Intellia, ReCode team up to develop gene-editing therapies for CF

Initial research will focus on patients with 'limited or no treatment options'

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Intellia Therapeutics and ReCode Therapeutics are joining forces to develop new genomic medicines for people with cystic fibrosis (CF).

The companies said initial research will focus on patients who have “limited or no treatment options available.” The ultimate goal is to create a new genomic medicine that can “precisely correct one or more CF disease-causing gene mutations,” they said.

“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,” John Leonard, MD, Intellia’s president and CEO, said in a company press release.

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“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 Shehnaaz Suliman, MD, ReCode’s CEO.

CF is caused by mutations in the gene CFTR. The gene codes for the CFTR protein, which normally helps regulate the production of mucus. In CF, it’s dysfunctional or absent, resulting in the production of unusually thick, sticky mucus that builds up in organs and drives most disease symptoms.

A recently developed class of medications called CFTR modulators can improve the functionality of the defective protein, but only in people with specific CF-causing mutations.

Intellia’s technology platform is based on CRISPR/Cas9, a gene-editing technology that can be used to alter the genetic code inside cells. The company has multiple therapies in clinical development for other diseases, using CRISPR to alter genes in the liver.

“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,” Leonard said.

The companies seek to deliver Intellia’s gene-editing technology to CF patients’ cells using ReCode’s lipid nanoparticle (LNP) platform, called Selective Organ Targeting (SORT).

“This collaboration provides further validation of ReCode’s SORT LNP platform to deliver diverse gene-editing modalities to specific cells and tissues,” Suliman said. “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.”

ReCode’s lead programs include RCT2100, an experimental inhaled therapy intended to deliver healthy CFTR messenger RNA into the lungs so they produce a functional CFTR protein. Messenger RNA is a temporary molecule created from DNA and used as a template to make proteins. RCT2100 is intended for CF patients caused by class 1 mutations — mutations that result in little to no CFTR protein production — who don’t respond to CFTR modulators.

“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,” Leonard said. “We are excited to combine our gene-editing expertise and platform with ReCode’s novel lung-directed LNP delivery platform.”