Ibiglustat
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MedKoo CAT#: 319547

CAS#: 1401090-53-6 (free base)

Description: Venglustat, also known as Ibiglustat, GZ402671, GZ-452; Genz-682452 and SAR402671, is a potent and selective Glucosylceramide synthase inhibitor and ceramide glucosyltransferase inhibitor. Ibiglustat blocks the formation of glucosylceramide (GL-1), a key intermediate in the synthesis of GL-3. Ibiglustat is potentially useful for treating Fabry disease. Fabry disease is a rare lysosomal storage disorder, which results in abnormal tissue deposits of a particular fatty substance called globotriaosylceramide (GL-3 or Gb3) throughout the body.


Chemical Structure

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Ibiglustat
CAS# 1401090-53-6 (free base)

Theoretical Analysis

MedKoo Cat#: 319547
Name: Ibiglustat
CAS#: 1401090-53-6 (free base)
Chemical Formula: C20H24FN3O2S
Exact Mass: 389.16
Molecular Weight: 389.490
Elemental Analysis: C, 61.68; H, 6.21; F, 4.88; N, 10.79; O, 8.22; S, 8.23

Price and Availability

Size Price Availability Quantity
10mg USD 150 Ready to ship
50mg USD 450 Ready to ship
100mg USD 750 Ready to ship
200mg USD 1250 Ready to ship
500mg USD 2450 Ready to ship
1g USD 3650 Ready to ship
2g USD 6450 Ready to ship
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Related CAS #: 1401090-53-6 (free base)   1629063-79-1 (HCl)   1629063-80-4 (succinic acid)   1629063-78-0 (malate)  

Synonym: GZ-402671; GZ402671; GZ 402671; SAR402671; SAR-402671; SAR 402671; Genz-682452-AA; GZ-452; Genz-682452; Ibiglustat; Venglustat;

IUPAC/Chemical Name: (3S)-1-azabicyclo[2.2.2]octan-3-yl N-{2-[2-(4-fluorophenyl)- 1,3-thiazol-4-yl]propan-2-yl}carbamate

InChi Key: YFHRCLAKZBDRHN-MRXNPFEDSA-N

InChi Code: 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

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

Appearance: Solid powder

Purity: >97% (or refer to the Certificate of Analysis)

Shipping Condition: Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.

Storage Condition: Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).

Solubility: Soluble in DMSO, not in water

Shelf Life: >2 years if stored properly

Drug Formulation: This drug may be formulated in DMSO

Stock Solution Storage: 0 - 4 C for short term (days to weeks), or -20 C for long term (months).

HS Tariff Code: 2934.99.9001

More Info: Related CAS# 1401090-53-6 1629063-78-0 (

Biological target: Ibiglustat is a selective, allosteric inhibitor of glucosylceramide synthase (GCS) with ability to cross the blood-brain barrier.
In vitro activity: TBD
In vivo activity: TBD

Solubility Data

Solvent Max Conc. mg/mL Max Conc. mM
Solubility
DMSO 37.5 96.28

Preparing Stock Solutions

The following data is based on the product molecular weight 389.49 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.

Recalculate based on batch purity %
Concentration / Solvent Volume / Mass 1 mg 5 mg 10 mg
1 mM 1.15 mL 5.76 mL 11.51 mL
5 mM 0.23 mL 1.15 mL 2.3 mL
10 mM 0.12 mL 0.58 mL 1.15 mL
50 mM 0.02 mL 0.12 mL 0.23 mL
Formulation protocol: TBD
In vitro protocol: TBD
In vivo protocol: TBD

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1: Peterschmitt MJ, Saiki H, Hatano T, Gasser T, Isaacson SH, Gaemers SJM, Minini P, Saubadu S, Sharma J, Walbillic S, Alcalay RN, Cutter G, Hattori N, Höglinger GU, Marek K, Schapira AHV, Scherzer CR, Simuni T, Giladi N, Sardi SP, Fischer TZ; MOVES-PD Investigators. Safety, Pharmacokinetics, and Pharmacodynamics of Oral Venglustat in Patients with Parkinson's Disease and a GBA Mutation: Results from Part 1 of the Randomized, Double-Blinded, Placebo- Controlled MOVES-PD Trial. J Parkinsons Dis. 2022;12(2):557-570. doi: 10.3233/JPD-212714. PMID: 34897099; PMCID: PMC8925113.


2: Viel C, Clarke J, Kayatekin C, Richards AM, Chiang MSR, Park H, Wang B, Shihabuddin LS, Sardi SP. Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model. Sci Rep. 2021 Oct 22;11(1):20945. doi: 10.1038/s41598-021-00404-5. PMID: 34686711; PMCID: PMC8536659.


