Glucosylceramide synthase-IN-1_product_DataBase

Bioactivity	Glucosylceramide synthase-IN-1 (T-036) a potent, brain-penetrant and orally active glucosylceramide synthase (GCS) inhibitor with IC50s 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].
Target	IC50: 31 nM (human GCS) and 51 nM (mouse GCS)
Invitro	Glucosylceramide synthase-IN-1 (T-036) potently reduces the GCS product, catalyze glucosylceramide (GlcCer), in the fibroblasts with Gaucher’s disease (EC50 of 7.6 nM)[1].
In Vivo	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].
Name	Glucosylceramide synthase-IN-1
CAS	2601393-20-6
Formula	C24H20F4N2O3
Molar Mass	460.42
Appearance	Solid
Transport	Room temperature in continental US; may vary elsewhere.
Storage	4°C, sealed storage, away from moisture and light *In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture and light)
Reference	[1]. Yuta Tanaka, et al. Discovery of Brain-Penetrant Glucosylceramide Synthase Inhibitors with a Novel Pharmacophore. J Med Chem. 2022 Mar 10;65(5):4270-4290

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