iQure is a clinical-stage biotech company advancing a new therapeutic approach for CNS disorders by restoring glutamate homeostasis.
Our science targets one of the core astrocytic functions - the regulation of glutamate uptake - essential to maintaining synaptic balance and protecting neurons from overstimulation. By enhancing the function of the main transporter involved in this process, we break the cycle of excitotoxicity - a feedback loop that drives neuronal damage and disease progression across a broad range of CNS conditions.
Our lead asset, iQ-007, is a first-in-class, orally available small molecule that enhances the function of the first glutamate transporter. Currently in Phase 1 clinical development for treatment-resistant epilepsy, iQ-007 provides the first clinical validation of our approach. Beyond epilepsy, we are advancing additional preclinical programs targeting neurodegeneration and pain.
iQ-007 has entered clinical development in epilepsy and is currently being evaluated in a Phase 1 trial
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Our
science
Excitotoxicity is a shared mechanism driving neuronal damage across CNS diseases. It stems from glutamate imbalance - when clearance fails, neurons are exposed to toxic levels.
The main glutamate transporter is responsible for over 90% of glutamate uptake. By enhancing its function, we restore glutamate homeostasis. This mechanism unlocks therapeutic opportunities across multiple indications, including epilepsy, Alzheimer’s Disease, Parkinson’s Disease, and neuroinflammatory conditions such as pain, and traumatic brain injury.
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.
The same mechanism is implicated in other CNS conditions, including pain and neurodegeneration.
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Latest news
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2 min
iQure Pharma Appoints Former UCB Chief Medical Officer and Head of Development, Prof. Iris Loew-Friedrich, MD, as Clinical and R&D Advisor
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2 min
iQure Pharma Receives Approval for iQ-007 Phase 1 First in Human Study
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5 min
iQure Pharma’s iQ-007 Receives FDA Orphan Drug Designation for Dravet’s Syndrome
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5 min
iQure Pharma Secures $4M Funding – Advancing Phase 1 Clinical Trial for iQ-007
