Drug intelligence / Profile preview

L-2,4-diaminobutyric acid

Development stage
Phase 2
Modality
Small Molecules
01

Overview

L-2,4-diaminobutyric acid is a non-proteinogenic amino acid belonging to the class of alpha-amino acids. It is an unnatural cationic amino acid analogue with two amino groups at positions 2 and 4 on the butyric backbone. This compound has been studied primarily as a research tool and chemical precursor in pharmaceutical development—especially for drugs targeting neurological disorders—and in biochemical studies of amino acid metabolism. Mechanistically, it acts as a noncompetitive inhibitor of GABA transaminase (GABA-T), thereby preventing the breakdown of gamma-Aminobutyric Acid (GABA) and increasing its levels in neural tissue. It also inhibits GABA reuptake[5]. These actions can elevate GABA concentrations in the brain. While it has shown anticonvulsant properties experimentally due to these effects on GABAergic neurotransmission, it is also considered neurotoxic and may cause liver damage at higher exposures[5]. The compound is not approved for clinical use but finds application in research settings including gene therapy delivery systems and diagnostic assay development[1][5].

Other names
(S)-2,4-diaminobutanoic acid(2S)-2,4-diaminobutanoic acidalpha,gamma-DiaminobutyrateL-2,4-diaminobutanoateL-diaminobutyric acidButanoic acid, 2,4-diamino-, (2S)-2,4-Diaminobutyric acid
02

Targets

ABAT (4-aminobutyrate aminotransferase)GAT (GABA transporter family)

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