genz-682452 Chemical Properties

Density 

1.30±0.1 g/cm3(Predicted)

storage temp. 

Store at -20°C

solubility 

DMSO:37.5(Max Conc. mg/mL);96.28(Max Conc. mM)

form 

A crystalline solid

pka

10.34±0.46(Predicted)

color 

White to off-white

InChI

InChI=1S/C20H24FN3O2S/c1-20(2,17-12-27-18(22-17)14-3-5-15(21)6-4-14)23-19(25)26-16-11-24-9-7-13(16)8-10-24/h3-6,12-13,16H,7-11H2,1-2H3,(H,23,25)/t16-/m1/s1

InChIKey

YFHRCLAKZBDRHN-MRXNPFEDSA-N

SMILES

C(O[C@H]1C2CCN(CC2)C1)(=O)NC(C1=CSC(C2=CC=C(F)C=C2)=N1)(C)C

genz-682452 Usage And Synthesis

Uses

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].

References

[1] 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. DOI:10.1038/s41598-021-00404-5
[2] Peterschmitt MJ, et al. Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers. Clin Pharmacol Drug Dev. 2021;10(1):86-98. DOI:10.1002/cpdd.865
[3] 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 DOI:10.1007/s00441-017-2704-y
[4] 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. DOI:10.1007/s10545-014-9724-5

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