CF, a genetic disorder triggered by mutations in the CFTR gene, leads to the accumulation of thick mucus in vital organs such as the lungs and digestive system, often resulting in severe respiratory complications and other life-threatening conditions.

The collaboration will merge Intellia's state-of-the-art CRISPR-based gene editing platform, including its DNA writing technology, with ReCode's Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform. The goal is to precisely correct one or more mutations responsible for CF. Initial research efforts will concentrate on therapeutic strategies for CF patients who have limited or no treatment options available, with the potential to broaden collaboration scope in later phases.

Under the terms, Intellia will design the editing strategy and provide research-grade components for investigational therapies, while ReCode will lead subsequent preclinical and clinical development, as well as global commercialization for certain programs. Intellia stands to receive specified development and commercial milestone payments, along with royalties on potential sales. Additionally, Intellia retains the option to spearhead commercialization in the U.S. for specific programs.

Intellia’s President and CEO, John Leonard, M.D., expressed enthusiasm for the partnership, emphasizing their commitment to extending the reach of gene editing beyond the liver to address critical unmet medical needs. He highlighted the progress made in advancing their proprietary DNA writing technology to enable precise editing strategies, anticipating transformative outcomes for CF patients.

ReCode's CEO, Shehnaaz Suliman, M.D. (MB ChB), M.B.A., M.Phil., echoed the sentiment, underscoring the collaboration's potential to deliver life-altering therapies to CF patients. Suliman emphasized the validation of ReCode's SORT LNP platform for targeted gene editing delivery and the synergistic capabilities between the two companies.

The partnership represents a significant stride towards realizing advanced gene editing therapies for CF, signifying a new frontier in precision medicine.