CombiGene : scientific founders talk about the quantum leap that the epilepsy project CG01 is about to take

The original scientific work on the neurotransmitter NPY and its receptor Y2 was done by CombiGene's scientific founders Professor Merab Kokaia and Associate Professor David Woldbye in 2010. In 2016, the preclinical development of the epilepsy project resulted in the selection of CG01 as the gene therapy candidate. The deal with Spark Therapeutics has the potential of being a quantum leap for the CG01 project, and we contacted Merab and David to get their reactions to the news.

Can you describe your feelings now that your NPY/Y2 discovery which forms the basis of CG01 is moving towards clinical studies with Spark Therapeutics, a large and experienced gene therapy company?

Merab:
"This is like a dream come true. After so many years of research and development, we finally see an opportunity to bring our preclinical studies to clinical application for patients with difficult-to-treat epilepsy. I get e-mails and calls from such patients and it is so frustrating that there is not much one can offer these people, who have exhausted all today's available drugs in their fight against epilepsy. It feels good that now we have hope that a novel treatment strategy with gene therapy will be tested in clinical application, and there could be a better future for these patients. It has been really fulfilling to experience the whole journey of translating our research in the lab into potential clinical treatment that may help many patients. I have been interacting with the team in Spark Therapeutics during our discussions, and I have full confidence that this is a right set of people to bring this treatment all the way from clinical trials to a finished product on the market."

David:
"I am thrilled to see that pioneering scientific work with the neurotransmitter NPY - the key component in CombiGene's gene therapy - may now result in new treatment for epilepsy patients. As a scientist, it is not often that your findings are translated into novel treatment for patients. I am very confident that CombiGene in Spark Therapeutics has found a perfect partner to ensure success of the project of developing the first gene therapy treatment for drugresistant temporal lobe epilepsy patients."

What would it mean for patients if the clinical program is successful?

Merab:
"If the program is successful, it will mean a lot for the patients suffering from drug-resistant epilepsy. These patients have very limited if any alternative treatments, but gene therapy may bring such alternative to them and change their CombiGene's scientific founders talk about the quantum leap that the epilepsy project CG01 is about to take n The original scientific work on the neurotransmitter NPY and its receptor Y2 was done by CombiGene's scientific founders Professor Merab Kokaia and Associate Professor David Woldbye in 2010. In 2016, the preclinical development of the epilepsy project resulted in the selection of CG01 as the gene therapy candidate. The deal with Spark Therapeutics has the potential of being a quantum leap for the CG01 project, and we contacted Merab and David to get their reactions to the news. life completely. Experiencing uncontrolled seizures, as they do, have a really negative impact on the quality of life, and if successful, this novel treatment strategy will change this dramatically."

David:

"If CombiGene's gene therapy, which targets the seizure focus selectively, proves successful in drug-resistant temporal lobe epilepsy patients, this will also open up the possibility that gene therapy can be used for the large group of epilepsy patients who currently do respond to antiseizure drugs but experience life-long side effects. Thus, it is my hope that gene therapy may reduce the use of traditional drugs for treatment of seizures among these patients and also let them experience significant improvement in quality of life."

Any other comments you want to make?

Merab:
"I would like to thank all the people that believed in our research as something valuable for a potential treatment of patients with epilepsy. This road has not been easy, but their dedication and hard work really made this possible. I really hope that the outcome of the clinical trials will be of value for epilepsy treatment, but also be an emotional reward for all of those people who contributed up to this point and all of those who will work on the project in coming years down the road."

David:
"It has been a life-time experience to develop the gene therapy project in collaboration with my long-term scientific partner Prof. Merab Kokaia as well as the dedicated and very competent team of CombiGene."