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[CAS NO. 1401090-53-6] Ibiglustat
| Ships within | Stock | Price | Qty | Total |
Please click "REQUEST A QUOTE" button if there is no stock, or you need other sizes or custom synthesis.
| Catalog: | HY-16743 |
| Brand: | MCE |
| CAS: | 1401090-53-6 |
Overview
| MDL | MFCD28502073 |
|---|---|
| Molecular Weight | 389.49 |
| Molecular Formula | C20H24FN3O2S |
| SMILES | O=C(O[C@@H]1CN2CCC1CC2)NC(C)(C3=CSC(C4=CC=C(F)C=C4)=N3)C |
1 Publications Citing Use of MCE
Summary
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] .
IC50 & Target
Glucosylceramide synthase [1] .
In Vitro
Ibiglustat (SAR402671) (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 can prevent additional GL-3 accumulation and could serve to ameliorate the abundant levels of this sphingolipid in FD cardiomyocytes [4]
MCE has not independently confirmed the accuracy of these methods. They are for reference only.
Clinical Trial
| NCT Number | Sponsor | Condition | Start Date | Phase |
|---|---|---|---|---|
| NCT05238714 | Genzyme, a Sanofi Company|Sanofi |
Disorders of Sphingolipid Metabolism (Healthy Volunteers)
|
May 26, 2020 | Phase 1 |
| NCT03523728 | Genzyme, a Sanofi Company|Sanofi |
Polycystic Kidney, Autosomal Dominant
|
October 4, 2018 | Phase 2|Phase 3 |
| NCT02906020 | Genzyme, a Sanofi Company|Sanofi |
Parkinson´s Disease
|
December 15, 2016 | Phase 2 |
| NCT04705051 | Sanofi |
Congenital Cystic Kidney Disease
|
February 9, 2021 | Phase 3 |
| NCT05222906 | Genzyme, a Sanofi Company|Sanofi |
Gaucher´s Disease Type III
|
April 18, 2022 | Phase 3 |
| NCT05206773 | Genzyme, a Sanofi Company|Sanofi |
Fabry Disease
|
March 11, 2022 | Phase 3 |
| NCT05280548 | Genzyme, a Sanofi Company|Sanofi |
Fabry´s Disease
|
May 3, 2022 | Phase 3 |
| NCT03687554 | Genzyme, a Sanofi Company|Sanofi |
Healthy Volunteers|Polycystic Kidney, Autosomal Dominant
|
October 5, 2018 | Phase 1 |
| NCT02843035 | Genzyme, a Sanofi Company|Sanofi |
Gaucher Disease Type 1|Gaucher Disease Type 3
|
January 4, 2017 | Phase 2 |
| NCT04221451 | Genzyme, a Sanofi Company|Sanofi |
Tay-Sachs Disease Sandhoff Disease
|
June 29, 2020 | Phase 3 |
Appearance
Solid
Shipping
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 |
Solvent & Solubility
DMSO : 50 mg/mL ( 128.37 mM ; Need ultrasonic)
Stock Solutions
| Concentration Solvent Mass | 1 mg | 5 mg | 10 mg |
|---|
| 1 mM | 2.5675 mL | 12.8373 mL | 25.6746 mL |
| 5 mM | 0.5135 mL | 2.5675 mL | 5.1349 mL |
| 10 mM | 0.2567 mL | 1.2837 mL | 2.5675 mL |
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.
- [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] 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
- [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.
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