Development assets are sourced from an external research network and developed to achieve clinical proof of concept. Drug candidates for common diseases will be co-developed and commercialized through strategic partnerships, while the company may manage this process on its own for drugs targeting niched patient populations.
The epilepsy project is currently being prepared for safety studies together with CombiGene’s partner Spark Therapeutics
CombiGene conducts no research under its own management, but sources development assets via collaboration with an external research network. The advantages with this type of collaboration are apparent for both parties. CombiGene is able to gain access to leading research from academia as well as industry without having to incur costs for resource-intensive research. In turn, the external researchers gain access to CombiGene’s considerable expertise in pharmaceuticals development and project management, and financial resources to enable preclinical and clinical development.
CombiGene has a team of very knowledgeable and experienced professionals, as well as solid, longstanding experience from the international pharma industry and the biotech arena, together with a thorough knowledge of different aspects of gene therapy. This combination of experience and expertise allows CombiGene, together with a network of selected external partners who complement CombiGene’s internal expertise, to conduct ground-breaking gene-therapeutic development very effectively.
To use the company’s resources as effectively as possible CombiGene seeks to develop manufacturing methods and conduct preclinical/clinical studies in collaboration with leading external partners in the respective areas. In this way CombiGene is able to choose the most suitable partner and thereby drive the projects as effectively and successfully as possible.
Under its own management, CombiGene’s ambition is to take its candidate drugs through preclinical development up to initial studies in humans. Candidate drugs for commonly occurring illnesses will be commercialized through strategic partnerships and CombiGene is making extensive partnering efforts in order to build long-term relations with interesting international pharmaceutical companies. For drugs targeting niched patient populations CombiGene may establish its own sales channels.
CombiGene originated in the 1990s, when the company’s scientific founders, Professor Merab Kokaia and Associate Professor David Woldbye, demonstrated that neuropeptide Y (NPY) inhibits epileptic seizures in animals and that this positive effect is further strengthened by the NPY receptor Y2.
The great challenge at that time was that there was no adequate means of applying this knowledge to create an effective treatment, since there was no way of administering the intended treatment to humans.
The discovery that a safe, non-pathogenic virus whose own DNA was largely removed and replaced with functional DNA sequences (so-called AAV vectors) can be used to “transport” gene-therapeutic drugs immediately presented entirely new possibilities for Professor Kokaia and Associate Professor Woldbye.
On 25 May 2015 CombiGene was listed as a public company on SPOTLIGHT Stock Market (then AktieTorget). The listing generated SEK 12.5 million in research capital which in 2016 enabled CombiGene to test and identify the final candidate drug, named CG01.
During 2017 and 2018 CombiGene carried out several preclinical studies with CG01. All studies confirmed the anti-epileptic effects of CG01 and during 2018 CombiGene began extensive preparations for clinical studies, i.e., human trials.
During the development of CG01 CombiGene has amassed a knowledge of gene therapy and a network that is unique among Nordic companies. This knowledge covers aspects that are decisive for the successful development of gene therapeutic drugs; for example, selection of viral vectors, methodology development for GMP-classed production of gene-therapeutic substances (which is considerably more complicated than production of conventional drugs) and the regulatory issues surrounding gene therapy. Concurrently, the company has established a broad international contact network within the global pharmaceutical industry.
CombiGene is currently pursuing two gene therapy projects: CG01, for treatment of focal epilepsy, and a project for treatment of lipodystrophy, a condition characterized by an abnormal distribution of fatty tissue in the body. CombiGene’s long-term ambition is to identify and develop additional projects in the gene therapy area.
Gene therapy technology is unique in that it makes it possible to replace defective or missing genes or, in the case of CombiGene’s method in the CG01 project, to increase the expression of existing genes.
Currently, some 2,800 such diseases that are caused by single defective genes are known and, for the majority of them, there are no adequate treatment methods. Gene therapy is one of the most exciting areas of development in the pharma industry, thanks to the unique possibilities for precisely targeted therapy and the potential for meeting very great medical needs.
Unlike conventional drug treatment of chronic diseases, which requires continuous medication, gene therapy has the great advantage of being able to achieve a long-term effect after just one or a few treatments. Significant advances in gene therapy have been made in the past 5-10 years and there is considerable interest in this technology among the major pharmaceutical companies, as evidenced by, among other things, the large number of acquisitions during recent years. Considering the many advantages of gene therapy, and the many research and development projects now under way throughout the world, it is reasonable to assume that the number of approved gene therapy products will continue to grow dramatically over the coming years. A large number of patients whose diseases cannot be adequately treated can thereby enjoy significantly better lives, thanks to the new therapies.
CombiGene’s candidate drug increases expression of NPY and its receptor Y2, suppresses glutamate release (the cause of epileptic seizures) and thereby reduces the number of epileptic seizures.
Through the collaboration and in-licensing agreement with Spark Therapeutics in 2021, CombiGene reached a new stage in the company’s development. Through this agreement, CombiGene became an internationally recognized gene therapy company. Our ambition now is to build on this success by the in-licensing of a third gene therapy project, and our efforts to accomplish this have intensified.
We are primarily seeking AAV-based projects because it is within this technology platform that the company has established knowledge in key areas such as vector design (design of drug candidate), safety aspects, and production. Similarly, the areas of disease that are in focus are those where CombiGene has a solid knowledge and collaboration network, i.e., diseases of the central nervous system and metabolic diseases. Having said that, we will at the same time have an open attitude towards all potential projects and evaluate each opportunity on its own merits.
Finding the right gene therapy project to in-license and negotiating the right terms are however no easy tasks, and it goes without saying that it is not possible to predict exactly when we will have a third project to complement our very promising portfolio.