CFTR

信号通路图

研究领域

抑制剂选择性比较
溶解性比较

目录号	产品目录	溶解度(25°C)
目录号	产品目录	水	DMSO	酒精
S6003	Ataluren (PTC124)	<1 mg/mL	57 mg/mL	<1 mg/mL
S7139	CFTRinh-172	<1 mg/mL	82 mg/mL	<1 mg/mL
S7329	IOWH032	<1 mg/mL	100 mg/mL	<1 mg/mL
S8094	GlyH-101	<1 mg/mL	98 mg/mL	<1 mg/mL
S1144	Ivacaftor (VX-770)	<1 mg/mL	78 mg/mL	<1 mg/mL
S8698	GLPG1837	<1 mg/mL	69 mg/mL	5 mg/mL
S1565	VX-809 (Lumacaftor)	<1 mg/mL	90 mg/mL	6 mg/mL
S7059	Tezacaftor (VX-661)	<1 mg/mL	100 mg/mL	<1 mg/mL

CFTR产品

所有产品 CFTR抑制剂(4) CFTR激活剂(2) CFTR调节剂(2) 新CFTR产品

产品目录	产品描述	文献引用	实验数据
S6003	Ataluren (PTC124) Ataluren (PTC124)选择性诱导核糖体转录通读，但不影响正常的终止密码子，在HEK293细胞中EC50为0.1 μM，可以治疗nonsense mutations (如CFTR无义突变引起的CF)造成的家族遗传性疾病。 Phase 3。	J Clin Invest, 2013, 124(1):111-6 FASEB J, 2013, 28(4):1593-9 Hum Mutat, 2012, 33(6):973-80	Effect of systemic ataluren treatment on mice with the Pax6^Sey+/- phenotype. The black arrowhead indicates the lenticular stalk; the black arrow indicates the cornea; and the asterisk indicates the ciliary margin. WT, Pax6^+/+; Mt, Pax6^Sey/+; L, lens; r, retina. Original magnification, x5.
S7139	CFTRinh-172 CFTRinh-172是电势差不依赖性的选择性CFTR抑制剂，K_i为300 nM，对于MDR1，ATP敏感性钾离子通道或者一系列的转运蛋白没有效果。	Nature, 2019, 567(7748):405-408 Biosci Rep, 2017, 37(4) Leuk Res, 2017, 57:9-19	CFTR attenuated ox-LDL-induced NF-
S7329	IOWH032 IOWH032是一种合成的CFTR抑制剂，在CHO-CFTR细胞测定中， IC50为1.01 μM。 Phase 2。
S8094	GlyH-101 GlyH-101是一种选择性且可逆的CFTR抑制剂，其K_i为4.3 μM。
S1144	Ivacaftor (VX-770) Ivacaftor (VX-770)是一种CFTR的选择性增强剂，靶向作用于G551D-CFTR和F508del-CFTR，在fisher大鼠甲状腺细胞中EC50分别为100 nM和25 nM。	Sci Transl Med, 2014, 6(246):246ra96 Am J Respir Crit Care Med, 2012, 186(7):694-6 Hepatology, 2018, 67(3):972-988	(A) CFTR Western blot of normal (NL) and CF HBE cultures treated with VX-809 (5 uM) ± VX-770 (5 uM) for 48 hours. “*” indicates the mature, complex glycosylated form of CFTR, band C; “•” indicates the immature band B. (B) Turnover of rescued ΔF508 in BHK-21 cells. ΔF508 was rescued at 27°C in the presence of VX-809 ± VX-770 for 24 hours. After adding cycloheximide (200 ug/ml, 37°C), cells were lysed at the indicated times and analyzed by Western blotting.
S8698^New	GLPG1837 GLPG1837是新型的CFTR增效剂，对F508del的EC50为3 nM。相较于Ivacaftor，它对具有III类突变的CFTR突变体具有增强效应。
S1565	VX-809 (Lumacaftor) VX-809 (Lumacaftor)通过促进突变型CFTR(F508del-CFTR)的成熟，从而纠正囊性纤维症中常见的CFTR突变，在fisher大鼠甲状腺细胞中EC50为0.1 μM。Phase 3。	Nat Med, 2013, 19(7):939-45 Sci Transl Med, 2014, 6(246):246ra96 Nat Protoc, 2015, 10(3):363-81	RAF1 and CAMKK1 single siRNA validation in flow cytometry. RAF1 and CAMKK1 single Dharmacon ON-TARGETplus siRNAs with or without VX-809 treatment. Measured surface CFTR fluorescence with a BD Accuri flow cytometer with Intellicyt HTFC. The grayed out region represents VX-809s average surface fluorescence. VX-809 + siRNA significance is shown from VX-809 negative control. Data are shown as mean ± SEM (3−4 replicates). One-way ANOVA with multiple comparison for significant changes from VX-809 neg. ctrl (0 nM) (red P-values). *P ≤ 0.001; P ≤ 0.01; *P ≤ 0.05.
S7059	Tezacaftor (VX-661) Tezacaftor (VX-661)是第二个F508del CFTR校正药物，协助CFTR蛋白到达细胞表面。Phase 2。	Sci Transl Med, 2014, 6(246):246ra96 NPJ Genom Med, 2017, 10.1038/s41525-017-0015-6 Sci Rep, 2018, 8(1):11404	(B)ΔISC response to forskolin observed in VX-661-treated CF HBE cells (P = 0.0021, VX661 vs. vehicle) was prevented by chronic VX-770 treatment and significantly different from VX-661-treated cells (#P=0.0126, VX661 vs. VX661+VX770). (C) The response to CFTR inh-172 that was observed in VX-661-treated cells (P = 0.0111, VX661 vs. vehicle) was significantly decreased in VX809+VX770 treated cells (#P = 0.0038, VX661 vs. VX661+VX770). Primary CF HBE cultures (ΔF508/ΔF508) were derived from 3 different patients, and 3-4 replicates were performed per patient.

