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Drug intelligence / Profile preview
methyl gamma-butyrobetaine
- Development stage
- Unknown
- Lead developer
- Grindeks
- Modality
- Small Molecules
- Administration
- Oral, Parenteral
01
Overview
Methyl gamma-butyrobetaine (methyl-GBB) is a synthetic small molecule inhibitor of gamma-butyrobetaine dioxygenase (BBOX1) and the organic cation/carnitine transporter 2 (OCTN2/SLC22A5). Developed by the Latvian Institute of Organic Synthesis and Grindeks, methyl-GBB acts to deplete L-carnitine and long-chain acylcarnitine levels in tissues. This depletion shifts cellular energy metabolism away from fatty acid beta-oxidation and toward glucose oxidation, which is more oxygen-efficient under ischemic conditions. Preclinical and clinical studies have demonstrated its potential to protect mitochondrial function, reduce myocardial infarct size, attenuate atherosclerosis, and improve insulin sensitivity and glucose tolerance. It has completed Phase 1 clinical development for the treatment of cardiovascular diseases.
- Other names
- 4-(ethyl(dimethyl)ammonio)butanoate4-[ethyl(dimethyl)ammonio]butanoatemethyl-GBBmethyl-gamma-butyrobetaine
02
Targets
BBOX1 (Gamma-butyrobetaine dioxygenase)SLC22A5 (Organic cation/carnitine transporter 2)
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