BEACON-CF: Investigational mRNA Therapy for Cystic Fibrosis

Trial Objectives

For many people living with cystic fibrosis, there are treatments available called cystic fibrosis transmembrane conductance regulator (CFTR) modulators that can help treat the underlying cause of cystic fibrosis. However, more than 5,000 people living with cystic fibrosis have a change in both of their CFTR genes that makes them not expected to benefit from CFTR modulators.

The BEACON-CF clinical research study is exploring an investigational study drug called VX-522, a type of messenger RNA (mRNA) therapy, to see if it is safe and well tolerated for those who are not expected to benefit from CFTR modulators.

mRNA therapy is designed to send a message that tells cells (the building blocks of the body) to produce normal proteins that can do the job of proteins that are faulty or missing. If the investigational study drug is effective, it will tell cells in the lungs to create normal CFTR protein, which may improve the way the lungs work in people with cystic fibrosis.

VX-522 will be given as a single dose using a modified PARI nebulizer, an investigational medical device that turns liquid medications into a mist, which is then inhaled.