Ibiglustat

Ibiglustat (Venglustat) is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. Ibiglustat can be used for the research of Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease[1][2].

Product Specifications

CAS Number

[1401090-53-6]

Product Name Alternative

Venglustat; SAR402671; GZ402671

UNSPSC

12352005

Hazard Statement

H302, H315, H319, H335

Target

Glucosylceramide Synthase (GCS)

Type

Reference compound

Related Pathways

Neuronal Signaling

Applications

Metabolism-sugar/lipid metabolism

Field of Research

Metabolic Disease

Assay Protocol

https://www.medchemexpress.com/Ibiglustat.html

Purity

99.76

Solubility

DMSO : 50 mg/mL (ultrasonic)

Smiles

O=C(O[C@@H]1CN2CCC1CC2)NC(C)(C3=CSC(C4=CC=C(F)C=C4)=N3)C

Molecular Formula

C20H24FN3O2S

Molecular Weight

389.49

Precautions

H302, H315, H319, H335

References & Citations

[1]Stojkovska I, et al. Molecular mechanisms of α-synuclein and GBA1 in Parkinson's disease. Cell Tissue Res. 2018;373 (1) :51-60. doi:10.1007/s00441-017-2704-y|[2]Itier JM, et al. Effective clearance of GL-3 in a human iPSC-derived cardiomyocyte model of Fabry disease. J Inherit Metab Dis. 2014 Nov;37 (6) :1013-22.|[3]Viel C, et al. Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model. Sci Rep. 2021;11 (1) :20945. Published 2021 Oct 22. |[4]Peterschmitt MJ, et al. Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers. Clin Pharmacol Drug Dev. 2021;10 (1) :86-98.