Intellia Therapeutics Inc. (NASDAQ:NTLA) and Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) have announced an expansion to their current collaboration for the development of CRISPR/Cas9 treatments.
Collaboration to develop hemophilia A and B treatments
The expansion of the collaboration will see Regeneron receive product development rights for more in vivo CRISPR/Cas9-based treatments. Also, it will result in the companies working together to co-develop possible treatment products for both hemophilia A/B through the co-owned focused transgene insertion capabilities.
Equally, Regeneron will get non-exclusive rights to develop independently as well as market ex vivo gene products. On the other hand, Intellia will be awarded $70 million as an upfront payment with Regeneron, also making an additional $30 million equity investment at $32.42 per share in Intellia.
Previously, the companies have collaborated to make major advances in the development of Intellia’s CRISPR/Cas9 platform, which permits targeted transgene therapeutic insertion of antibodies and proteins. The expansion of the collaboration will enable the companies to leverage the co-developed targeted transgene therapeutic insertion capabilities fully.
CRISPR/Cas9 transgene insertion could help with Factor IX gene
Through preclinical studies, the companies have shown the first CRISPR/Cas9-based transgene insertion in non-human primates’ liver that generated normal to higher circulating levels of Factor IX. Normally Factor IX is the blood-clotting protein that is absent or defective in patients with hemophilia B. according to results from the clinical trial, the insertion of the transgene could offer a functional Factor IX gene that will encode for this vital protein.
Regeneron President, Chief Scientific Officer, and Co-founder George Yancopoulos indicated that the company has been working hard to push science and technology boundaries. He added that Regeneron believes in the accurate in vivo transgene insertion capabilities that the companies have developed jointly, which can be a therapeutic platform with huge potential in several diseases.
John Leonard, the CEO of Intellia, indicated that they were delighted to expand the collaboration with Regeneron to develop possible cures for hemophilia A/B. He added that the CRISPR/Cas9-based tech can address challenges in the current gene therapy and replacement approaches.