Ivacaftor (VX-770)

製品コードS1144 バッチS114401

印刷

化学情報

Chemical Structure Synonyms VX-770 Storage
(From the date of receipt)		 3 years -20°C powder
1 years -80°C in solvent
化学式

C24H28N2O3
分子量 392.49 CAS No. 873054-44-5
Solubility (25°C)* 体外 DMSO 79 mg/mL (201.27 mM)
Ethanol 2 mg/mL (5.09 mM)
Water Insoluble
体内 （毎回新しく調製した物を用意してください）
Homogeneous suspension
CMC-NA
≥5mg/ml Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
* <1 mg/ml means slightly soluble or insoluble.
* Please note that Selleck tests the solubility of all compounds in-house, and the actual solubility may differ slightly from published values. This is normal and is due to slight batch-to-batch variations.

溶剤液（一定の濃度）を調合する

生物活性

製品説明 Ivacaftor (VX-770) is a selective potentiator of CFTR targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM in fisher rat thyroid cells, respectively.
in vitro

Ivacaftor (VX-770) (10 μM) significantly increases the forskolin-stimulated Cl- secretion (IT) by ~4-fold with an EC50 of 100 nM in the recombinant Fisher rat thyroid (FRT) cells expressing G551D gating mutation of CFTR, and by ~6-fold with an EC50 of 25 nM in the recombinant cells expressing temperature-corrected F508del processing mutation of CFTR. Consistent with the increases in the forskolin-stimulated IT, this compound increases the open probability (Po) of G551D-, F508del-, and wild-type CFTR by ~6-fold, ~5-fold and ~2-fold, respectively, indicating that it acts directly on CFTR to increase its gating activity. In primary cultured human CF bronchial epithelia (HBE) carrying the G551D and F508del CFTR mutations, Ivacaftor potently increases the forskolin-stimulated IT by ~10-fold from 5% to a maximum level of 48% of that measured in non-CF HBE, with an EC50 of 236 nM displaying ~70-fold more potency compared with the commonly used CFTR potentiator genistein, which has an EC50 of 16 μM. In HBE with F508del homozygous CFTR, it causes a significant increase in the forskolin-stimulated IT with an EC50 of 22 nM, to a less extent from 4% to 16% of non-CF HBE compared with the effect in G551D/F508del HBE. Due to CFTR potentiation, this compound inhibits excessive ENaC-mediated Na+ and fluid absorption with an IC50 of 43 nM, and decreases the response, resulting in an increase in the surface fluid and cilia beat frequency (CBF) in G551D/F508del HBE. [1]
特徴 The first potent and orally available CFTR potentiator to enter human clinical trials.

プロトコル(参考用のみ)

キナーゼアッセイ Ussing Chamber Recordings
The effect of Ivacaftor (VX-770) on CFTR-mediated Cl- secretion is characterized by measuring the CFTR-mediated IT in chambers using recombinant Fisher rat thyroid (FRT) cells expressing G551D, or F508del CFTR. This compound's impact is assessed with cells grown on Costar Snapwell cell culture inserts maintained at 37 °C before recording. The cell culture inserts are mounted into an Ussing chamber to record IT in the voltage-clamp mode (Vhold = 0 mV). For FRT cells, the basolateral membrane is a basolateral to apical Cl- gradient is established. The basolateral bath solution contains 135 mM NaCl, 1.2 mM CaCl2, 1.2 mM MgCl2, 2.4 mM K2HPO4, 0.6 mM KHPO4, 10 mM N-2-hydroxyethylpiperazine-N
細胞アッセイ 細胞株 FRT and NIH 3T3 cells
濃度 100/25 nM
反応時間 24 h
実験の流れ

Cells were treated with various concentrations of Ivacaftor (VX-770).
動物実験 動物モデル Male Sprague-Dawley rats
投薬量 3 mg/kg
投与方法 i.v.

参考

  • https://pubmed.ncbi.nlm.nih.gov/19846789/
  • https://pubmed.ncbi.nlm.nih.gov/25441013/

カスタマーフィードバック

Data from [Data independently produced by Sci Transl Med, 2014, 6(246), 246ra96]

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Selleckの高級品が、幾つかの出版された研究調査結果（以下を含む）で使われた：

ACE-tRNAs are a platform technology for suppressing nonsense mutations that cause cystic fibrosis [ Nucleic Acids Res, 2025, 53(13)gkaf675] PubMed: 40650978
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CFTR negatively reprograms Th2 cell responses, and CFTR potentiation restrains allergic airway inflammation [ JCI Insight, 2025, 10(9)e191098] PubMed: 40131363
Human induced pluripotent stem cells for in vitro modeling of impaired mucociliary clearance in cystic fibrosis lung disease [ Stem Cell Res Ther, 2025, 16(1):573] PubMed: 41116195
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Inflammation-induced loss of CFTR-expressing airway ionocytes in non-eosinophilic asthma [ Respirology, 2025, 30(1):25-40] PubMed: 39358991
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Human Induced Lung Organoids: A Promising Tool for Cystic Fibrosis Drug Screening [ Int J Mol Sci, 2025, 26(2)437] PubMed: 39859153
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Elexacaftor/Tezacaftor/Ivacaftor Supports Treatment for CF with ΔI1023-V1024-CFTR [ Int J Mol Sci, 2025, 26(11)5306] PubMed: 40508114

長期の保管のために-20°Cの下で製品を保ってください。

人間や獣医の診断であるか治療的な使用のためにでない。

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