Description
VX-809 is a novel CFTR corrector that modulates the folding and trafficking of CFTR protein. VX-809 stabilizes an N-terminal domain in CFTR that contains only MSD1 (membrane-spanning domain 1) and efficaciously restores function to CFTR forms that have missense mutations in MSD1. The action of VX-809 on MSD1 appears to suppress folding defects in F508del-CFTR by enhancing interactions among the NBD1, MSD1, and MSD2 domains.
VX-809 is a novel CFTR corrector that modulates the folding and trafficking of CFTR protein. VX-809 stabilizes an N-terminal domain in CFTR that contains only MSD1 (membrane-spanning domain 1) and efficaciously restores function to CFTR forms that have missense mutations in MSD1. The action of VX-809 on MSD1 appears to suppress folding defects in F508del-CFTR by enhancing interactions among the NBD1, MSD1, and MSD2 domains.
Alternate Name/Synonyms: 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid; Lumacaftor
Appearance: White to off-white solid
Formulation: N/A
CAS Number: 936727-05-8
Structure Available?: Yes
Peptide sequence: N/A
Salt Form: No
Molecular Formula: C₂₄H₁₈F₂N₂O₅
Molecular Weight: 452.41
Cell-Permeable?: Yes
Purity: ≥98% by HPLC
Solubilities: DMSO (>90 mg/ml)
Handling: Protect from air and light
Country of Origin: USA
Tag Line: A novel CFTR corrector
MDL Number: N/A
PubChem CID: 16678941
SMILES: CC1=C(N=C(C=C1)NC(=O)C2(CC2)C3=CC4=C(C=C3)OC(O4)(F)F)C5=CC(=CC=C5)C(=O)O
InChi: InChI=1S/C24H18F2N2O5/c1-13-5-8-19(27-20(13)14-3-2-4-15(11-14)21(29)30)28-22(31)23(9-10-23)16-6-7-17-18(12-16)33-24(25,26)32-17/h2-8,11-12H,9-10H2,1H3,(H,29,30)(H,27,28,31)
InChi Key: UFSKUSARDNFIRC-UHFFFAOYSA-N
Additional Information
Storage Condition: |
-20°C |
Shipping Condition: |
Gel Pack |
Shelf Life: |
36 months |