CombiGene’s vision is to provide patients affected by severe life-altering diseases with the prospect of a better life through novel gene therapies

Business Concept and Vision

CombiGene’s business concept is to develop effective gene therapies for severe life-altering diseases where adequate treatment is currently lacking.

Development assets are sourced from an external research network and developed to achieve preclinical/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 pain program – COZY

The pain program COZY is being developed jointly with the Danish company Zyneyro. The goal is to develop an effective treatment for severe chronic nerve pain. The program consists of two projects – a peptide treatment and a gene therapy treatment (AAV).

The lipodystrophy project – CGT2

The CGT2 project aims to develop a gene therapy treatment for partial lipodystrophy, a rare disease for which effective treatments are currently lacking

The epilepsy project – CG01

The focus is on developing a treatment method for epilepsy patients who cannot be helped by currently available therapies​

In order to prepare CG01 to meet the needs of a global submission the remaining preclinical program will be expanded and, in some parts, complemented with additional studies in collaboration with Spark


Development of research assets in collaboration with external research entities

CombiGene is a virtual company and conducts research and development by utilizing Contract Research Organizations (CRO´s) or in academic collaborations. All managed by CombiGene´s skilled and qualified staff. 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.

Expertise and experience

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. 

Methodology development and preclinical studies in collaboration with leading international players

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. 

Commercialization in collaboration with leading pharmaceutical companies

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's history in brief

The 1990s

The first steps were taken during the 1990s

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 early 2000s

A decisive discovery

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.

2015 to 2018

CombiGene as a listed company

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.

The present and the future

CombiGene is currently conducting three gene therapy development projects and one peptide project: the pain program COZY consists of one peptide treatment and one gene therapy, which both are based on the same unique mechanism of action; the epilepsy project CG01 is developed for the treatment of drug-resistant focal epilepsy; and the CGT2 project for the treatment of lipodystrophy, a rare condition characterized by an abnormal distribution of fatty tissue in the body. In October 2021, the epilepsy project CG01 was outlicensed to Spark Therapeutics.

The gene therapy explorer

Since the company was founded, 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.

About gene therapy

Gene therapy technology is unique in that it makes it possible to treat diseases caused by single defective genes, alternatively, to give the body's cells an instruction on how to produce a specific substance.

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, one can 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.

We continue to look for new projects

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 additional gene therapy projects, and our efforts to accomplish this have intensified.

In January 2023, we entered a collaboration with the Danish company Zyneyro to jointly develop their pain program. However, our efforts to expand our portfolio does not end there. We continue to look for new and interesting project, primarily AAV-based since it is within this technology platform that we have established knowledge in key areas such as vector design (design of drug candidate), safety aspects, and production.