CombiGenes drug candidate CG01 is being developed as a new treatment for drug-resistant focal epilepsy. The company is targeting a patient population of approximately 47 new cases each year in the US, EU000, UK, Japan and China alone.
Unlike today's treatment options, which only relieve symptoms and require lifelong treatment, CombiGene aims to cure the disease after only one or a couple of treatment sessions with CG01.
Looking for a new partner
In early 2024, CombiGene reacquired the rights to the CG01 project from American Spark TherapeuticsThe biotechnology company is now in the analysis phase of the data generated during the past two years of collaboration, which will be compiled into a package that can be presented to potential partners who have a strategic interest in gene therapy in epilepsy.
The Danish investment company Orphazyme sees the potential in the project and recently acquired 10 percent of the shares in CombiGene. Read more in BioStock's interview with Orphazyme's chairman of the board Michael Hove here.
CEO comments on the approval of patents in new countries

Patents are important for creating an attractive project for potential partners. To date, CombiGene has had two approved patents for CG01 – in the USA and Russia. Last week, patents were granted for CG01 in two countries, Australia and India. How important is it for CombiGene to secure patents in these countries, and does the gene therapy field differ from traditional pharmaceutical projects in terms of patents?
BioStock contacted the CEO Peter Ekolind to get his views on intellectual property rights and the two patents specifically.
Peter, how do gene therapies differ from other pharmaceuticals when it comes to protecting their innovations?
— In fact, not that much at all. In order to dare to invest in a long and costly development phase, as is the case for gene therapy development, it is important to be sure that the innovation can be out-licensed to a potential recipient, and then strong patent protection is the means available.
What do the patents now granted cover and how long does the patent protection last?
— These two patents belong to patent family 2 and cover the gene therapy candidate CG01. The protection extends to different lengths for different countries, for Australia and India the protection lasts until the beginning of 2037.
You work within the framework of the Patent Cooperation Treaty (PCT) which covers around 150 countries with a single application. Are the new patents obtained through the PCT?
— We first filed a PCT application for these patents and then we have selected about ten countries to enter into national applications for. With this announcement, Australia and India are now approved national applications
Finally, what is happening right now in CombiGene?
— The focus of our work is on analyzing the data that we are still receiving from SPARK, which will form the basis for a new out-licensing to interested companies. Work on the COSY program is progressing at a rapid pace.