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CRISPR for the liver
May 2016
EDIT CONNECT
SHARING OPTIONS:
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.”
Code: E051607 Back |
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