Glucosylceramide synthase-IN-1 (C09-1135-440)

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 researchIn VitroGlucosylceramide 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). MCE has not independently confirmed the accuracy of these methods. They are for reference only.In VivoGlucosylceramide 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 . MCE has not independently confirmed the accuracy of these methods. They are for reference only.Form:SolidIC50& Target:IC50: 31 nM (human GCS) and 51 nM (mouse GCS). Specification: 0.99 Molecular Formula: C24H20F4N2O3 Molecular Weight: 460.42