RCT2100 is an inhaled messenger RNA (mRNA) therapy. It aims to deliver a full-length copy of CFTR mRNA to lung cells using a lipid nanoparticle. Lung cells would then use the instructions in the mRNA to create functional CFTR protein. This type of therapy could work for any person with CF, including those with nonsense (also known as “x” or “stop”) and other rare mutations that do not respond to CFTR modulators.

Status

A Phase 1 study to test the safety of RCT2100 in healthy volunteers is currently underway. 

Sponsor

This program is sponsored by ReCode Therapeutics and partially funded by the Cystic Fibrosis Foundation.