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首页 / 抑制剂/激动剂 / 膜转运蛋白 / CFTR / Lumacaftor/鲁玛卡托

Lumacaftor/鲁玛卡托 {[allProObj[0].p_purity_real_show]}

货号：A121674 同义名： VX-809; VRT 826809

Lumacaftor (VX-809; VRT 826809) 是一种CFTR调节剂，可纠正CFTR蛋白的折叠和运输。

Lumacaftor/鲁玛卡托 化学结构 CAS号：936727-05-8
Lumacaftor/鲁玛卡托 化学结构
CAS号：936727-05-8
Lumacaftor/鲁玛卡托 3D分子结构
CAS号：936727-05-8
Lumacaftor/鲁玛卡托 化学结构 CAS号：936727-05-8
Lumacaftor/鲁玛卡托 3D分子结构 CAS号：936727-05-8
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Lumacaftor/鲁玛卡托 纯度/质量文件 产品仅供科研

货号：A121674 标准纯度： {[allProObj[0].p_purity_real_show]}
批次查询： 批次纯度:

全球学术期刊中引用的产品

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产品名称 CFTR ↓ 其他靶点 纯度
Ataluren ✔ 98%
Lumacaftor ++++

F508del-CFTR, EC50: 0.1 μM

 		98%
CFTR(inh)-172 +++

CFTR, Ki: 300 nM

 		99%+
GlyH-101 +

CFTR, Ki: 4.3 μM

 		99%+
IOWH-032 ++

CFTR, IC50: 1.01 μM

 		99%+
Tezacaftor ✔ 99%+
1. 鼠标悬停在“+”上可以显示相关IC50的具体数值。"+"越多，抑制作用越强。2. "✔"表示该化合物对相应的亚型有抑制作用，但抑制强度暂时没有相关数据。

Lumacaftor/鲁玛卡托 生物活性

靶点

  • CFTR

    F508del-CFTR, EC50:0.1 μM
描述 Cystic fibrosis (CF) is a genetic disorder with the loss of chloride transport due to the defects in CF transmembrane conductance regulator (CFTR) protein. VX-809 is a CFTP corrector that partially increases the delivery of CFTR to cell surface, thereby restoring the function of chloride transportation. It improved the maturation of F508del-CFTR, the most common CFTR mutation, in FRT cells with an EC50 value at 0.1 μM; and increased F508del-CFTR-mediated chloride transport with an EC50 at 0.5 μM. Data from F508del-HEK293 cells showed that incubation with 3 μM VX-809 for 24 hours increased F508del-CFTR exit from the endoplasmic reticulum by 6 folds compared with control cells. Also, cultured F508del-HBE cells treated with 3 μM VX-809 for 48 hours showed 14% higher maximal level of chloride transport compared to normal HBE cells[5]. In a human lung epithelium-derived cell line that was transfected with horseradish peroxidase (HRP)-tagged CFTR (F508-HRP CFBE41o-), cells treated with 2 μM VX-809 at 37C for 24 hours showed 4-fold HRP luminescence signal increase compared to DMSO-treated cells. Similar result was also found in R1070W-HRP CFBE41o- cells with 2.5-fold signal increase after VX-809 treatment at the concentration of 2 μM[6]. A phase 2 study in homozygous CF patients reported that daily intake of 100 and 200 mg VX-809 for 28 days significantly decreased their elevated sweat chloride values[7].
作用机制 VX-809 suppresses the folding defects of transmembrane conductance regulator (CFTR) protein by stabilizing an N-terminal domain in the CFTR that contains membrane-spanning domain 1[8].

Lumacaftor/鲁玛卡托 细胞实验

Cell Line
Concentration Treated Time Description References
Human monocytes 5 μM 1 h Lumacaftor/ivacaftor treatment restored the calcium response to thapsigargin in CF monocytes, suggesting that Lumacaftor/ivacaftor may reduce the overexpression of MMP9 in CF patients by modulating calcium homeostasis. J Cyst Fibros. 2020 Mar;19(2):245-254.
Human Bronchial Epithelial Cells (HBE) 3 μM 24 h To evaluate the efficacy of Lumacaftor on F508del and other CFTR mutants, results showed that A561E/A561E and F508del/F508del cells responded significantly to Lumacaftor, while N1303K/G542X cells showed no significant response. EBioMedicine. 2014 Dec 17;2(2):147-53.
Fischer rat thyroid (FRT) cells 3 μM 24 h To evaluate the effect of Lumacaftor on P67L CFTR protein expression, results showed that Lumacaftor significantly increased the expression of the mature, fully glycosylated form (band C) of P67L CFTR, reaching levels comparable to those of wild-type CFTR. JCI Insight. 2016 Sep 8;1(14):e86581.
Calu-3 2B4 cells 50 µM 48 h To test the effect of CFTR modulators on SARS-CoV-2 replication, results showed that Lumacaftor significantly reduced viral titers. mBio. 2023 Feb 28;14(1):e0313622.

