GAO-3-02 / GAOMA Therap - LARVOL DELTA

Synaptic and cellular mechanisms involved in the anti epileptic effect of GAO-3-02 (Neuroscience 2024) - "These data provide new insights into the mechanism of GAO-3-02 in the treatment of neuroinflammation-related diseases. Therefore, GAO-3-02 may be a potential therapeutic option for FIRES."

Late-breaking abstract • CNS Disorders • Epilepsy

Progress report on new medications for seizures and epilepsy: A summary of the 17th Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVII). I. Drugs in preclinical and early clinical development. (PubMed, Epilepsia) - "These include AMT-260, a gene therapy designed to downregulate the expression of Glu2K subunits of kainate receptors, in development for the treatment of drug-resistant seizures associated with mesial temporal sclerosis; BHV-7000, a selective activator of heteromeric Kv7.2/7.3 potassium channels, in development for the treatment of focal epilepsy; ETX101, a recombinant adeno-associated virus serotype 9 designed to increase NaV1.1 channel density in inhibitory γ-aminobutyric acidergic (GABAergic) neurons, in development for the treatment of SCN1A-positive Dravet syndrome; GAO-3-02, a compound structurally related to synaptamide, which exerts antiseizure activity at least in part through an action on cannabinoid type 2 receptors; LRP-661, a structural analogue of cannabidiol, in development for the treatment of seizures associated with Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex; OV329, a selective inactivator of GABA aminotransferase, in..."

Journal • Preclinical • CNS Disorders • Developmental Disorders • Epilepsy • Gene Therapies • Immunology • NAV1

Synaptamide Phosphonate-GAO-3-02-potentiates GABAergic transmission in the rat lithium-pilocarpine model of temporal lobe epilepsy via activation of CB2 receptor (Neuroscience 2023) - "However, bath perfusion of GW842166X, a potent and selective CB2 receptor agonist, which is undergoing clinical development, failed to significantly increase the current amplitude of eIPSCs. The present study suggests that GAO-3-02 enhances GABAergic transmission onto CA1 pyramidal neurons through activating CB2 receptor. Our results may provide a cellular and molecular mechanism that helps explain the anti-seizure effects of GAO-3-02 in the rat lithium-pilocarpine model of temporal lobe epilepsy."

Preclinical • CNS Disorders • Epilepsy

Gao-3-02: a promising lipidic drug-candidate in epilepsy, reducing seizures and improving cognition, with anti-inflammatory activity (Neuroscience 2022) - "In the Pilo rat model, GAO-3-02 significantly ameliorated spatial learning deficits and completely rescued hippocampal Long-Term Potentiation (LTP), contrary to other ASMs tested (sodium valproate, levetiracetam and cannabidiol). Overall, GAO-3-02 displayed robust anti-seizure effects across validated seizure and epilepsy models and ameliorated spatial learning and synaptic plasticity, possibly via inhibition of inflammatory cytokine secretion. GAO-3-02 may therefore be a novel, promising therapeutic candidate for epilepsy and associated cognitive impairments."

Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Epilepsy

Progress report on new antiepileptic drugs: A summary of the Sixteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVI): I. Drugs in preclinical and early clinical development. (PubMed, Epilepsia) - "This progress report summarizes preclinical and, when available, phase 1 clinical data on five different investigational compounds in preclinical or early clinical development, namely GAO-3-02, GRT-X, NBI-921352 (formerly XEN901), OV329, and XEN496 (a pediatric granular formulation of retigabine/ezogabine). Overall, the data presented in this report illustrate novel strategies for developing antiseizure medications, including an interest in novel molecular targets, and a trend to pursue potential new treatments for rare and previously neglected severe epilepsy syndromes."

Journal • Preclinical • CNS Disorders • Epilepsy • Pediatrics