Glucosylceramide synthase-IN-3 (compound T-690) 是一种有效的、可透过血脑屏障的和具有口服活性的glucosylceramide synthase (GCS)抑制剂，对人 GCS 的IC50为 16 nM。Glucosylceramide synthase-IN-3 可用于戈谢病研究。

Glucosylceramide synthase-IN-3 (compound BZ1) is a potent, brain-penetrant and orally active glucosylceramide synthase (GCS) inhibitor with IC 50 s of 16 nM for human GCS.Glucosylceramide synthase-IN-3 can be used for Gaucher's disease research [1] [2].

Glucosylceramide synthase-IN-2 (compound BZ1) causes measuring the reduction of glucosylceramide and the cellular IC50 was determined to be 94 nM in human and 160 nM in mouse with cellular activity was confirmed using a fibroblast assay [1]. Glucosylceramide synthase-IN-2 has the IC50 of 20 nM in primary neurons [1]. Glucosylceramide synthase-IN-2 (10, 30, 100, 300 nM) produces a dose-dependent reduction in glycosphingolipids in WT and D409V mouse cortical neurons. Glucosylceramide synthase-IN-2 decreases the amount of detergent-insoluble pS129 α-syn [1].

Eight hours after a single dose of Glucosylceramide synthase-IN-2 (compound BZ1; 6, 20 or 100 mg/kg; oral gavage; formulated in 30% captisol), plasma GlcCer C:16:0 is reduced in a dose-dependent fashion up to ~75% of concentration in vehicle treated animals. Brain GlcCer is also significantly reduced to concentrations of ~48% of vehicle treated controls in C57BL6 mice (8 weeks of age, male) [1]. Glucosylceramide synthase-IN-2 (6, 20 or 100 mg/kg/day for 4 days; oral gavage) causes larger reductions in GlcCer [1]. Glucosylceramide synthase-IN-2 has good pharmaceutical properties with high permeability (pApp=26.54) and is not a substrate of P-gp [1].

C21H20FN3O3

381.4

(< 1 mg/ml refers to the product slightly soluble or insoluble )
Powder: -20°C for 3 years
In solvent: -80°C for 2 years