The results of the study show that CG01 has clinically relevant anti-seizure effects.
On February 19th 2018 Professor Merab Kokaia, one of CombiGene’s scientific founders, together with lead scientist Esbjörn Melin presented final data from the preclinical proof-of-concept study for CG01 that was conducted in 2017. The study confirmed that CG01 has clinically relevant anti-seizure effects in the form of fewer and shorter seizures. Following peer review, the study has now been presented in Molecular Therapy: Methods & Clinical Development.
The study has been designed so that the results can be adapted for treatment of epilepsy in humans. With the aid of magnetic resonance imaging (MRI), the part of the hippocampus, i.e., the part of the brain in which the epileptic seizure arises and to which treatment is to be targeted, was localized. Subsequently, a unilateral focal treatment with gene therapy was administered to the epileptic focus. Results of the treatment have been analysed with video EEG (electroencephalography) to observe and measure electrical activity in the brain during a seizure, and histological analysis (tissue analysis) has been used to find detailed answers with respect to deviations in brain tissue and to determine how CG01 is expressed in the brain.
The results of the study show that CG01 has clinically relevant anti-seizure effects. Epileptic seizures were fewer and shorter in duration and some of the animals were completely free of seizures after being treated.
The article is available here:
https://www.cell.com/molecular-therapy-family/methods/fulltext/S2329-0501(19)30102-030102-0 “https://www.cell.com/molecular-therapy-family/methods/fulltext/S2329-0501(19)30102-0”)
