VX-661

Short Summary : F508del CFTR corrector

Category : Membrane Transporter/Ion Channel|CFTR

Purity : 0.98

CAS Number : 1152311-62-0

Formula : C26H27F3N2O6

Molecular Weight : 520.5

SMILE : CC(C)(CO)C1=CC2=CC(=C(C=C2N1CC(CO)O)F)NC(=O)C3(CC3)C4=CC5=C(C=C4)OC(O5)(F)F

Solubility : >21.8mg/mL in DMSO

Storage : Store at -20°C

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 VX11C661-101 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

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