The American biotechnology company iQure Pharma has announced the award of a prestigious research grant by The Michael J. Fox Foundation for Parkinson’s Research (MJFF) for the development of a therapeutic program aimed at modulating the function of the EAAT2 transporter in Parkinson’s disease. A key element of the project is the iQ-007 molecule, developed by scientists from the Faculty of Pharmacy at the Jagiellonian University Medical College (UJ CM).
iQ-007 is an innovative EAAT2 modulator that regulates glutamate levels in the central nervous system. Disorders in its uptake play a significant role in the pathogenesis of neurodegenerative diseases, including Parkinson’s disease. The aim of the research is to demonstrate the relationship between the regulation of glutamate levels in the brain and functional effects in Parkinson’s disease models, as well as to identify biomarkers to help select patients who may benefit most from the proposed therapy.
As a reminder, iQ-007 was originally developed by a team led by Prof. Krzysztof Kamiński from the UJ CM Faculty of Pharmacy. It is a first-in-class allosteric EAAT2 activator currently advancing in clinical trials. Its action focuses on astrocytes and restoring the glutamate balance in the central nervous system. The compound is also being developed for other neurological indications, such as drug-resistant epilepsy, ALS, and migraines. In 2020, the molecule was commercialized by the Jagiellonian University through the Centre for Technology Transfer CITTRU. The license was granted to iQure Pharma, an American biotechnology company. The preclinical phase was carried out by the company in cooperation with UJ CM scientists. In January of last year, the U.S. FDA granted the molecule orphan drug designation for the treatment of Dravet syndrome, and three months later, the Human Research Ethics Committee in Australia approved the first phase of clinical trials involving healthy volunteers for the iQ-007 molecule.
Obtaining prestigious funding, such as the MJFF grant, is an excellent example of the successful transfer of basic research results into the development of therapies with real clinical potential. “Patients suffering from Parkinson’s disease need drugs that will be able to significantly slow down or modify the course of the disease. The development of such a therapy would be a real breakthrough in its treatment,” emphasizes Professor Kamiński.
The awarding of the grant confirms the growing importance of EAAT2 modulation as a new therapeutic direction in neurodegenerative diseases. It also highlights the international potential of research conducted at the Jagiellonian University Medical College, along with the key role of iQure Pharma in its further development and translation into clinical applications.
The Michael J. Fox Foundation for Parkinson’s Research is the world’s largest nonprofit organization funding Parkinson’s disease research. Founded in 2000 by actor Michael J. Fox, who lives with the disease, it supports the development of new therapies, biomarkers, and diagnostic methods, while accelerating the implementation of research results into clinical practice. The Foundation has already invested over $2.5 billion in research projects carried out worldwide and is recognized as one of the most important partners funding innovative research on Parkinson’s disease.