Science

Impaired glutamate uptake is a well-established driver of excitotoxicity and  neuronal overstimulation and dysfunction. Yet, it remains broadly unaddressed by available therapies across CNS disorders.

At iQure, we target this core mechanism through selective enhancement of EAAT2, the main astrocytic glutamate transporter, to restore the brain’s natural capacity to clear excess glutamate.

Advancing a High-Impact and Underexploited Axis in CNS Drug Development

Astrocytes as a therapeutic control point in CNS diseaseAstrocytes are central regulators of glutamate clearance, synaptic homeostasis, metabolic support and inflammatory signaling. Yet, they remain largely untapped as therapeutic targets. 

At the heart of this biology lies EAAT2, the main glutamate transporter in the brain, responsible for over 90% of extracellular glutamate uptake. EAAT2 dysfunction is a driver of seizures, neurodegeneration, and chronic inflammation, making it a validated yet still unaddressed target across multiple CNS indications.

Unlike indirect or non-selective approaches, iQure's strategy focuses on functional enhancement of EAAT2 through selective allosteric modulation, restoring physiological glutamate uptake without blocking glutamate receptors or directly suppressing neuronal activity.

We are advancing a clinical-stage pipeline of small molecules designed to restore glutamate balance by enhancing EAAT2 function. Our lead compound, iQ-007, is a first-in-class, orally available EAAT2 positive allosteric modulator in early clinical development.

In epilepsy, iQ-007 is designed to enhance astrocyte-mediated glutamate clearance and intervene upstream in the excitotoxic feedback loop that contributes to seizure activity. Beyond epilepsy, the same astrocyte / EAAT2 axis supports a focused expansion strategy in Parkinson’s disease, migraine and ALS.

Explore our pipeline >>