产品目录	产品描述	文献引用	实验数据
S6003	Ataluren (PTC124) Ataluren (PTC124)选择性诱导核糖体转录通读，但不影响正常的终止密码子，在HEK293细胞中EC50为0.1 μM，可以治疗nonsense mutations (如CFTR无义突变引起的CF)造成的家族遗传性疾病。 Phase 3。	J Clin Invest, 2013, 124(1):111-6 FASEB J, 2013, 28(4):1593-9 Hum Mutat, 2012, 33(6):973-80	Effect of systemic ataluren treatment on mice with the Pax6^Sey+/- phenotype. The black arrowhead indicates the lenticular stalk; the black arrow indicates the cornea; and the asterisk indicates the ciliary margin. WT, Pax6^+/+; Mt, Pax6^Sey/+; L, lens; r, retina. Original magnification, x5.
S7139	CFTRinh-172 CFTRinh-172是电势差不依赖性的选择性CFTR抑制剂，K_i为300 nM，对于MDR1，ATP敏感性钾离子通道或者一系列的转运蛋白没有效果。	Nature, 2019, 567(7748):405-408 Biosci Rep, 2017, 37(4) Leuk Res, 2017, 57:9-19	CFTR attenuated ox-LDL-induced NF-
S7329	IOWH032 IOWH032是一种合成的CFTR抑制剂，在CHO-CFTR细胞测定中， IC50为1.01 μM。 Phase 2。
S8094	GlyH-101 GlyH-101是一种选择性且可逆的CFTR抑制剂，其K_i为4.3 μM。

