Glucosylceramide synthase-IN-1 (T-036) 是一种有效的、可穿过血脑屏障的和具有口服活性的glucosylceramide synthase (GCS)抑制剂，对人 GCS 和小鼠 GCS 的IC50分别为 31 nM 和 51 nM。Glucosylceramide synthase-IN-1 可用于戈谢病研究。

Glucosylceramide synthase-IN-1 (T-036) a potent, brain-penetrant and orally active glucosylceramide synthase (GCS) inhibitor with IC 50 s of 31 nM and 51 nM for human GCS and mouse GCS, respectively. Glucosylceramide synthase-IN-1 can be used for Gaucher's disease research [1].

Glucosylceramide synthase-IN-1 (T-036) potently reduces the GCS product, catalyze glucosylceramide (GlcCer), in the fibroblasts with Gaucher’s disease (EC 50 of 7.6 nM) [1].

Glucosylceramide synthase-IN-1 (T-036) has good oral exposure (BA = 67%) and moderate brain penetration ( Kpuu,brain = 0.11). Administration of a single dose of Glucosylceramide synthase-IN-1 (T-036) reduces GlcCer in the plasma and cerebral cortex of wild-type mice, and administration of Glucosylceramide synthase-IN-1 (T-036) for 2 months significantly reduces glucosylsphingosine (GlcSph) in the cerebral cortex of the Gaucher’s disease mouse model [1].

2601393-20-6

C24H20F4N2O3

460.42

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