Background:

VX-661, one of vertex derivatives, corrects F508del-CFTR trafficking and increases F508del-CFTR protein activity in vitro [1].

VX-661 treated alone or in combination with ivacaftor have shown to enhance F508del-CFTR traf?cking to the cell surface. VX-661 has been at phase 2 study [1].

参考文献:
[1] S. Donaldson, J. Pilewski, M. Griese, Q. Dong, P.-S. Lee, for the VX11–661-101 Study Group. WS7.3?VX-661, an investigational CFTR corrector, in combination with ivacaftor, a CFTR potentiator, in patients with CF and homozygous for the?F508Del-CFTR?mutation: Interim analysis. Journal of Cystic Fibrosis,?Volume 12, Supplement 1,?June 2013,?Page S14