Page Title
Drug Development Pipeline
VX-522 mRNA
Status
Phase TwoTherapeutic Approach
Genetic Therapy
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, including those with nonsense (also known as “x” or “stop”) and other rare mutations that do not respond to CFTR modulators.
Status
A Phase 2 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.
Sponsor
This program is sponsored by Vertex Pharmaceuticals. It is being conducted within the Therapeutics Development Network (TDN).
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