Intellia, Regeneron to take CRISPR collaboration outside the liver with revised deal

Intellia Therapeutics and Regeneron Pharmaceuticals hope to take their CRISPR partnership outside the liver with an expansion on their existing work that yielded first-in-human gene editing data in 2021.

The two companies announced an expansion of their existing research collaboration to develop in vivo CRISPR-based therapies for neurological and muscular diseases in a Tuesday press release.

Regeneron will supply its proprietary antibody-targeted adeno-associated virus vectors and delivery systems, while Intellia will bring its proprietary Nme2 CRISPR/Cas9 systems that are adapted for viral vector delivery and designed to precisely modify a target gene.

Gene editing has so far been confined to delivery in the liver. Intellia and Regeneron want to change that with new technology from the latter company that has been shown preclinically to deliver payloads to different targeted tissue types and in multiple disease settings.

The deal will involve no upfront payment to or from either company but will involve two in vivo non-liver targets initially.

Intellia will head up design of the editing methodology, while Regeneron will design the targeted viral vector delivery approach.

Each company will lead development and commercialization for a product candidate for one target, while the other can opt into a 50/50 co-development and co-commercialization agreement.

Regeneron and Intellia began working together in 2016 and have revised their partnership a few times to add different targets. The original deal was worth $75 million upfront for Intellia plus milestone and royalty payments that were not disclosed for up to 10 targets. A 2020 revision added $70 million upfront plus a $30 million equity investment from Regeneron and extended the work up to April 2024. Milestones and royalties were also included but not disclosed.

The partnership has produced the in vivo CRISPR/Cas9 genome editing candidate NTLA-2001 for patients with transthyretin amyloidosis. The companies made waves in 2021 with first-in-human gene editing results from the therapy, which showed it reduced serum levels of transthyretin, a key biomarker for the disease.

Intellia also has hemophilia A and B and other undisclosed research programs in the works with Regeneron.