Tezacaftor

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Chemical Structure| 1152311-62-0 同义名 : VX-661
CAS号 : 1152311-62-0
货号 : A210449
分子式 : C26H27F3N2O6
纯度 : 99%+
分子量 : 520.498
MDL号 : MFCD23106064
存储条件：

Pure form Inert atmosphere,2-8°C

In solvent -20°C:3-6个月-80°C:12个月
溶解度 :

DMSO: 50 mg/mL(96.06 mM)，配合低频超声助溶，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
动物实验配方：

IP 2% DMSO+2% Tween80+40% PEG300+water 10 mg/mL clear

PO 0.5% CMC-Na 52 mg/mL suspension

生物活性
靶点

  • CFTR
描述 Cystic fibrosis is caused by a reduced quantity or function of cystic fibrosis transmembrane conductance regulator (CFTR) protein, owing to mutations in CFTR. VX-661 is a first-generation CFTR corrector that has shown enhanced CFTR function and improved lung function in a phase 2 clinical trial involving patients who were homozygous for the Phe508del mutation or heterozygous for the Phe508del and G551D mutations [3]. The combination of VX-445 (2 μM) and VX-661 (18 μM) increased levels of mature CFTR protein and led to an increase in human bronchial epithelial cells from Phe508del–MF (a minimal-function mutation) and Phe508del–Phe508del (the Phe508del CFTR mutation) donors [4]. All doses of [VX-659-VX-661-ivacaftor] resulted in significant improvement in the percentage of predicted FEV1 (forced expiratory volume in 1 second) as compared with the respective within-group baseline values at day 29 in patients with Phe508del-MF or Phe508del-Phe508del genotypes [5].
实验方案
1mg 5mg 10mg

1 mM

5 mM

10 mM

1.92mL

0.38mL

0.19mL

9.61mL

1.92mL

0.96mL

19.21mL

3.84mL

1.92mL
参考文献

[1]Taylor-Cousar JL, Munck A, McKone EF, van der Ent CK, Moeller A, Simard C, Wang LT, Ingenito EP, McKee C, Lu Y, Lekstrom-Himes J, Elborn JS. Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del. N Engl J Med. 2017 Nov 23;377(21):2013-2023. doi: 10.1056/NEJMoa1709846. Epub 2017 Nov 3. PMID: 29099344.

[2]Keating D, Marigowda G, Burr L, Daines C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Sass LA, Tullis E, McKee CM, Moskowitz SM, Robertson S, Savage J, Simard C, Van Goor F, Waltz D, Xuan F, Young T, Taylor-Cousar JL; VX16-445-001 Study Group. VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. N Engl J Med. 2018 Oct 25;379(17):1612-1620. doi: 10.1056/NEJMoa1807120. Epub 2018 Oct 18. PMID: 30334692; PMCID: PMC6289290.

[3]Davies JC, Moskowitz SM, Brown C, Horsley A, Mall MA, McKone EF, Plant BJ, Prais D, Ramsey BW, Taylor-Cousar JL, Tullis E, Uluer A, McKee CM, Robertson S, Shilling RA, Simard C, Van Goor F, Waltz D, Xuan F, Young T, Rowe SM; VX16-659-101 Study Group. VX-659-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. N Engl J Med. 2018 Oct 25;379(17):1599-1611. doi: 10.1056/NEJMoa1807119. Epub 2018 Oct 18. PMID: 30334693; PMCID: PMC6277022.