RARE Daily

Regeneron and Intellia Expand CRISPR/Cas9 Collaboration

June 1, 2020

Rare Daily Staff

Regeneron Pharmaceuticals will pay Intellia $70 million up front and make an additional equity investment in Intellia of $30 million as part of an agreement to expand their existing collaboration 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.

As part of the expanded agreement, Regeneron will receive non-exclusive rights to independently develop and commercialize ex vivo gene edited products as part of the agreement.

Regeneron and Intellia have worked together to make advances with Intellia’s CRISPR/Cas9 platform to enable the targeted insertion of therapeutic proteins and antibodies. The companies said the expansion will allow them to better leverage their jointly developed targeted transgene insertion capabilities and potentially accelerate efforts to discover and develop new therapeutics.

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.

“The Regeneron team works hard to push the boundaries of science and technology, and we believe the precise in vivo gene insertion capabilities jointly developed with Intellia could be a promising therapeutic platform with significant potential in many diseases, including those that have been historically difficult to treat,” said George Yancopoulos, co-founder, president and chief scientific officer of Regeneron.  

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. As currently set forth in the existing collaboration, the parties will jointly research these in vivo targets, and thereafter certain targets may be developed by Regeneron or Intellia or co-developed under certain conditions.

Per the terms of the original agreement, Regeneron will pay potential royalties and milestone payments for the in vivo products it independently develops. In addition, 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.

“We believe that our CRISPR/Cas9-based technology addresses the limitations of current replacement and gene therapy approaches, and importantly, may provide a durable, potentially life-long solution to these genetic diseases,” said Intellia President and CEO John Leonard.


Photo: George Yancopoulos, co-founder, president and chief scientific officer of Regeneron

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