Regeneron and Intellia to develop new haemophilia therapies

Regeneron Pharmaceuticals has expanded its ongoing partnership with Intellia Therapeutics for the development of therapies for haemophilia A and B.

The updated alliance also provides rights for Regeneron to develop products for more in-vivo CRISPR / Cas9-based therapeutic targets. The company also gains non-exclusive rights to develop and commercialise ex-vivo gene-edited products.

In turn, Intellia will get an upfront payment of $70m, along with an equity investment of $30m from Regeneron.

Regeneron Pharmaceuticals co-founder, president and chief scientific officer George  Yancopoulos said: “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.”

Regeneron and Intellia previously leveraged the latter’s CRISPR / Cas9 platform for targeted insertion of therapeutic proteins and antibodies.

This expansion is intended to leverage jointly-developed targeted transgene insertion capabilities and advance their discovery and development of therapies, including haemophilia A and B treatments.

The partners tested their first CRISPR / Cas9-mediated targeted transgene insertion in the liver of non-human primates. Following the insertion, the animals could produce normal or higher levels of circulating human Factor IX, a blood-clotting protein missing or defective in patients with haemophilia B.

According to the companies, these findings indicate that transgene insertion could offer a functional Factor 9 gene, which encodes for Factor IX.

Intellia Therapeutics CEO and president John Leonard said: “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 expanded deal, the term of the companies’ alliance comes with an extension until April 2024. Regeneron holds an option to renew the collaboration for an additional two years.

Last month, Regeneron announced the repurchase of around $5bn of its common stock directly from France’s Sanofi.