SML4003
TP-060
≥98% (HPLC)
Synonym(s):
2-(4-Fluoro-2-(2,2,2-trifluoroethoxy)phenyl)-8-(4-(1-hydroxy-1-methylethyl)phenyl)-3,8-diaza-bicyclo[4.3.0]nona-1(6),2,4-trien-7-one, 4-[4-Fluoro-2-(2,2,2-trifluoroethoxy)phenyl]-2,3-dihydro-2-[4-(1-hydroxy-1-methylethyl)phenyl]-1H-pyrrolo[3,4-c]pyridin-1-one, 4-[4-Fluoro-2-(2,2,2-trifluoroethoxy)phenyl]-2-[4-(2-hydroxypropan-2-yl)phenyl]-2,3-dihydro-1H-pyrrolo[3,4-c]pyridin-1-one, Glucosylceramide synthase-IN-1, T 036, T-036, T036, TP 060, TP060
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About This Item
Quality Level
assay
≥98% (HPLC)
form
powder
color
white to beige
solubility
DMSO: 2 mg/mL, clear (Warmed)
storage temp.
2-8°C
SMILES string
O=C1C2=C(CN1C3=CC=C(C(C)(C)O)C=C3)C(C4=C(OCC(F)(F)F)C=C(F)C=C4)=NC=C2
InChI key
OAZAPIKYVGRNCO-UHFFFAOYSA-N
Biochem/physiol Actions
TP-060 (T-036) is an orally active, brain-penetrant, substrate-noncompetitive, potent and selective glucosylceramide synthase (GCS) inhibitor (human/mouse IC50 = 31/51 nM) that effectively downregulates glucosylceramide (GlcCer) level in GBA mutant human fibroblasts containing mutated glucosylceramidase (IC50 = 7.6 nM). TP-060 is efficacious in lowering both GlcCer and glucosylsphingosine (GlcSph) levels in the plasma and cerebral cortex of Gba D409V KI mice in vivo following a two-month treatment in the murine Gaucher’s disease (GD) model (10 and 30 mg/kg/day p.o.).
Storage Class
11 - Combustible Solids
flash_point_f
Not applicable
flash_point_c
Not applicable
Regulatory Information
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Global Trade Item Number
| SKU | GTIN |
|---|---|
| SML4003-25MG | 04065272154734 |
| SML4003-5MG | 04065272154741 |