| Identification | Back Directory | [Name]
OGT2378 | [CAS]
441061-33-2 | [Synonyms]
OGT2378
ACT-519276
Sinbaglustat
3,4,5-Piperidinetriol, 2-(hydroxymethyl)-1-pentyl-, (2S,3R,4R,5S)- | [Molecular Formula]
C11H23NO4 | [MDL Number]
MFCD07772309 | [MOL File]
441061-33-2.mol | [Molecular Weight]
233.3 |
| Chemical Properties | Back Directory | [Boiling point ]
405.5±45.0 °C(Predicted) | [density ]
1.201±0.06 g/cm3(Predicted) | [form ]
Solid | [pka]
13.71±0.70(Predicted) | [color ]
Light yellow to yellow |
| Hazard Information | Back Directory | [Uses]
Sinbaglustat (OGT2378) is a dual inhibitor of glucosylceramide synthase (GCS) and non-lysosomal glucosyl ceramidase (GBA2). Sinbaglustat is an orally available N-alkyl iminosugar that crosses the blood-brain barrier. Sinbaglustat can be used for the research of central neurodegenerative diseases associated with lysosomal dysfunctions[1][2]. | [in vivo]
Sinbaglustat (OGT2378; administered p.o., in the powdered chow, at a dose of 2500 mg/kg/day, corresponding to 35-40 mg of Sinbaglustat per mouse per day) is highly effective in impeding melanoma tumor growth?in vivo. The effectiveness of p.o. Sinbaglustat in this murine model suggests that inhibition of glycosphingolipid synthesis is a promising approach to inhibit tumor progression[1]. | Animal Model: | Female syngeneic C57BL/6 mice, 6-8 weeks old bearing MEB4 melanoma tumor[1] | | Dosage: | 35-40 mg per mouse per day | | Administration: | Administered p.o., in the powdered chow, at a dose of 2500 mg/kg/day | | Result: | Inhibited MEB4 melanoma tumor growth in a syngeneic, orthotopic murine model.
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| [References]
[1] Michael Weiss,?et al. Inhibition of melanoma tumor growth by a novel inhibitor of glucosylceramide synthase. Cancer Res.?2003 Jul 1;63(13):3654-8. PMID:12839955
[2] Martine Gehin,?et al. Assessment of Target Engagement in a First-in-Human Trial with Sinbaglustat, an Iminosugar to Treat Lysosomal Storage Disorders. Clin Transl Sci.?2021 Mar;14(2):558-567. DOI:10.1111/cts.12911 |
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