An investigational compound that promotes the maturation of delta F508 CFTR
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Tezacaftor

Item No. 21480

Technical Information
Formal Name
1-(2,2-difluoro-1,3-benzodioxol-5-yl)-N-[1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(2-hydroxy-1,1-dimethylethyl)-1H-indol-5-yl]-cyclopropanecarboxamide
CAS Number
1152311-62-0
Synonyms
  • VX-661
Molecular Formula
C26H27F3N2O6
Formula Weight
Purity
≥98%
A crystalline solid
DMF: 30 mg/mlDMSO: 30 mg/mlDMSO:PBS(pH 7.2) (1:3): 0.25 mg/mlEthanol: 25 mg/ml
λmax
233, 299 nm
SMILES
FC1(F)OC2=CC=C(C3(CC3)C(N(C4=C(F)C=C(N(C[C@H](CO)O)C(C(C)(C)CO)=C5)C5=C4)[H])=O)C=C2O1
InChi Code
InChI=1S/C26H27F3N2O6/c1-24(2,13-33)22-8-14-7-18(17(27)10-19(14)31(22)11-16(34)12-32)30-23(35)25(5-6-25)15-3-4-20-21(9-15)37-26(28,29)36-20/h3-4,7-10,16,32-34H,5-6,11-13H2,1-2H3,(H,30,35)/t16-/m1/s1
InChi Key
MJUVRTYWUMPBTR-MRXNPFEDSA-N
Shipping & Storage Information
Storage
-20°C
Shipping
Room temperature in continental US; may vary elsewhere
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    Product Description

    Tezacaftor is an investigational compound that promotes the maturation of delta F508 mutants of the cystic fibrosis transmembrane conductance regulator (CFTR).1 Delta F508 CFTR represents a class of CFTR mutation that is characterized by impaired processing of misfolded CFTR proteins and reduced accumulation of the protein at the cell surface.1 Tezacaftor is intended to facilitate trafficking of CFTR to the epithelial cell membrane. It may be combined with the CFTR potentiator ivacaftor (Item No. 15145) to stimulate both CFTR accumulation and opening at the apical epithelial surface.1,2

    WARNING This product is not for human or veterinary use.

    References & Product Citations
    Product Description References

    1. Petit, R.S., and Fellner, C. CFTR modulators for the treatment of cystic fibrosis. P.T. 39(7), 500-511 (2014).

    2. Veit, G., Avramescu, R.G., Perdomo, D., et alSome gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression. Sci. Transl. Med. 6(246), 246RA297 (2014).