Regeneron Pharmaceuticals and Intellia Therapeutics have expanded their existing research collaboration to develop additional CRISPR-based gene editing therapies for neurological and muscular diseases.
The partnership will combine Regeneron’s proprietary antibody-targeted adeno-associated virus vectors and delivery technology with Intellia’s proprietary Nme2 CRISPR/Cas9 systems that are adapted for viral vector delivery and designed to precisely modify a target gene.
The companies originally announced a licensing and collaboration agreement in 2016 aimed at advancing CRISPR/Cas gene-editing technology for in vivo therapeutic development.
This was later expanded in 2020 to cover the co-development of potential haemophilia A and B treatments using their jointly owned targeted transgene insertion capabilities, with Regeneron also gaining the rights to develop products for additional in vivo targets and new rights for ex vivo product development.
Under the terms of the latest agreement, the companies will initially research two in vivo non-liver targets.
Intellia will lead the design of the editing methodology, while Regeneron will be responsible for the design of the targeted viral vector delivery approach.
Each company will have the opportunity to lead potential development and commercialisation of product candidates for one target, and the partner that is not leading development and commercialisation will have the option to enter into a co-development and co-commercialisation agreement for the target.
Aris Baras, senior vice president and co-head of Regeneron Genetic Medicines, said: “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.”
Intellia’s president and chief executive officer, John Leonard, added: “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.”
The agreement comes just days after Regeneron announced that its investigational bispecific antibody odronextamab had been accepted for priority review by the US Food and Drug Administration as a treatment option for certain non-Hodgkin lymphoma patients.
Odronextamab is specifically under review as a treatment for adults with relapsed/refractory follicular lymphoma or R/R diffuse large B-cell lymphoma who have progressed after at least two prior systemic therapies.