Lumacaftor/鲁玛卡托 动物实验

Species
Animal Model
Administration Dosage Frequency Description References
Mice Wild-type mice (C57BL/6N) Intraperitoneal injection 3 mg/kg Twice daily for 2 days Lumacaftor pretreatment abolishes the circadian rhythm in cerebrovascular myogenic reactivity and eliminates the time-of-day difference in SAH-induced neurological injury. Stroke. 2022 Jan;53(1):249-259
Mice K18-hACE2 mice Intraperitoneal injection 12 mg/kg Once daily until 8 days post-infection To test the protective effect of CFTR modulators in SARS-CoV-2-infected mice, results showed that Lumacaftor significantly improved survival rates. mBio. 2023 Feb 28;14(1):e0313622.
Mice Colorectal cancer model Intraperitoneal injection 30 mg/kg Every 2 days until sacrificed Combination of Lumacaftor with anti-PD-1 antibody inhibits CRC growth and enhances anti-tumor immunity Nat Commun. 2024 Jul 12;15(1):5851

Lumacaftor/鲁玛卡托 动物研究

Dose Mice: 1.5 mg/kg[3] (i.v.) Rat: 1 mg/kg[4] (p.o.)， 1 mg/kg - 600 mg/kg[3] (p.o.), 1 mg/kg - 5 mg/kg[3] (i.v.)
Administration i.v., p.o.
Pharmacokinetics
Animal Rats[3]
Dose 30 mg/kg (p.o.)
3 mg/kg (i.v.)
Administration p.o.
i.v.
F 69.7% (p.o.)
T1/2 6.46 h (p.o.)
8.62 h (i.v.)
Tmax 2.0 h (p.o.)
0.083 h (i.v.)
Cmax 66200 ng/g (p.o.)
24600 ng/g (i.v.)
AUC0→∞ 410000 ng·h/g (p.o.)
58900 ng·h/g (i.v.)
AUC0→t 410000 ng·h/g (p.o.)
58900 ng·h/g (i.v.)

Lumacaftor/鲁玛卡托 临床研究

NCT号 适应症或疾病 临床期 招募状态 预计完成时间 地点
NCT03474042 Cystic Fibrosis Phase 2 Completed - Germany ... 展开 >> Study Site II Berlin, Germany Study Site X Dresden, Germany Study Site III Essen, Germany Study Site IV Frankfurt, Germany Study Site I Heidelberg, Germany Study Site V Köln, Germany Study Site VI München, Germany Study Site IX Stuttgart, Germany Study Site VIII Tübingen, Germany 收起 <<
NCT02821130 - Active, not recruiting December 2019 Canada, British Columbia ... 展开 >> UBC James Hogg Research Centre, St. Paul's Hospital Vancouver, British Columbia, Canada, V6Z1Y6 收起 <<
NCT02965326 Cystic Fibrosis Not Applicable Recruiting October 2020 France ... 展开 >> Necker Hospital Recruiting Paris, France, 75014 Contact: SERMET Isabelle, Professor    01 44 49 48 87    isabelle.sermet@nck.aphp.fr    Principal Investigator: SERMET Isabelle, Professor 收起 <<

Lumacaftor/鲁玛卡托 参考文献

[1]lumacaftor

[2]Van Goor F, Hadida S, et al. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc Natl Acad Sci U S A. 2011 Nov 15;108(46):18843-8.

[3]108(46):18843-8.

[4]86(1):42-51.

[5]Van Goor F, Hadida S, Grootenhuis PD, Burton B, Stack JH, Straley KS, Decker CJ, Miller M, McCartney J, Olson ER, Wine JJ, Frizzell RA, Ashlock M, Negulescu PA. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc Natl Acad Sci U S A. 2011 Nov 15;108(46):18843-8.

[6]Phuan PW, Veit G, Tan J, Roldan A, Finkbeiner WE, Lukacs GL, Verkman AS. Synergy-based small-molecule screen using a human lung epithelial cell line yields ΔF508-CFTR correctors that augment VX-809 maximal efficacy. Mol Pharmacol. 2014 Jul;86(1):42-51.

Lumacaftor/鲁玛卡托 实验方案

计算器
存储液制备 1mg 5mg 10mg

1 mM

5 mM

10 mM

2.21mL

0.44mL

0.22mL

11.05mL

2.21mL

1.11mL

22.10mL

4.42mL

2.21mL

Lumacaftor/鲁玛卡托 技术信息

CAS号936727-05-8
分子式C24H18F2N2O5
分子量 452.41
SMILES Code O=C(O)C1=CC=CC(C2=NC(NC(C3(C4=CC=C(OC(F)(F)O5)C5=C4)CC3)=O)=CC=C2C)=C1
MDL No. MFCD16659051
别名 VX-809; VRT 826809; Orkambi
运输蓝冰
InChI Key UFSKUSARDNFIRC-UHFFFAOYSA-N
Pubchem ID 16678941
存储条件

In solvent -20°C: 3-6个月 -80°C: 12个月

Pure form Sealed in dry,2-8°C
溶解方案

DMSO: 25 mg/mL(55.26 mM)，配合低频超声，并水浴加热至45℃助溶，注意：DMSO长时间开封后，会吸水并导致溶解能力下降，请避免使用长期开封的DMSO

请根据您的动物给药指南选择适当的溶解方案。
以下溶解方案都请先按照体外实验的方式配制澄清的储备液，再依次添加助溶剂：
——为保证实验结果的可靠性，澄清的储备液可以根据储存条件，适当保存；体内实验的工作液，建议现用现配，当天使用； 以下溶剂前显示的百分比是指该溶剂在终溶液中的体积占比；如在配制过程中出现沉淀、析出现象，可以通过加热和/或超声的方式助溶
方案 一
方案 二

Tags: Lumacaftor | VX-809;VRT 826809;Orkambi;​Lumacaftor | CFTR | AmBeed-信号通路专用抑制剂 | Lumacaftor/鲁玛卡托 纯度/质量文件 | Lumacaftor/鲁玛卡托 亚型比较 | Lumacaftor/鲁玛卡托 生物活性 | Lumacaftor/鲁玛卡托 动物研究 | Lumacaftor/鲁玛卡托 临床数据 | Lumacaftor/鲁玛卡托 参考文献 | Lumacaftor/鲁玛卡托 实验方案 | Lumacaftor/鲁玛卡托 技术信息

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