This compound is a customized synthesis product. We have a strong synthesis team with excellent synthesis technology and capabilities. However, due to various objective factors, there is a low probability that the synthesis will not be successful. If you need to learn more, please feel free to consult us, we will serve you wholeheartedly.
This compound is a customized synthesis product. We have a strong synthesis team with excellent synthesis technology and capabilities. However, due to various objective factors, there is a low probability that the synthesis will not be successful. If you need to learn more, please feel free to consult us, we will serve you wholeheartedly.
Glucosylceramide synthase-IN-3
Catalog No. T61632
Glucosylceramide synthase-IN-3 (compound BZ1) is a highly potent, brain-penetrant, and orally active inhibitor of glucosylceramide synthase (GCS), exhibiting an IC50 value of 16 nM for human GCS. This compound, designated as Glucosylceramide synthase-IN-3, finds potential applications in Gaucher's disease research [1] [2].
All products from TargetMol are for Research Use Only. Not for Human or Veterinary or Therapeutic Use.
This compound is a customized synthesis product. We have a strong synthesis team with excellent synthesis technology and capabilities. However, due to various objective factors, there is a low probability that the synthesis will not be successful. If you need to learn more, please feel free to consult us, we will serve you wholeheartedly.
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Biological Description
Chemical Properties
Storage
& Solubility Information
Description
Glucosylceramide synthase-IN-3 (compound BZ1) is a highly potent, brain-penetrant, and orally active inhibitor of glucosylceramide synthase (GCS), exhibiting an IC50 value of 16 nM for human GCS. This compound, designated as Glucosylceramide synthase-IN-3, finds potential applications in Gaucher's disease research [1] [2].
In vitro
Glucosylceramide Synthase-IN-2 (compound BZ1) effectively reduces glucosylceramide levels, with cellular IC50 values of 94 nM in humans and 160 nM in mice, as confirmed through a fibroblast assay [1]. Additionally, it exhibits a potent IC50 of 20 nM in primary neurons [1]. Compound BZ1 demonstrates a dose-dependent decrease in glycosphingolipids across both wild-type and D409V mouse cortical neurons at concentrations of 10, 30, 100, and 300 nM. Moreover, it significantly reduces the levels of detergent-insoluble pS129 α-syn [1], indicating its broad-spectrum efficacy in biological models.
In vivo
Eight hours following administration of a single dose, Glucosylceramide synthase-IN-2 (compound BZ1; administered at 6, 20, or 100 mg/kg through oral gavage and formulated in 30% captisol) demonstrates a dose-dependent reduction in plasma GlcCer C:16:0 levels, achieving a decrease of approximately 75% compared to vehicle-treated animals. Additionally, this compound significantly lowers brain GlcCer levels to about 48% relative to vehicle-treated controls in male C57BL6 mice aged 8 weeks. Extended administration of Glucosylceramide synthase-IN-2 (6, 20, or 100 mg/kg/day over four days) results in even greater reductions in GlcCer levels. The compound also exhibits favorable pharmaceutical properties, including high permeability (pApp=26.54) and a lack of P-gp substrate activity.
Molecular Weight
381.4
Formula
C21H20FN3O3
Storage
Powder: -20°C for 3 years | In solvent: -80°C for 1 year
Dose Conversion
You can also refer to dose conversion for different animals.
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Method for preparing DMSO master liquid: mg
drug pre-dissolved in μL DMSO (Master liquid concentration
mg/mL),
Method for preparing in vivo formulation:Take μL
DMSO master liquid, next add μL PEG300, mix and clarify, next add μL
Tween 80,mix and clarify, next add μL ddH2O, mix and clarify.
Method for preparing in vivo formulation:Take μL
DMSO master liquid, next add μL Corn oil,mix and clarify.
Note:
Be sure to add the solvent(s) in order. Physical methods such as vortex, ultrasound or hot water bath can be used to aid dissolving.
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Tech Support
Please see Inhibitor Handling Instructions for more frequently ask questions. Topics include: how to prepare stock solutions, how to store products, and cautions on cell-based assays & animal experiments, etc.