With Woldbye and Kokaia’s scientific studies as a solid foundation, CombiGene is now developing the concept of AAV vector-induced overexpression of NPY and Y2 into a safe and effective therapeutic method for the treatment of epilepsy.
The target patient population for this method is believed to be those with partial epilepsy in which the affected part of the brain can be clearly identified and who are resistant to drugs and not helped by other treatment options. The prevalence for this group may be as high as 0.3% of the population, corresponding to about 2.5 M people in the US and Europe alone.
The project is currently in the preclinical stage and the final candidate vector is planned to be selected 2016. Following that, tech transfer to a GMP manufacturer will be made and a series of toxicological studies will be conducted, potentially paving the way for a Phase I/II safety study during 2018.
In addition to a GMP manufacturer of AAV vectors, the project also involves a number of well-reputed CROs, consultants and clinical advisors.