Regeneron Pharmaceuticals, Inc. and Intellia Therapeutics, Inc. expanded their research collaboration to develop additional in vivo CRISPR-based gene editing therapies focused on neurological and muscular diseases. This builds on the success of the companies’ existing collaboration and combines both companies’ biology and technology expertise. The collaboration will leverage Regeneron’s antibody-targeted adeno-associated virus (AAV) vectors and delivery systems and Intellia’s Nme2 CRISPR/Cas9 (Nme2Cas9) systems adapted for viral vector delivery and designed to precisely modify a target gene.
 
Under 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. This expansion of our longstanding and productive collaboration with Intellia is taking advantage of new technology and innovations to unlock these opportunities,” said Aris Baras, M.D., Senior Vice President and Co-Head of Regeneron Genetic Medicines.
 
“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,” said Intellia President and Chief Executive Officer John Leonard, M.D. “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.”