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OV329 is a next-generation, highly potent small molecule inhibitor of GABA aminotransferase (GABA-AT), the enzyme responsible for degrading γ-aminobutyric acid (GABA), the main inhibitory neurotransmitter in the brain. By inhibiting GABA-AT, OV329 increases endogenous GABA levels in the central nervous system, enhancing inhibitory signaling and reducing neuronal hyperexcitability associated with seizures. It is being developed primarily for rare and treatment-resistant forms of epilepsy and seizures, including drug-resistant epilepsies. Preclinical studies indicate that OV329 provides robust seizure reduction at low doses with a preferable safety profile compared to first-generation GABA-AT inhibitors like vigabatrin—specifically showing less risk of ocular toxicity due to rapid tissue clearance and lack of retinal accumulation. Additionally, preclinical data suggest potential efficacy in neuropathic and inflammatory pain without apparent abuse liability or tolerance development[1][2][3][4][5][6][8].
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