Regeneron and Intellia Expand Collaboration to Develop CRISPR-Based Therapies

Rare Daily Staff

Regeneron Pharmaceuticals and Intellia Therapeutics expanded their research collaboration to develop additional in vivo CRISPR-based gene editing therapies focused on neurological and muscular diseases.

The agreement builds on the success of the companies’ existing collaboration and combines Intellia’s leading genome editing platform, including its proprietary Nme2Cas9 technology, with Regeneron’s proprietary antibody-targeted viral vector delivery technologies to jointly advance in vivo programs outside of the liver for neurological and muscular diseases.

“To date, the widespread use of genetic medicines has generally been limited by the inability to deliver a genetic payload to cells of interest in the body beyond the liver,” said Aris Baras, senior vice president and co-head of Regeneron Genetic Medicines. “This expansion of our longstanding and productive collaboration with Intellia is taking advantage of new technology and innovations to unlock these opportunities.”

Under the terms of the expanded agreement, the companies will initially research two in vivo non-liver targets. Intellia will lead the design of the editing methodology and Regeneron will lead the design of the targeted viral vector delivery approach. Each company will have the opportunity to lead potential development and commercialization of product candidates for one target, and the company that is not leading development and commercialization will have the option to enter into a co-development and co-commercialization agreement for the target.

Photo: Aris Baras, senior vice president and co-head of Regeneron Genetic Medicines