Glycerol phenylbutyrate

Synonyms: GT4P, HPN-100M, Ravicti

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. Glycerol phenylbutyrate is a prodrug in which phenylbutyrate (PBA) is released from the glycerol backbone by lipases in the gastrointestinal tract.

Glycerol phenylbutyrate Chemical Structure

Glycerol phenylbutyrate Chemical Structure

CAS No. 611168-24-2

Purity & Quality Control

Glycerol phenylbutyrate Related Products

Biological Activity

Description 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. Glycerol phenylbutyrate is a prodrug in which phenylbutyrate (PBA) is released from the glycerol backbone by lipases in the gastrointestinal tract.
Targets
σ2 receptor [2]
8.02(pKi)
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

Chemical Information & Solubility

Molecular Weight 530.65 Formula

C33H38O6

CAS No. 611168-24-2 SDF --
Density 1.13 g/mL
Smiles O=C(CCCC1=CC=CC=C1)OCC(COC(=O)CCCC2=CC=CC=C2)OC(=O)CCCC3=CC=CC=C3
Storage (From the date of receipt) 2 years -20°C liquid

In vitro
Batch:

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

Water : Insoluble

Ethanol : Insoluble


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In vivo
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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. )

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

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Answers to questions you may have can be found in the inhibitor handling instructions. Topics include how to prepare stock solutions, how to store inhibitors, and issues that need special attention for cell-based assays and animal experiments.

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