Regeneron, Intellia to develop CRISPR/Cas9-based treatments

By The Science Advisory Board staff writers

June 1, 2020 -- Regeneron Pharmaceuticals and Intellia Therapeutics have expanded their existing collaboration to provide Regeneron with rights to develop in vivo CRISPR/Cas9-based therapeutic targets and for the two firms together to develop treatments for hemophilia A and B.

Under terms of the deal, Regeneron will pay Intellia $70 million up front and make an additional investment in the firm of $30 million in the near future. The new agreement expands the companies' collaboration through April 2024. In preclinical studies, the companies demonstrated a CRISPR/Cas9-mediated targeted transgene insertion in the liver of nonhuman primates, which generated normal or higher levels of circulating human factor IX.

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 also receive a royalty-bearing, nonexclusive license to certain Intellia intellectual property to develop and commercialize up to 10 ex vivo CRISPR/Cas9 products in defined cell types.

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