3: Peng Y, Liou B, Lin Y, Fannin V, Zhang W, Feldman RA, Setchell KDR, Grabowski GA, Sun Y. Substrate Reduction Therapy Reverses Mitochondrial, mTOR, and Autophagy Alterations in a Cell Model of Gaucher Disease. Cells. 2021 Sep 2;10(9):2286. doi: 10.3390/cells10092286. PMID: 34571934; PMCID: PMC8466461.


4: Nicoli ER, Annunziata I, d'Azzo A, Platt FM, Tifft CJ, Stepien KM. GM1 Gangliosidosis-A Mini-Review. Front Genet. 2021 Sep 3;12:734878. doi: 10.3389/fgene.2021.734878. PMID: 34539759; PMCID: PMC8446533.


5: Vitner EB, Achdout H, Avraham R, Politi B, Cherry L, Tamir H, Yahalom-Ronen Y, Paran N, Melamed S, Erez N, Israely T. Glucosylceramide synthase inhibitors prevent replication of SARS-CoV-2 and influenza virus. J Biol Chem. 2021 Jan- Jun;296:100470. doi: 10.1016/j.jbc.2021.100470. Epub 2021 Feb 25. PMID: 33639165; PMCID: PMC7904475.


6: Wilson MW, Shu L, Hinkovska-Galcheva V, Jin Y, Rajeswaran W, Abe A, Zhao T, Luo R, Wang L, Wen B, Liou B, Fannin V, Sun D, Sun Y, Shayman JA, Larsen SD. Optimization of Eliglustat-Based Glucosylceramide Synthase Inhibitors as Substrate Reduction Therapy for Gaucher Disease Type 3. ACS Chem Neurosci. 2020 Oct 21;11(20):3464-3473. doi: 10.1021/acschemneuro.0c00558. Epub 2020 Oct 9. PMID: 33035424; PMCID: PMC7919060.


7: Schneider SA, Hizli B, Alcalay RN. Emerging Targeted Therapeutics for Genetic Subtypes of Parkinsonism. Neurotherapeutics. 2020 Oct;17(4):1378-1392. doi: 10.1007/s13311-020-00920-8. PMID: 32914362; PMCID: PMC7483040.


8: Peterschmitt MJ, Crawford NPS, Gaemers SJM, Ji AJ, Sharma J, Pham TT. Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers. Clin Pharmacol Drug Dev. 2021 Jan;10(1):86-98. doi: 10.1002/cpdd.865. Epub 2020 Aug 26. PMID: 32851809; PMCID: PMC7818513.


9: van der Veen SJ, Hollak CEM, van Kuilenburg ABP, Langeveld M. Developments in the treatment of Fabry disease. J Inherit Metab Dis. 2020 Sep;43(5):908-921. doi: 10.1002/jimd.12228. Epub 2020 Mar 2. PMID: 32083331; PMCID: PMC7540041.


10: Schneider SA, Alcalay RN. Precision medicine in Parkinson's disease: emerging treatments for genetic Parkinson's disease. J Neurol. 2020 Mar;267(3):860-869. doi: 10.1007/s00415-020-09705-7. Epub 2020 Jan 23. PMID: 31974807; PMCID: PMC7035220.


11: Marshall J, Nietupski JB, Park H, Cao J, Bangari DS, Silvescu C, Wilper T, Randall K, Tietz D, Wang B, Ying X, Leonard JP, Cheng SH. Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity. Mol Ther. 2019 Aug 7;27(8):1495-1506. doi: 10.1016/j.ymthe.2019.05.018. Epub 2019 Jun 4. PMID: 31208914; PMCID: PMC6697407.


12: Marshall J, Sun Y, Bangari DS, Budman E, Park H, Nietupski JB, Allaire A, Cromwell MA, Wang B, Grabowski GA, Leonard JP, Cheng SH. CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease. Mol Ther. 2016 Jun;24(6):1019-1029. doi: 10.1038/mt.2016.53. Epub 2016 Mar 7. PMID: 26948439; PMCID: PMC4923322.


13: Ashe KM, Budman E, Bangari DS, Siegel CS, Nietupski JB, Wang B, Desnick RJ, Scheule RK, Leonard JP, Cheng SH, Marshall J. Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease. Mol Med. 2015 Apr 30;21(1):389-99. doi: 10.2119/molmed.2015.00088. PMID: 25938659; PMCID: PMC4559530.