CombiGene has recently successfully completed a so-called cell tropism study with the company’s gene therapy CG01, which is being developed for the treatment of drug resistant focal epilepsy. CG01 is intended to reach the nerve cells in the hippocampus, that is, the cells that triggers the epileptic seizures. The now finished tropism study shows that CG01 works just like intended: it is taken up by the nerve cells of the hippocampus, but not in the supporting cells, the so-called glial cells.
The results from the study are very encouraging as they further confirm that CG01 reaches the brain cells as intended. The knowledge from the tropism study provides additional understanding of how CG01 works and provides answers to questions about the drug candidate’s tropism as asked for by the regulatory authorities.