NEW YORK – Intellia Therapeutics and ReCode Therapeutics on Thursday said they are collaborating to develop treatments that correct one or more CFTR genetic mutations known to cause cystic fibrosis.
The companies will initially focus on developing therapeutic approaches for cystic fibrosis patients who currently lack treatments, but the partners have the option to expand the scope of the collaboration in the future.
Cambridge, Massachusetts-based Intellia, which will provide access to its CRISPR gene-editing platform, is responsible for designing the editing strategy and will lead the research stage of the collaboration. Intellia has in vivo and ex vivo programs focused on developing treatments for cancer and autoimmune diseases, in which it is using CRISPR to edit disease-causing genes directly inside the body and engineer human cells outside the body, respectively.
Menlo Park, California-based ReCode will lead preclinical and clinical development and provide access to its selective organ-targeting (SORT) lipid nanoparticle delivery platform, which allows it to precisely deliver genetic medicines to target tissues, organs, and cells. The company has used the SORT platform to formulate its two lead mRNA-based therapeutic programs, RCT1100 and RCT2100. The first, RCT1100, is an investigational treatment for primary ciliary dyskinesia caused by DNAI1 pathogenic mutations, and the second, RCT2100, is under evaluation for treating the 10 percent to 13 percent of cystic fibrosis patients who have Class I CFTR mutations and don't respond to currently available CFTR modulators.
If the companies are successful in bringing products to market, ReCode will lead global commercialization efforts, and Intellia has the option to lead commercialization in the US for certain programs. Intellia will also be eligible to receive milestone payments related to development and commercial activities and royalties on potential sales. The companies didn't disclose further financial details of their collaboration.