The initial discovery
CombiGene is at the start of an exciting journey of research. The company’s scientific founders, Merab Kokaia, Professor of Neurophysiology and head of the Epilepsy Center at Lund University’s Faculty of Medicine, and David Woldbye, Associate Professor at the University of Copenhagen, were among the first in the world to demonstrate that a neuropepticle in the brain, NPY (Neuropeptide Y), suppresses epileptic seizures in animals and that the effect could be further strengthened with the addition of the Y2 NPY receptor. Since that discovery, CombiGene has successively built up a unique body of knowledge concerning gene-therapy mechanisms in relation to various neurological illnesses, with an initial focus on epilepsy.
Challenge and breakthrough
At the start of the millenium, the great challenge was how to apply knowledge of NPY’s antiepileptic properties to create a drug. Quite simply, an effective means of administering the treatment to humans was lacking. A breakthrough in gene therapy research that had major significance for CombiGene was the discovery that a harmless, non-pathogenic virus whose DNA had been largely removed and replaced with functional DNA sequences (so-called AAV vectors) can be used to “transport” gene-therapy medicines. Various AAV vectors are now being used in more than 100 clinical studies throughout the world.
With the original discoveries surrounding neuropeptide Y/receptor Y2 and the new AAV technology as a basis, the tempo of development picked up. On 25th May 2015 CombiGene was listed on AktieTorget.
The offering provided the company with 12.5 million kronor for research. In 2016 a strong financial position enabled CombiGene to test a final candidate drug and a dose-response study was completed in early 2017. The study confirmed, as expected, that CombiGene’s candidate drug CG01 has the intended dose-dependent antiepileptic effect. The positive outcome meant that, during the second quarter of 2017, CombiGene was able to initiate the preclinical long-term study which is the next important milestone on the road to an approved drug. Preliminary results from the study is expected to be ready during the last quarter of 2017.
Activities are broadened
In the summer of 2017 CombiGene was awarded a “Seal of Excellence” by Horizon 2020, the EU’s framework programme for research and innovation. Soon after, CombiGene received 500,000 kronor from Vinnova for drawing up a detailed business plan for the company’s epilepsy project. The business plan will also include a broader programme of research.