Ibiglustat succinate_product_DataBase

Bioactivity

Ibiglustat (Venglustat) succinate is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. Ibiglustat succinate 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].

Invitro

Ibiglustat (SAR402671) succinate (1 μM, 15 days; Fabry disease (FD) cells) is close to the physiological level in untreated WT cells in GL-3 levels, suggesting that Ibiglustat succinate can prevent additional GL-3 accumulation and could serve to ameliorate the abundant levels of this sphingolipid in FD cardiomyocytes[4].

Name

Ibiglustat succinate

CAS

1629063-80-4

Formula

C24H30FN3O6S

Molar Mass

507.57

Appearance

Solid

Transport

Room temperature in continental US; may vary elsewhere.

Storage

Powder	-20°C	3 years
	4°C	2 years
In solvent	-80°C	6 months
	-20°C	1 month

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;37(6):1013-1022.

PeptideDB

Ibiglustat succinate

CAS: 1629063-80-4 F: C24H30FN3O6S W: 507.57