NIH R01 · 2024
Modulation of Ca2+ -activated K+ channels by CFTR Correctors
Cystic fibrosis (CF) results from mutations in the CFTR Cl- channel resulting in diminished transepithelial Cl- secretion. The most common CFTR mutation, F508del, results in a channel that fails to correctly fold/traffic. Thus, compounds were sought to improve the folding/trafficking of this misfolded CFTR - referred to as correctors. Vertex pioneered the identification of CFTR correctors, and in 2019 introduced Trikafta, consisting of the correctors, VX-661 (Tezacafor) and VX-445 (Elexacaftor), together with the CFTR potentiator VX-770 (Ivacaftor). This highly effective modulator therapy (HEMT) has proved to be highly efficacious. Unfortunately, real-world studies have reported adverse…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.