Regeneron, Intellia Expand CRISPR/Cas9 Alliance

Regeneron Pharmaceuticals and Intellia Therapeutics expanded their existing collaboration to provide Regeneron with rights to develop products for additional in vivo CRISPR/Cas9-based therapeutic targets and for the companies to jointly develop potential products for the treatment of hemophilia A and B. Regeneron also receives non-exclusive rights to independently develop and commercialize ex vivo gene edited products. Intellia will receive an upfront payment of $70 million, and Regeneron will make an additional equity investment in Intellia of $30 million.

The companies have worked together advance Intellia's CRISPR/Cas9 platform to enable the targeted insertion of therapeutic proteins and antibodies. This collaboration expansion allows the companies to leverage more fully their jointly-developed targeted transgene insertion capabilities. In preclinical studies, the companies demonstrated the first CRISPR/Cas9-mediated targeted transgene insertion in the liver of non-human primates, which 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. These results suggest that transgene insertion may provide a functional Factor 9 gene, which encodes for this important protein.

Under the amended agreement, the term of the companies' existing collaboration is extended until April 2024, with Regeneron having an option to renew for an additional two years. Regeneron will have 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.

Regeneron will pay potential royalties and milestones for the in vivo products it independently develops. Regeneron will receive a royalty-bearing, non-exclusive license to certain Intellia intellectual property to develop and commercialize up to 10 ex vivo CRISPR/Cas9 products in defined cell types.