Astrocyte-targeted therapeutics to restore glutamate balance in CNS disorders

iQure Pharma is a clinical-stage biotech company developing first-in-class astrocyte-targeted therapeutics designed to restore glutamate balance in CNS disorders.

The company’s science is built around one of the core functions of astrocytes: the regulation of glutamate uptake through EAAT2, the main transporter involved in maintaining synaptic balance and protecting neurons from overstimulation. By enhancing EAAT2 function, iQure aims to break the cycle of excitotoxicity, a feedback loop linked to neuronal overstimulation and dysfunction across CNS conditions including epilepsy, Parkinson’s disease, migraine and ALS.

Our lead asset, iQ-007, is a first-in-class, orally available EAAT2 positive allosteric modulator currently in early clinical development. Epilepsy is the first clinical validation path, supported by strong translational models and clear clinical endpoints. In parallel, iQure is building a Parkinson’s disease expansion strategy, with migraine and ALS as additional mechanism-driven opportunities supported by EAAT2 biology and preclinical work.

iQ-007 is advancing in early clinical development — iQ-007 is advancing in early clinical development — iQ-007 is advancing in early clinical development — iQ-007 is advancing in early clinical development — iQ-007 is advancing in early clinical development — iQ-007 is advancing in early clinical development — iQ-007 is advancing in early clinical development — iQ-007 is advancing in early clinical development — iQ-007 is advancing in early clinical development — iQ-007 is advancing in early clinical development —

iQ-007 is advancing in early clinical development

OUR TEAM Site visit at Prof. Kaminski's lab in Krakow with members of the iQure team and early investors. Read more about our team

Our
science

Astrocytes regulate glutamate clearance through EAAT2, the main transporter responsible for maintaining synaptic balance and protecting neurons from overstimulation. When this function is impaired, glutamate can accumulate and fuel excitotoxicity, a self-amplifying process linked to neuronal stress and dysfunction.

iQure is developing first-in-class EAAT2 positive allosteric modulators designed to restore physiological glutamate clearance through astrocyte-driven uptake. This mechanism supports a focused CNS strategy, with epilepsy as the first clinical validation path, Parkinson’s disease as the next strategic pillar, and migraine and ALS as additional mechanism-driven opportunities.

Find out more

iQure’s approach is demonstrated in peer-reviewed research published in Annals of Neurology

Our approach is supported by co-authored research validating our mechanism as a novel antiseizure strategy, with preclinical proof of concept.

The work was conducted in collaboration with Harvard Medical School, NIH, Boston Children’s Hospital, and Jagiellonian University.

Read the publication

iQ-007
breaking the seizure cycle

Explore our pipeline

Epilepsy is driven by a reinforcing loop: glutamate accumulation, excitotoxicity, and progressive neuronal damage. EAAT2, the main astrocytic glutamate transporter, plays a central role in controlling this cycle by clearing excess glutamate from the synaptic space. iQ-007 is the first clinical-stage compound designed to enhance its activity. By restoring glutamate uptake capacity, iQ-007 is designed to intervene upstream in the pathological feedback mechanism that drives seizure activity.

iQ-007 is in early clinical development and is being prepared for Phase 2 proof-of-concept in epilepsy. The same astrocyte / EAAT2 axis also supports broader expansion across CNS conditions where glutamate imbalance is implicated. More about clinical development.

More about clinical development