Page Title
Drug Development Pipeline
ReCode Therapeutics/Intellia Therapeutics
Status
Pre-clinicalTherapeutic Approach
Genetic Therapy
This program is 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 is 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 concentrates on the delivery system, Intellia is working to develop the gene editing therapy being delivered. Intellia’s therapy is designed to target permanent or near-permanent correction of a specific nonsense mutation (also called a “stop” mutation) in the CFTR gene. Other rare mutations that are not eligible for CFTR modulators will be explored as the editing technology matures.
Status
Laboratory studies to develop and test this technology are underway.
Sponsor
This program is sponsored by ReCode Therapeutics in collaboration with Intellia Therapeutics. ReCode’s program is partially funded by the Cystic Fibrosis Foundation.

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