Regeneron Pharmaceuticals and Intellia Therapeutics announced on Monday that they will be expanding an existing collaboration. Regeneron will have the rights to develop products for additional in viro CRISPR/Cas9-based therapeutic targets. In addition, both companies will work on creating products for the treatment of hemophilia A and B.

Regeneron will receive the non-exclusive rights to independently develop and commercialize ex vivo gene edited products, and Intellia will receive an upfront payment of $70 million. Regeneron will make an additional equity investment in Intellia of $30 million.

"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."

Both companies have made significant advances with Intellias CRISPR/Cas9 platform to perform the targeted insertion of therapeutic proteins and antibodies. In preclinical studies, Intellia and Regeneron demonstrated the first CRISPR/Cas9-mediated targeted transgene insertion in the liver of non-human primates. This generated normal or higher levels of circulating Factor IX, a blood-clotting protein that is missing or defective in hemophilia B patients.

"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."

Regeneron and Intellia arent the only companies looking for treatments for hemophilia. BioMarin Pharmaceutical, Inc. provided updates to the previously reported results from its open-label Phase 1/2 study on May 31. The trial looked into valoctocogene roxaparvovec as a form of treatment for adults with severe hemophilia A. People with this condition lack the necessary functioning Factor VIII proteins to help their blood clot.

All subjects in both the 6e13 vg/kg and 4e13 vg/kg cohorts remain off prophylactic Factor VIII treatment since being given a single dose of valoctocogene roxaparvovec. Cumulative mean annualized bleed rates remain less than one in both cohorts and below pre-treatment baseline levels.

Overall, the safety profile of valoctocogene roxaparvovec remains consistent with previously reported data, and no participants experienced thrombotic events. The U.S. Food and Drug Administration is currently reviewing the biologics license application from BioMarin.

"It's been a privilege to participate in this pioneering research and to observe how the patients on the study have done so much to improve our understanding of gene therapy research for hemophilia A. This additional data is an important step toward a potential first treatment of its kind for this devastating disease," said Professor John Pasi, M.B., Ch.B., Ph.D., from Barts and the London School of Medicine and Dentistry; chief investigator for the valoctocogene roxaparvovec Phase 1/2 study, and a principal investigator for the Phase 3 study. "Each year of data increases our knowledge of safety and efficacy and contributes to the growing body of scientific data on gene therapies in general and hemophilia A in particular."

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Regeneron and Intellia Expand Their Hunt for Hemophilia A and B Treatments - BioSpace

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