Regeneron Pharmaceuticals will license Intellia Therapeutics’ CRISPR/Cas gene-editing technology in a collaboration focused primarily on developing therapies for diseases that may be treated by editing genes in the liver, the companies said today.

The collaboration could generate more than $125 million for Intellia.

Under the companies’ six-year agreement, Regeneron has the exclusive right to discover and develop CRISPR-based products against up to 10 targets.

Of the 10, Regeneron can select up to 5 nonliver targets. However, the collaboration will exclude nonliver targets from Intellia's ongoing and planned research, as well as targets that are included in another Intellia collaboration.

The only other collaboration previously announced by Intellia is an alliance with Novartis launched last year. It focuses on ex vivo development of new CRISPR/Cas9-based therapies using chimeric antigen receptor T cells and hematopoetic stem cells.

Intellia and Regeneron also agreed to co-develop and co-commercialize an unspecified number of targets to be generated during the collaboration. Transthyretin amyloidosis will be the first target to be jointly developed and potentially commercialized by the companies.

“Together, we aim to bring potential cures to patients who are suffering from life-threatening rare diseases and genetic diseases,” Nessan Bermingham, Ph.D., Intellia’s CEO and founder, said in a statement.

In addition to discovery, development and commercialization of new therapies, the companies said they will focus on development of the CRISPR/Cas platform.

In return for its rights, Regeneron agreed to pay Intellia $75 million upfront, as well as pay undisclosed “significant” payments tied to achieving milestones, plus royalty payments on potential Regeneron products.

“Our industry-leading human genetics research with the Regeneron Genetics Center is already identifying important genetic targets, building on our long-standing expertise in genetic engineering,” added George D. Yancopoulos, M.D., Ph.D., Regeneron CSO 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.”

Regeneron has agreed to invest up to $50 million in Intellia's next equity financing, the companies said.

In September, Intellia secured $70 million in series B financing, saying it intended to use the proceeds to speed up development of its pipeline, expand its gene editing and delivery platforms, and strengthen its intellectual property portfolio.


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