ReCode Therapeutics CF gene correction program

This gene correction program is being developed by ReCode Therapeutics in collaboration with Intellia Therapeutics for the treatment of cystic fibrosis (CF). The therapy utilizes Intellia's proprietary CRISPR-based gene editing platform, specifically its DNA-writing technology, delivered via ReCode's Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform. The program aims to permanently or near-permanently correct mutations in the CFTR gene at the genomic level to restore functional protein production. Designed for delivery via inhalation, the therapy initially targets lung cells to address the respiratory complications of CF. This approach is specifically intended for the approximately 10% of CF patients with genetic mutations that are non-responsive to or intolerant of existing CFTR modulator therapies. The program is currently in the discovery phase and has received up to $15 million in funding from the Cystic Fibrosis Foundation.

Other names

CF gene correction programIntellia/ReCode CF gene correction programCFTR gene correction program