Brief Description

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].
References:
[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

Solubility

DMSO 100 mg/ml; Water < 1 mg/ml

Melting point

Boiling point

Density

Formula Weight:

Flash point

Storage conditions

Store at -20°C 2 years

Appearance:

White to off-white solid