- Regeneron Pharmaceuticals and Intellia Therapeutics are expanding a gene editing collaboration agreed to four years ago, announcing Monday that they have added projects to treat hemophilia to their joint development program.
- Intellia will receive $100 million, adding to the $125 million it was paid in the initial round of the collaboration in 2016. The agreement also gives Regeneron the right to use Intellia’s technology to develop and commercialize therapies that re-engineer cells to help fight disease.
- While gene replacement therapies like Luxturna and Zolgensma are beginning to transform treatment in a few rare diseases, Regeneron has opted to pursue a theoretically simpler approach of editing faulty genes, albeit one that has never been approved for use in humans. However, with hemophilia gene therapies from BioMarin Pharmaceutical and UniQure nearing the market, Regeneron and Intellia may find fewer available patients for a once-and-done cure.
The collaboration comes just a week after Regeneron’s longtime collaborator Sanofi announced it will sell off its stake in the big biotech as the two companies continue to loosen ties. Regeneron pledged to buy back $5 billion in shares, but with $7 billion in cash as of March 31 it may be looking to make more deals to fill its pipeline in the post-Sanofi era.
Regeneron’s payment to Intellia will consist of $70 million in cash and a $30 million equity investment at $32.42 a share, an 85% premium above Intellia's closing share price on Friday. The deal gives Regeneron a roughly 7% stake in Intellia.
Intellia's share price rose 10% to $19.31 in Monday morning trading.
The original collaboration had been set to expire in April 2022, with an option to extend to 2024 for $25 million. As a result of this transaction, the expanded collaboration now is set to expire in 2024, with an option to extend to 2026.
So far, the collaboration has generated an experimental therapy to treat a genetically driven disease that results in a dangerous buildup of the protein amyloid in organs. That project, called NTLA-2001, is in preclinical testing.
The original 2016 deal gave Regeneron the right to develop up to 10 gene editing projects using a technology called CRISPR/Cas9 that is also used by such companies as Crispr Therapeutics and Editas Medicine. A Regeneron spokesperson said the company is not disclosing whether the hemophilia projects announced today account for any of the 10 outlined in the 2016 deal.
Regeneron and Intellia say they have used CRISPR technology in non-human primates to stimulate production of a protein called factor IX that is lacking in patients with hemophilia B. The two will face competition as they move forward, as several gene therapies are in development for hemophilia B. UniQure has one, for instance, in late-stage testing.
A new addition to the collaboration is Regeneron's non-exclusive rights to use Intellia's CRISPR platform to develop cell therapies that can be used to combat disease. Novartis has also partnered with Intellia on similar projects.
Intellia's own work on cell therapies has focused on developing tumor targeting cells as well as cells that would turn off immune responses in autoimmune disease.