Intellia Therapeutics and ReCode Therapeutics Forge Alliance to Pave the Way for Innovative Gene Editing Treatments for Cystic Fibrosis

CF, a genetic disorder stemming from mutations in the CFTR gene, triggers the accumulation of thick mucus in vital organs like the lungs and digestive system
Health & Medicine
February 16, 2024

Intellia Therapeutics, Inc. (NASDAQ:NTLA), a pioneering clinical-stage gene editing firm dedicated to transforming healthcare with CRISPR-based treatments, and ReCode Therapeutics, an innovative genetic medicines company specializing in tissue-specific delivery, have announced a strategic partnership to develop groundbreaking genomic therapies for cystic fibrosis (CF).

CF, a genetic disorder stemming from mutations in the CFTR gene, triggers the accumulation of thick mucus in vital organs like the lungs and digestive system, posing severe health risks including life-threatening infections and respiratory failure.

The collaboration capitalizes on Intellia's cutting-edge CRISPR-based gene editing platform, featuring its DNA writing technology, and ReCode's proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform. Together, they aim to precisely rectify one or more CF-related gene mutations.

In the initial phase, the focus will be on developing therapeutic interventions for CF patients with limited or no treatment options, with potential for expansion in subsequent stages. Intellia will spearhead the design of editing strategies and research-grade components for the investigational therapies, while ReCode will lead preclinical and clinical development. Furthermore, ReCode will oversee global commercialization for specific programs stemming from the collaboration.

Under the agreement, Intellia stands to receive predefined development and commercial milestone payments, along with royalties on potential sales. Additionally, Intellia retains the option to take the lead in commercializing certain programs in the U.S.

“Intellia’s vision to realize the full promise of gene editing includes extending the reach of our industry-leading CRISPR-based platform to targets outside the liver. This collaboration with ReCode is aimed at achieving that goal as we work together to accelerate the development of potentially life-changing therapies for people with cystic fibrosis,” said Intellia President and Chief Executive Officer John Leonard, M.D. “Building on our CRISPR/Cas9 capabilities, we have made important progress advancing our proprietary DNA writing technology to enable a range of precise editing strategies. We are excited to combine our gene editing expertise and platform with ReCode’s novel lung-directed LNP delivery platform.”

“We are excited to partner with Intellia, a clear leader in the gene editing space, with the ultimate goal of bringing life-altering therapies to CF patients,” said ReCode Chief Executive Officer Shehnaaz Suliman, M.D. (MB ChB), M.B.A., M.Phil. “This collaboration provides further validation of ReCode's SORT LNP platform to deliver diverse gene editing modalities to specific cells and tissues. By combining our highly synergistic technologies and capabilities, we are excited about the potential to enable a faster path for next-generation gene editing therapeutics to CF patients.”

This strategic alliance between Intellia and ReCode holds the promise of delivering groundbreaking therapies for cystic fibrosis, marking a significant advancement in the realm of genetic medicine and offering hope to patients grappling with this debilitating condition.

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