Regeneron Expands Collaboration on Gene Editing Therapies for Neurological and Muscular Diseases

BCW Member Regeneron Pharmaceuticals Inc. announced this week that it has expanded its research collaboration with Intellia Therapeutics, Inc. to develop additional in vivo CRISPR-based gene editing therapies focused on neurological and muscular diseases.

The collaboration will leverage Regeneron’s proprietary antibody-targeted adeno-associated virus vectors and delivery systems and Intellia’s proprietary Nme2 CRISPR/Cas9 systems adapted for viral vector delivery and designed to precisely modify a target gene. 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.

“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 Dr. 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.”

“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,” added Intellia President and CEO Dr. John Leonard. “At Intellia, we are continuously innovating our editing and delivery solutions to realize the full potential of CRISPR gene editing as a new therapeutic modality. This collaboration is representative of our long-standing belief that the most groundbreaking solutions will come from selecting the best tools for each individual application, all of which are enabled by our industry-leading genome editing toolbox.”