For research use only. Not for therapeutic Use.
Ibiglustat (Venglustat) L-Malic acid is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. Ibiglustat L-Malic acid 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].
Ibiglustat (SAR402671) (1 μM, 15 days) L-Malic acid treated Fabry disease (FD) cells are close to the physiological level in untreated WT cells in GL-3 levels, suggesting that Ibiglustat L-Malic acid can prevent additional GL-3 accumulation and could serve to ameliorate the abundant levels of this sphingolipid in FD cardiomyocytes[4].
| Catalog Number | I019735 |
| CAS Number | 1629063-78-0 |
| Synonyms | [(3S)-1-azabicyclo[2.2.2]octan-3-yl] N-[2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl]carbamate;(2S)-2-hydroxybutanedioic acid |
| Molecular Formula | C24H30FN3O7S |
| Purity | ≥95% |
| InChI | InChI=1S/C20H24FN3O2S.C4H6O5/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;5-2(4(8)9)1-3(6)7/h3-6,12-13,16H,7-11H2,1-2H3,(H,23,25);2,5H,1H2,(H,6,7)(H,8,9)/t16-;2-/m10/s1 |
| InChIKey | SQXUKOJKIWCALK-AAXLQGCPSA-N |
| SMILES | CC(C)(C1=CSC(=N1)C2=CC=C(C=C2)F)NC(=O)OC3CN4CCC3CC4.C(C(C(=O)O)O)C(=O)O |
| Reference | [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. [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. [3]. Iva Stojkovska, et al. Molecular mechanisms of α-synuclein and GBA1 in Parkinson’s disease. Cell Tissue Res. 2017. [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. |
