Intellia Therapeutics and ReCode Therapeutics have joined forces in a collaborative effort to develop treatments targeting cystic fibrosis (CF) caused by various CFTR genetic mutations. Initially concentrating on patients lacking effective treatments, the collaboration allows for potential expansion in the future.
Cambridge, Massachusetts-based Intellia will leverage its CRISPR gene-editing platform, contributing expertise in designing the editing strategy and leading the research phase. Intellia’s CRISPR technology focuses on in vivo and ex vivo programs aimed at treating cancer and autoimmune diseases, involving direct gene editing within the body and engineering human cells externally.
ReCode, headquartered in Menlo Park, California, will take the lead in preclinical and clinical development, offering its Selective Organ-Targeting (SORT) lipid nanoparticle delivery platform. This platform ensures precise delivery of genetic medicines to specific tissues, organs, and cells. ReCode has successfully utilized the SORT platform in formulating its lead mRNA-based therapeutic programs, RCT1100 and RCT2100, targeting primary ciliary dyskinesia and specific CFTR mutations, respectively.
In the event of successful product development, ReCode will spearhead global commercialization efforts, while Intellia retains the option to lead commercialization in the US for certain programs. Intellia stands to receive milestone payments linked to development and commercial activities, along with royalties from potential sales. The financial details of the collaboration remain undisclosed.