Partnership to develop gene editing therapies for neurological, muscular diseases

Regeneron Pharmaceuticals Inc. and Intellia Therapeutics Inc. have announced an expanded research collaboration intended to develop additional CRISPR-based gene editing therapies focused on neurological and muscular diseases.

According to a release from Intellia, the expanded partnership seeks to combine Regeneron’s proprietary antibody-targeted adeno-associated virus vectors and delivery systems with Intellia’s proprietary Nme2 CRISPR/Cas9 (Nme2Cas9) systems adapted for viral vector delivery which modify targeted genes.

“We are excited to expand our successful collaboration with Regeneron to now accelerate the development of CRISPR-based therapies outside of the liver for the treatment of neurological and muscular diseases with significant unmet need,” Intellia President and CEO John Leonard, MD, said in the release.

Under the terms of a newly expanded agreement, the companies will initially research two in vivo non-liver targets, with Intellia leading the design of editing methodology while Regeneron will lead design of the targeted viral vector delivery approach. Also per the release, the agreement further stipulates that each partner may lead potential development and commercialization of product candidates for one target, while the company that is not leading development and commercialization may enter into a co-development and co-commercialization agreement for that target.

“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,” Aris Baras, MD, senior vice president and co-head of Regeneron Genetic Medicines, said in the release. “This expansion of our longstanding and productive collaboration with Intellia is taking advantage of new technology and innovations to unlock these opportunities.”