Regeneron has announced an expansion of its collaboration agreement with Intellia Therapeutics to develop CRISPR-based gene editing therapies targeting neurological and muscular diseases. The expanded collaboration will bring together Regeneron’s proprietary delivery systems and antibody-targeted adeno-associated viral (AAV) vectors with Intellia’s Nme2 CRISPR/Cas9 gene editing technologies.
Initially, the partners will focus on two in vivo non-liver targets, with each company having the opportunity to lead the development and commercialization of candidates for one of the targets. The company not taking the lead on a particular target will have the option to enter into a co-development and co-commercialization agreement for that target.
Genetic medicines have shown promise as a therapeutic modality, but their widespread use has been limited by challenges in delivering genetic payloads to specific cells beyond the liver. Regeneron’s expansion of its collaboration with Intellia aims to leverage new technology and innovations to overcome these challenges.
Regeneron and Intellia first entered into their CRISPR collaboration in April 2016, with Regeneron investing $75 million upfront in Intellia’s gene editing platform. This platform utilizes the CRISPR/Cas9 system for in vivo and ex vivo gene editing programs.
They initially focused on transthyretin amyloidosis, and their investigational gene editing therapy NTLA-2001 showed promising results in reducing disease-causing protein levels in Phase I data.
The partnership was expanded in June 2020 to include hemophilia A and B as target diseases. Currently, the companies are working on Investigational New Drug (IND)-enabling activities for the hemophilia B candidate, while the hemophilia A program is in the early research stages. This expanded collaboration signifies the importance of gene editing technologies in the development of potential treatments for a range of genetic diseases.