CFTR

Class

CFTR potentiator

CFTR (cystic fibrosis transmembrane conductance regulator) is a chloride- and bicarbonate-conducting channel found on the surface of epithelial cells lining the lungs, pancreas, intestine, and other secretory tissues. Its normal function is to move ions across the cell membrane, which draws water along with them and keeps the mucus layer coating these tissues thin and fluid. In cystic fibrosis, inherited mutations in the gene encoding CFTR impair this channel, either by preventing it from folding correctly and reaching the cell surface, or by allowing it to reach the surface but open poorly once there. The result is thick, sticky mucus that obstructs airways and ducts, predisposing patients to chronic lung infection and pancreatic insufficiency. Drugs targeting CFTR aim to restore chloride transport by acting directly on the mutant protein rather than treating downstream symptoms such as infection or inflammation. Because different mutations disrupt the channel in different ways, therapies have been designed to address distinct defects: some improve the protein's folding and trafficking to the membrane, while others act on channels already present at the surface to increase how often they open. This mechanism-based approach has reshaped treatment of cystic fibrosis by targeting the root molecular cause rather than only its consequences.