Eliglustat hemitartrate

本公司产品仅用于科研实验，不得用于人体或动物的临床与诊断

Eliglustat Hemitartrate 的 IC50 为 24 nM，是一种特异性、有效、口服活性的葡萄糖脑糖苷酶抑制剂。Eliglustat tartrate 显示出良好的效力，IC50 为 24 nM，并且对目标酶具有特异性。一种新型的葡萄糖神经酰胺合酶抑制剂 (Genz-112638) ）并评估其在戈谢病小鼠模型（D409V/null）中的活性。 Genz-112638 的生化表征显示出良好的效力（IC(50) 约为 24nM）和针对靶酶的特异性。

Eliglustat Hemitartrate, with an IC50 of 24 nM, is a specific, potent, oral active glucocerebral glycosidase inhibitor.Eliglustat tartrate shows good potency with an IC50 of 24 nM and specificity against the target enzyme.A novel inhibitor of glucosylceramide synthase (Genz-112638) and assessed its activity in a murine model of Gaucher disease (D409V/null).?Biochemical characterization of Genz-112638 showed good potency (IC(50) approximately 24nM) and specificity against the target enzyme.?Mice that received drug prior to significant accumulation of substrate (10 weeks of age) showed reduced levels of glucosylceramide and number of Gaucher cells in the spleen, lung and liver when compared to age-matched control animals.?Treatment of older mice that already displayed significant amounts of tissue glucosylceramide (7 months old) resulted in arrest of further accumulation of the substrate and appearance of additional Gaucher cells in affected organs.?These data indicate that substrate inhibition therapy with Genz-112638 represents a viable alternate approach to enzyme therapy to treat the visceral pathology in Gaucher disease.

Eliglustat tartrate shows good potency with an IC50 of 24 nM and specificity against the target enzyme.Incubating K562 or B16/F10 cells for 72 h with increasing amounts of Genz-112638 (0.6-1000 nM) results in a dose-dependent reduction of cell surface levels of both GM1 and GM3. The mean IC50 value for inhibiting the cell surface presentation of GM1 in K562 cells was 24 nM (range 14-34 nM) and that for GM3 in B16/F10 cells was 29 nM (range 12-48 nM).

Mice that received drug prior to significant accumulation of substrate (10 weeks of age) showed reduced levels of glucosylceramide and number of Gaucher cells in the spleen, lung and liver when compared to age-matched control animals.

Genz-112638,Eliglustat tartrate

Others

Other Targets

glucocerebroside synthase

Metabolism

Gaucher′s Disease

928659-70-5

959.17

C50H78N4O14

>98% (HPLC)

H2O:50 mg/mL (52.13 mM);DMSO:50 mg/mL (52.13 mM)

CCCCCCCC(N[C@H](CN1CCCC1)[C@@H](C2=CC=C(OCCO3)C3=C2)O)=O.CCCCCCCC(N[C@H](CN4CCCC4)[C@@H](C5=CC=C(OCCO6)C6=C5)O)=O.O=C(O)[C@H](O)[C@@H](O)C(O)=O

——

(-20℃)

With Ice Pack

≥ 2 years