CombiGene’s research is recognized internationally after careful scientific review

Associate Professor David Woldbye, one of CombiGene’s scientific founders, presents in a new peer-reviewed article in Frontiers in Molecular Neuroscience the outcome of CombiGene’s preclinical pharmacokinetic study, the company’s preclinical learning and memory study and the preclinical tropism study. All studies have been conducted within the framework of the epilepsy project CG01.

The pharmacokinetic study indicates that one can expect NPY and Y2, the active substances encoded by CG01, to have a long-term expression in the human brain. The results of the pharmacokinetic study provide important knowledge to plan the future safety studies (toxicology and biodistribution) in an optimal way. The knowledge from the pharmacokinetic study will also be a central part of the knowledge pool when the first study in humans is designed.

The results of the learning and memory study show that NPY and Y2 do not have a significant negative impact on cognitive functions. Associate Professor Woldbye has previously commented on the study results as follows: “The results of previous academic studies on the impact of NPY on memory and learning are ambiguous. It is therefore good that we can now, with CG01, show that we do not affect memory and learning in animals when we inject CG01 into one of the brain’s two hippocampi, just as we intend to do in humans.”

The conducted tropism study shows that CG01 occurs as expected, i.e., the drug candidate is absorbed into the nerve cells of the hippocampus, but not in the supporting cells, the so-called glial cells. The results of the study are very encouraging as they provide further confirmation that CG01 reaches the brain cells as intended.

The results, presented in Frontiers in Molecular Neuroscience, have been reviewed by researchers and experts in the field and data from the studies have been rigorously assessed, ensuring scientific quality.

Facts about the article
Journal: Frontiers in Molecular Neuroscience

Title: Gene therapy vector encoding neuropeptide Y and its receptor Y2 for future treatment of epilepsy: preclinical data in rats


Authors: Julia Alicja Szczygiel, Kira Iben Danielsen, Esbjörn Melin, Søren Hofman Rosenkranz, Stanislava Pankratova, Annika Ericsson, Karin Agerman, Merab Kokaia, David Paul Drucker Woldbye