Genes carry information that tells the body how to work. The cystic fibrosis transmembrane conductance regulator (CFTR) gene tells the body to create the CFTR protein, which is responsible for controlling the flow of salt and water in and out of different parts of the body, such as the lungs.
If there is not enough CFTR protein or it does not work properly, this leads to a problem with the balance of salt and water in the body. This causes a thick, sticky mucus to develop in different parts of the body, especially the lungs, and stops them from working as they should.
For many people living with cystic fibrosis, there are treatments available called 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 unresponsive to CFTR modulators.
The BEACON-CF clinical research study’s goal is to find out whether the investigational study drug is safe and may work for those who are not expected to benefit from CFTR modulators.
Many people living with cystic fibrosis can be treated with a type of treatment called CFTR modulators. This 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 do not respond to CFTR modulators.
For cystic fibrosis, mRNA therapy works by sending 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.
The investigational study drug, 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.