeutics Inc. recently announced they had inked a six-year licensing and collaboration agreement to advance CRISPR/Cas gene-editing technology for in-vivo therapeutic development.
Under this deal, Regeneron has the exclusive right to discover and develop CRISPR-based products against as many as 10 targets, focused primarily on therapies for a broad range of 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. That “other collaboration” would almost certainly be a deal forged with Novartis last year that focuses on ex-vivo development of new CRISPR/Cas9-based therapies using chimeric antigen receptor T cells and hematopoetic stem cells.
Regeneron and Intellia, in addition to pursuing the discovery, development and commercialization of new therapies, will also put major effort behind technology development of the CRISPR/Cas platform itself.
“Our industry-leading human genetics research with the Regeneron Genetics Center is already identifying important genetic targets, building on our longstanding expertise in genetic engineering,” said Dr. George D. 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.”
Intellia will receive a $75 million upfront payment and is eligible to receive significant milestone and royalty payments on potential Regeneron products. Intellia and Regeneron have agreed to co-develop and co-commercialize a certain number of targets that are generated during the collaboration. Transthyretin amyloidosis is the first target to be jointly developed and potentially commercialized by the companies. Regeneron has also agreed to invest up to $50 million in Intellia’s next equity financing.
“We are excited to be partnering with Regeneron, an industry leader in human genetics research,” said Dr. Nessan Bermingham, CEO and founder of Intellia Therapeutics. “Regeneron’s focus on advancing science to medicine is an excellent fit with Intellia’s approach, and together, we aim to bring potential cures to patients who are suffering from life-threatening rare diseases and genetic diseases.”
Intellia is also using the relationship with such a big biotech player—along with the Novartis connection—as part of its efforts to launch a $120-million initial public offering.
As for Regeneron, Zacks Investment Research noted of the deal: “We remind investors that Regeneron has been pursuing strategic collaboration in order to develop the candidates in its pipeline. Earlier this month, the company entered into a licensing agreement with MedImmune, the global biologics research and development arm of AstraZeneca plc, to produce antibody drug conjugates, using MedImmune’s pyrrolobenzodiazepine-based warhead and linker technology for the treatment of cancer.”