This program was a collaboration between ReCode Therapeutics and Intellia Therapeutics to develop a gene editing therapy that could be delivered into the lung cells of people with cystic fibrosis.

ReCode focused on developing a version of its lipid nanoparticles that is optimized for delivery to lung stem cells, the ideal target for a permanent gene editing therapy. Lung stem cells are important because they create new lung cells to replace damaged or dying ones.

While ReCode concentrated on the delivery system, Intellia worked to develop the gene editing therapy being delivered. Intellia’s therapy was designed to target permanent or near-permanent correction of a specific nonsense mutation (also called a “stop” mutation) in the CFTR gene.

Status

This collaboration is completed. There is no active development in CF at this time.

Sponsor

This program was sponsored by ReCode Therapeutics in collaboration with Intellia Therapeutics. ReCode’s program was partially funded by the Cystic Fibrosis Foundation.