Drug Development Pipeline
VX-522 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, regardless of their CFTR mutations.
A Phase 1 study to test the safety and tolerability of VX-522 is underway. The study is for adults with CF who have CFTR mutations that are not responsive to CFTR modulator therapy.
This program is sponsored by Vertex Pharmaceuticals. It is being conducted within the Therapeutics Development Network (TDN).
Recent VX-522 mRNA Studies
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