Elexacaftor (VX-445)

Elexacaftor (VX-445) (CAS No.: 2216712-66-0) is a synthetic small molecule categorized as a next generation cystic fibrosis transmembrane conductance regulator corrector and is widely used in biomedical research as a mechanistic probe for epithelial ion transport biology, protein quality control, and mutation associated defects in membrane protein maturation; functionally, it acts at the level of CFTR proteostasis by enhancing folding efficiency, intracellular processing, and trafficking of misprocessed CFTR variants, including Phe508del CFTR, thereby increasing the abundance of mature channel protein at the plasma membrane and supporting restoration of chloride transport phenotypes in experimental systems, with activity commonly characterized in cell based assays at nanomolar to micromolar concentrations depending on genotype, assay sensitivity, and co treatment design; current research positioning places this molecule in translational pharmacology and drug discovery workflows focused on target validation, combination modulator profiling, and structure activity guided optimization of CFTR rescue strategies, while mechanistic investigations often evaluate effects on endoplasmic reticulum quality control, vesicular trafficking routes, and functional channel readouts in recombinant cell lines, patient derived epithelial cultures, and relevant preclinical animal contexts, and the concentrations or doses used in experiments typically depend on specific experimental designs and research objectives.