Science

Impaired glutamate uptake is a well-established driver of excitotoxicity and neuronal damage. Yet, it remains broadly unaddressed by available therapies for Epilepsy, Alzheimer’s Disease, ALS, Parkinson's Disease, and other CNS conditions.

At iQure, we target this core mechanism through selective astrocyte modulation to enable a disease-modifying approach across CNS indications.

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

Astrocytes are central regulators of glutamate clearance, inflammation, and synaptic homeostasis. Yet, they remain largely untapped as therapeutic targets. Their dysfunction is not secondary: in diseases like epilepsy, ALS, Alzheimer’s Disease, and Parkinson’s Disease, astrocytic impairment directly contributes to pathogenesis.

At the heart of this dysregulation 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, improving transporter trafficking without overexpression.

We are advancing a clinical-stage pipeline of small molecules designed to restore glutamate homeostasis by enhancing EAAT2 function. Our lead compound, iQ-007, is now in Phase 1 development following strong preclinical efficacy across multiple epilepsy models and translational assays, validating our therapeutic approach.

By addressing a shared mechanism of neuronal damage, this strategy underpins a broader portfolio of EAAT2-focused programs across CNS diseases where astrocyte dysfunction and glutamate imbalance remain unaddressed, including neurodegeneration and pain.

Explore our pipeline >>