产品目录	产品描述	文献引用	实验数据
S1144	Ivacaftor (VX-770) Ivacaftor (VX-770)是一种CFTR的选择性增强剂，靶向作用于G551D-CFTR和F508del-CFTR，在fisher大鼠甲状腺细胞中EC50分别为100 nM和25 nM。	Sci Transl Med, 2014, 6(246):246ra96 Am J Respir Crit Care Med, 2012, 186(7):694-6 Hepatology, 2018, 67(3):972-988	(A) CFTR Western blot of normal (NL) and CF HBE cultures treated with VX-809 (5 uM) ± VX-770 (5 uM) for 48 hours. “*” indicates the mature, complex glycosylated form of CFTR, band C; “•” indicates the immature band B. (B) Turnover of rescued ΔF508 in BHK-21 cells. ΔF508 was rescued at 27°C in the presence of VX-809 ± VX-770 for 24 hours. After adding cycloheximide (200 ug/ml, 37°C), cells were lysed at the indicated times and analyzed by Western blotting.
S8698^New	GLPG1837 GLPG1837是新型的CFTR增效剂，对F508del的EC50为3 nM。相较于Ivacaftor，它对具有III类突变的CFTR突变体具有增强效应。

产品目录

产品描述

文献引用

实验数据

S1144

Ivacaftor (VX-770)

Ivacaftor (VX-770)是一种CFTR的选择性增强剂，靶向作用于G551D-CFTR和F508del-CFTR，在fisher大鼠甲状腺细胞中EC50分别为100 nM和25 nM。

Sci Transl Med, 2014, 6(246):246ra96

Am J Respir Crit Care Med, 2012, 186(7):694-6

Hepatology, 2018, 67(3):972-988

S8698^New

GLPG1837

GLPG1837是新型的CFTR增效剂，对F508del的EC50为3 nM。相较于Ivacaftor，它对具有III类突变的CFTR突变体具有增强效应。

产品目录	产品描述	文献引用	实验数据
S1565	VX-809 (Lumacaftor) VX-809 (Lumacaftor)通过促进突变型CFTR(F508del-CFTR)的成熟，从而纠正囊性纤维症中常见的CFTR突变，在fisher大鼠甲状腺细胞中EC50为0.1 μM。Phase 3。	Nat Med, 2013, 19(7):939-45 Sci Transl Med, 2014, 6(246):246ra96 Nat Protoc, 2015, 10(3):363-81	RAF1 and CAMKK1 single siRNA validation in flow cytometry. RAF1 and CAMKK1 single Dharmacon ON-TARGETplus siRNAs with or without VX-809 treatment. Measured surface CFTR fluorescence with a BD Accuri flow cytometer with Intellicyt HTFC. The grayed out region represents VX-809s average surface fluorescence. VX-809 + siRNA significance is shown from VX-809 negative control. Data are shown as mean ± SEM (3−4 replicates). One-way ANOVA with multiple comparison for significant changes from VX-809 neg. ctrl (0 nM) (red P-values). *P ≤ 0.001; P ≤ 0.01; *P ≤ 0.05.
S7059	Tezacaftor (VX-661) Tezacaftor (VX-661)是第二个F508del CFTR校正药物，协助CFTR蛋白到达细胞表面。Phase 2。	Sci Transl Med, 2014, 6(246):246ra96 NPJ Genom Med, 2017, 10.1038/s41525-017-0015-6 Sci Rep, 2018, 8(1):11404	(B)ΔISC response to forskolin observed in VX-661-treated CF HBE cells (P = 0.0021, VX661 vs. vehicle) was prevented by chronic VX-770 treatment and significantly different from VX-661-treated cells (#P=0.0126, VX661 vs. VX661+VX770). (C) The response to CFTR inh-172 that was observed in VX-661-treated cells (P = 0.0111, VX661 vs. vehicle) was significantly decreased in VX809+VX770 treated cells (#P = 0.0038, VX661 vs. VX661+VX770). Primary CF HBE cultures (ΔF508/ΔF508) were derived from 3 different patients, and 3-4 replicates were performed per patient.

产品目录

产品描述

文献引用

实验数据

S1565