Refractory epilepsy is a common, severe neurological disorder for which there are limited treatment options. Fewer than 10% of people with refractory epilepsy are suitable for curative resective brain surgery, predominantly because of proximity of the epileptic focus to eloquent cortex (regions involved with critical functions).
We have harnessed decades of clinical genetic and neurophysiological research to create a portfolio of gene therapy approaches designed to ‘calm down’ the cortical networks that drive seizures. These approaches offer the potential to restore epileptic tissue to normal physiological activity, targeting only the critical area of the brain involved in the triggering of epilepsy. To increase the translational potential of our therapies, we have established appropriate chronic models of epilepsy, EEG analysis, and blinded, randomised pre-clinical trials of efficacy.
We are using a range of revolutionary approaches that include the utilisation of new engineered genes or the manipulation and modification of genes already present in the patients` genome. As a group we aim to conduct our first in-human clinical trial for epilepsy gene therapy in the next 3 years.