Page Title
Drug Development Pipeline
VX-522 mRNA
Status
DiscontinuedTherapeutic Approach
Genetic Therapy
This program studied VX-522, an inhaled messenger RNA (mRNA) therapy. VX-522 was designed 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 was stopped early because of tolerability issues.
Sponsor
This program was sponsored by Vertex Pharmaceuticals. It was conducted within the Therapeutics Development Network (TDN).
CONTACT THE CLINICAL TRIAL NAVIGATOR
Get personalized assistance and answers to your clinical trial questions.
Learn MoreSign up for clinical trial alerts
Get email updates about clinical trials that matter to you.
Explore clinical trials
Be a part of the movement transforming the future of cystic fibrosis treatment.
Learn More