The expanded collaboration combines Intellia’s genome editing platform with Regeneron’s proprietary antibody-targeted viral vector delivery technologies to develop therapies for neurological and muscular diseases


Regeneron and Intellia have expanded their drug discovery partnership. (Credit: National Cancer Institute on Unsplash)

US-based Regeneron Pharmaceuticals and biotechnology company Intellia Therapeutics have expanded their research collaboration to develop CRISPR-based gene editing therapies.

Both companies will work together to develop additional in vivo CRISPR-based therapies for the treatment of neurological and muscular diseases.

The partnership will use Regeneron’s proprietary antibody-targeted adeno-associated virus (AAV) vectors and delivery systems and Intellia’s proprietary Nme2 CRISPR/Cas9 (Nme2Cas9) systems.

Intellia’s system is updated for viral vector delivery and created to modify a target gene precisely.

Regeneron genetic medicines SVP and co-head Aris Baras 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.”

According to the terms of the expanded deal, the companies will initially assess two in vivo non-liver targets.

Regeneron Pharmaceuticals will take the lead in developing the tailored viral vector delivery strategy, while Intellia will design the editing methodology.

Each company will have the chance to take the lead on potential product candidates for one target’s development and commercialisation.

The agreement also includes an option for the company not to take the lead to enter into a co-development and co-commercialisation agreement for the target.

Intellia president and CEO John Leonard said: “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.

“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.”

Both organisations first collaborated in 2020 to co-develop potential treatments for haemophilia A and B.