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Glycerol phenylbutyrate Sigma Receptor chemical

Cat.No.S6981

Glycerol phenylbutyrate (GT4P, HPN-100M, Ravicti) is a nitrogen-binding agent and acts as a sigma-2 (σ2) receptor ligand with a pKi of 8.02. This compound is a prodrug in which phenylbutyrate (PBA) is released from the glycerol backbone by lipases in the gastrointestinal tract.
Glycerol phenylbutyrate Sigma Receptor chemical Chemical Structure

Chemical Structure

Molecular Weight: 530.65

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Quality Control

Chemical Information, Storage & Stability

Molecular Weight 530.65 Formula

C33H38O6

 Storage (From the date of receipt) 2 years -20°C liquid
CAS No. 611168-24-2 -- Storage of Stock Solutions

Synonyms GT4P, HPN-100M, Ravicti Smiles O=C(CCCC1=CC=CC=C1)OCC(COC(=O)CCCC2=CC=CC=C2)OC(=O)CCCC3=CC=CC=C3

Solubility

In vitro
Batch:

DMSO : 100 mg/mL ( (188.44 mM) Moisture-absorbing DMSO reduces solubility. Please use fresh DMSO.)

Water : Insoluble

Ethanol : Insoluble

Molarity Calculator

Mass Concentration Volume Molecular Weight

In vivo
Batch:

In vivo Formulation Calculator (Clear solution)

Step 1: Enter information below (Recommended: An additional animal making an allowance for loss during the experiment)

mg/kg g μL

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% DMSO % % Tween 80 % ddH2O
%DMSO %

Calculation results:

Working concentration: mg/ml;

Method for preparing DMSO master liquid: mg drug pre-dissolved in μL DMSO ( Master liquid concentration mg/mL, Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug. )

Method for preparing in vivo formulation: Take μL DMSO master liquid, next addμL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O, mix and clarify.

Method for preparing in vivo formulation: Take μL DMSO master liquid, next add μL Corn oil, mix and clarify.

Note: 1. Please make sure the liquid is clear before adding the next solvent.
2. Be sure to add the solvent(s) in order. You must ensure that the solution obtained, in the previous addition, is a clear solution before proceeding to add the next solvent. Physical methods such
as vortex, ultrasound or hot water bath can be used to aid dissolving.

Mechanism of Action

Targets/IC50/Ki
σ2 receptor [2]
8.02(pKi)
References

Clinical Trial Information

(data from https://clinicaltrials.gov, updated on 2024-05-22)

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05983588 Recruiting
Corticobasal Syndrome (CBS)
Technical University of Munich
 December 12 2023 Phase 2
NCT05019417 Unknown status
Monocarboxylate Transporter 8 Deficiency
Kaplan Medical Center|Weizmann Institute of Science
 June 30 2021 Phase 2|Phase 3
NCT02323100 Terminated
Cystic Fibrosis
National Jewish Health|University of Alabama at Birmingham|Children''s Hospital of Philadelphia|Johns Hopkins University|Horizon Pharma Ireland Ltd. Dublin Ireland
 December 2 2018 Phase 1|Phase 2
NCT02246218 Completed
Urea Cycle Disorder
Horizon Therapeutics LLC|Horizon Pharma Ireland Ltd. Dublin Ireland
 December 31 2014 Phase 4

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