Sinbaglustat

• CAT Number: I008403
• CAS Number: 441061-33-2
• Molecular Formula: C11H23NO4
• Molecular Weight: 233.30
• Purity: ≥95%
• Synonyms: (2S,3R,4R,5S)-2-(hydroxymethyl)-1-pentylpiperidine-3,4,5-triol
• InChI: InChI=1S/C11H23NO4/c1-2-3-4-5-12-6-9(14)11(16)10(15)8(12)7-13/h8-11,13-16H,2-7H2,1H3/t8-,9-,10+,11+/m0/s1
• InChIKey: HCZQIIVHWYFIPW-UKKRHICBSA-N
• SMILES: CCCCCN1CC(C(C(C1CO)O)O)O

Sinbaglustat (Cat No.: I008403) is an orally bioavailable inhibitor of glucosylceramide synthase (GCS) and non-lysosomal glucosylceramidase (GBA2), enzymes involved in glycosphingolipid metabolism. By blocking these targets, sinbaglustat reduces the synthesis and accumulation of glycosphingolipids, which are implicated in lysosomal storage disorders and other metabolic diseases. It has been investigated for potential therapeutic applications in Gaucher disease, Fabry disease, and Parkinson’s disease. Sinbaglustat is also used in research to study sphingolipid biology, neurodegeneration, and metabolic regulation, offering a dual-mechanism approach to modulating glycosphingolipid homeostasis.

Reference

[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.
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[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.
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