Product Name :
VX-809
Description:
VX-809, is a potent CFTR corrector, many be useful for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation. Cystic fibrosis (CF) is a genetic disorder that causes multiorgan morbidity and premature death, most commonly from pulmonary dysfunction.
CAS:
936727-05-8
Molecular Weight:
452.41
Formula:
C24H18F2N2O5
Chemical Name:
3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid
Smiles :
CC1=CC=C(NC(=O)C2(CC2)C2=CC3OC(F)(F)OC=3C=C2)N=C1C1=CC(=CC=C1)C(O)=O
InChiKey:
UFSKUSARDNFIRC-UHFFFAOYSA-N
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)
Purity:
≥98% (or refer to the Certificate of Analysis)
Shipping Condition:
Shipped under ambient temperature as non-hazardous chemical or refer to Certificate of Analysis
Storage Condition :
Dry, dark and -20 oC for 1 year or refer to the Certificate of Analysis.{{MARK-IN-2} medchemexpress|{MARK-IN-2} PI3K/Akt/mTOR|{MARK-IN-2} NF-κB|{MARK-IN-2} Technical Information|{MARK-IN-2} Formula|{MARK-IN-2} manufacturer}
Shelf Life:
≥12 months if stored properly.
Stock Solution Storage:
0 – 4 oC for 1 month or refer to the Certificate of Analysis.
Additional information:
VX-809, is a potent CFTR corrector, many be useful for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation. Cystic fibrosis (CF) is a genetic disorder that causes multiorgan morbidity and premature death, most commonly from pulmonary dysfunction.|Product information|CAS Number: 936727-05-8|Molecular Weight: 452.41|Formula: C24H18F2N2O5|Synonym:|VRT-826809|Lumacaftor|Chemical Name: 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid|Smiles: CC1=CC=C(NC(=O)C2(CC2)C2=CC3OC(F)(F)OC=3C=C2)N=C1C1=CC(=CC=C1)C(O)=O|InChiKey: UFSKUSARDNFIRC-UHFFFAOYSA-N|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)|Technical Data|Appearance: Solid Power|Purity: ≥98% (or refer to the Certificate of Analysis)|Solubility: DMSO: 90 mg/mL(198.{{GMP EGF Protein, Human} MedChemExpress|{GMP EGF Protein, Human} Purity & Documentation|{GMP EGF Protein, Human} In Vitro|{GMP EGF Protein, Human} manufacturer|{GMP EGF Protein, Human} Autophagy} 93 mM). Water: Insoluble.|Shipping Condition: Shipped under ambient temperature as non-hazardous chemical or refer to Certificate of Analysis|Storage Condition: Dry, dark and -20 oC for 1 year or refer to the Certificate of Analysis.|Shelf Life: ≥12 months if stored properly.|Stock Solution Storage: 0 – 4 oC for 1 month or refer to the Certificate of Analysis.|Drug Formulation: To be determined|HS Tariff Code: 382200|How to use|In Vitro:|VX-809 acts at the level of the ER to allow a fraction of the F508del-CFTR to adopt a properly folded form, to exit the ER and mobilize to the cell surface for normal functioning.PMID:25955218 In Fischer rat thyroid (FRT) cells expressing F508del-CFTR, VX-809 treatment significantly improves F508del-CFTR maturation by 7.1 fold with an EC50 of 0.1 μM, and enhances F508del-CFTR-mediated chloride transport by approximately 5 fold with EC50 of 0.5 μM, while VRT-768 has higher EC50 values of 7.9 μM and 16 μM, respectively. In HEK-293 cells expressing F508del-CFTR, VX-809 (3 μM) treatment increases F508del-CFTR exit from the ER by 6 fold, reaching levels comparable to 34% of CFTR. In primary human bronchial epithelial (HBE) cells with F508del-CFTR mutation, VX-809 increases CFTR maturation and enhances chloride secretion with EC50 of 350 nM and 81 nM, respectively, more efficacious than Corr-4a and VRT-325. F508del-CFTR corrected by VX-809 exhibits single-channel open probability of 0.39 similar to normal CFTR of 0.40. Unlike VX-770, VX-809 is not a CFTR potentiator, as acute addition of VX-809 has no effect on F508del-CFTR function. In contrast to VRT-325 and Corr-4a, VX-809 does not improve the processing of the normal or mutant forms of hERG or P-gp, as well as other disease-causing mislocalized proteins, including α1-antitrypsin Z mutant (E342K-α1-AT) or N370S-β-glucosidase, suggesting that VX-809 is specific for CFTR. VX-809 in combination with VRT-325 or Corr-4a has additive effect on CFTR-mediated chloride transport in cultured F508del-HBE.|References:|Van Goor F, et al. Proc Natl Acad Sci U S A, 2011, 108(46), 18843-18848.Products are for research use only. Not for human use.|
