Elexacaftor (VX-445, Compound 1) is a modulator of cystic fibrosis transmembrane conductance regulator (CFTR). Elexacaftor (VX-445, Compound 1) facilitates the processing and trafficking of CFTR to increase the amount of CFTR at the cell surface.
性状
Solid
IC50 & Target[1][2]
CFTR.
体外研究(In Vitro)
Elexacaftor (VX-445) is a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector designed to restore Phe508del CFTR protein function. Elexacaftor (VX-445) has the potential to treat cystic fibrosis. VX-445-Tezacaftor-VX-770 significantly improves Phe508del CFTR protein processing, trafficking, and chloride transport to a greater extent than any two of these agents in dual combination. has not independently confirmed the accuracy of these methods. They are for reference only.
运输条件
Room temperature in continental US; may vary elsewhere.
储存方式
Powder -20°C 3 years;4°C 2 years
ClinicalTrial
参考文献
[1]. Alexander Russell Abela, et al. MODULATOR OF THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR , PHARMACEUTICAL COMPOSITIONS , METHODS OF TREATMENT , AND PROCESS FOR MAKING THE MODULATOR. US 20180162839 A1.[2]. Keating D, et al. VX-445-Tezacaftor-VX-770 in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. N Engl J Med. 2018 Oct 25;379(17):1612-1620.
溶解度数据
In Vitro: DMSO : 125 mg/mL (209.15 mM; Need ultrasonic)配制储备液
[1]. Alexander Russell Abela, et al. MODULATOR OF THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR , PHARMACEUTICAL COMPOSITIONS , METHODS OF TREATMENT , AND PROCESS FOR MAKING THE MODULATOR. US 20180162839 A1.[2]. Keating D, et al. VX-445-Tezacaftor-VX-770 in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. N Engl J Med. 2018 Oct 25;379(17):1612-1620.