Figure 1.

Functional evaluation of VX-445 corrector activity in heterologous expression systems. Compounds were tested on CFBE41o- and FRT cells expressing F508del-CFTR and the halide-sensitive yellow fluorescent protein (HS-YFP). (A) Dose-responses relationships for VX-445 and its (R)-enantiomer (i.e., distomer), as single agents or in the presence of VX-809 (1 µM) on CFBE41o- cells. (B) Evaluation of corrector combination. The graphs report F508del-CFTR activity in CFBE41o- (left) and FRT (right) cells treated with vehicle alone (DMSO) or VX-809 (3 µM), VX-661 (10 µM), corr-4a (5 µM), 3151 (10 µM), 4172 (10 µM), racVX-445 (3 µM), or their combinations. Symbols indicate statistical significance versus treatment with racVX-445 alone: *, p < 0.05; **, p < 0.01; ***, p < 0.001.