Regeneron and Intellia Therapeutics Expand Collaboration to Develop CRISPR/Cas9-Based Treatments for Hemophilia

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Intellia Therapeutics, Inc. (NASDAQ: NTLA) announced an expansion of 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 at $32.42 per share.

Regeneron and Intellia have worked together to make significant advances with 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 and potentially accelerate efforts to discover and develop new therapeutics, including products for hemophilia A and B. 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 D. Yancopoulos, M.D., Ph.D., Co-Founder, President and Chief Scientific Officer, Regeneron. “We’re pleased to expand our work with Intellia, a like-minded group of scientists focused on maximizing the potential of CRISPR/Cas9 in order to help as many patients as possible.”

“We’re excited to work with Regeneron on what could potentially be a cure for hemophilia A and B in this expansion of our successful collaboration that builds on our leading insertion capabilities,” said Intellia’s Chief Executive Officer and President, John M. Leonard, M.D. “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.” 

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.

About Regeneron Pharmaceuticals, Inc.
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to seven FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, infectious diseases, pain and rare diseases. 

Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® technologies, including VelocImmune® which uses a unique genetically-humanized mouse to produce optimized fully-human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world. For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.

About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients’ diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow us on Twitter @intelliatweets.

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