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iQ-007 is a first-in-class small molecule positive allosteric modulator of the Excitatory Amino Acid Transporter 2 (EAAT2), also known as solute carrier family 1 member 2. EAAT2 is responsible for up to 90% of glutamate uptake in the brain. By enhancing EAAT2 function in astrocytes, iQ-007 increases the reuptake of excess glutamate from the synaptic cleft. This mechanism aims to prevent excitotoxicity and neurodegeneration associated with elevated glutamate levels—a key driver in conditions such as drug-resistant epilepsy and other neurodegenerative diseases including Alzheimer’s and Parkinson’s disease. Preclinical studies have shown efficacy and safety; pivotal toxicology studies were completed in late 2024. The drug has received orphan drug designation from the FDA for Dravet syndrome and entered Phase 1 clinical trials in healthy volunteers as of April 2025[3][5][6][7].
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