CombiGene’s proprietary platform comprises the use of gene therapy vectors delivering a combination of transgenes encoding two or more of NPY, NPY receptors, galanin, galanin receptors, somatostatin, and/or somatostatin receptors, for the treatment of neurological or psychiatric diseases in humans or animals. Patent applications have been approved in the US (US 8,901.094 B2) and in Europe/EPO (EP 2 046 394 B1), claiming priority from July 4th 2006.
The preferred vector type is the AAV vector, and CombiGene uses well-reputed contract providers of non-GMP AAV vector supply as well as large-scale GMP.
Current focus is on the development of an epilepsy treatment by means of an AAV vector carrying the transgenes for the neurotransmitter NPY and one of its receptors, the G protein-coupled receptor Y2. CombiGene’s scientific founders have shown in a series of in vitro and in vivo studies that AAV vector-mediated upregulation of these proteins can inhibit epileptic seizures.