SML4003
TP-060
≥98% (HPLC)
别名:
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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关于此项目
经验公式（希尔记法）:
C24H20F4N2O3
化学文摘社编号:
分子量:
460.42
UNSPSC Code:
12352202
MDL number:
NACRES:
NA.21
InChI key
OAZAPIKYVGRNCO-UHFFFAOYSA-N
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
assay
≥98% (HPLC)
form
powder
color
white to beige
solubility
DMSO: 2 mg/mL, clear (Warmed)
storage temp.
2-8°C
Quality Level
Biochem/physiol Actions
Orally active, brain-penetrant, substrate-noncompetitive glucosylceramide synthase (GCS) inhibitor with therapeutic efficacy in a murine Gaucher’s disease (GD) model in vivo.
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.).
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.).
存储类别
11 - Combustible Solids
flash_point_f
Not applicable
flash_point_c
Not applicable
法规信息
新产品
此项目有
Yuta Tanaka et al.
Journal of medicinal chemistry, 65(5), 4270-4290 (2022-02-22)
Inhibition of glucosylceramide synthase (GCS) is a major therapeutic strategy for Gaucher's disease and has been suggested as a potential target for treating Parkinson's disease. Herein, we report the discovery of novel brain-penetrant GCS inhibitors. Assessment of the structure-activity relationship
Junsi Wang et al.
Bioorganic & medicinal chemistry letters, 77, 129039-129039 (2022-11-08)
Glucosylceramide synthase (GCS) has drawn much attention as an attractive protein target in the disease pathways of Parkinson's Disease (PD) and lysosomal storage disorders, such as Gaucher's Disease (GD). In previous our study, T-036 and its analogue, 2a, were discovered
Takahiro Fujii et al.
Journal of neurochemistry, 159(3), 543-553 (2021-08-17)
Gaucher disease (GD), the most common lysosomal storage disorders, is caused by GBA gene mutations resulting in glycosphingolipids accumulations in various tissues, such as the brain. While suppressing glycosphingolipid accumulation is the central strategy for treating peripheral symptoms of GD
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