VX-272

VX-272 is a small molecule Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) corrector developed by Vertex Pharmaceuticals. It was designed to address the underlying protein folding defect in cystic fibrosis, specifically for patients carrying the F508del mutation. As a corrector, VX-272 acts as a pharmacological chaperone that assists in the processing and trafficking of the CFTR protein to the cell surface, where it can then be activated by a potentiator (such as ivacaftor). VX-272 was evaluated in Phase 1 clinical trials as a potential component of a next-generation triple-combination regimen. However, following the successful clinical results of other candidates like elexacaftor (VX-445) and VX-659, Vertex Pharmaceuticals discontinued the development of VX-272 to focus on more promising leads for their CF portfolio.