CombiGene is an integrated part of a dynamic gene-therapeutic landscape

Jan 20, 2020

This is an article from Ingenious number four 2019.

Read the whole Ingenious here >>


Gene therapy is now one of the hottest areas in the pharma industry. Naturally, this is due mainly to the great expectations that medicine is placing on gene therapy. Suddenly, there is hope that patients who suffer from serious illnesses may receive effective treatments that will decisively improve their quality of life and the possibilities for living without limitations.

370 candidate drugs in the clinical phase
Today, 370 clinical studies in gene therapy are currently running (ARM Q3, 2019), of which 32 are in clinical phase III. Many of the candidate drugs under development are targeted at serious and rare diseases for which effective forms of treatment are currently lacking. For many people, the quality of life could thereby be considerably improved. For certain diseases, gene therapy can potentially mean the difference between life and death.

On August 31st 2017, the US Food and Drug Administration (FDA) approved the first gene-therapeutic drug, for clinical treatment of acute lymphoblastic leukaemia. Since then, additional gene therapies have achieved market approval and the number of approved gene-therapy treatments is expected to increase dramatically in the coming years. In other words, there is good reason to assume that gene therapy will play a major role in future health care.

Significant commercial potential
The advances in medicine also imply potentially great commercial successes for the companies that succeed in developing effective gene therapies. Belief in the commercial viability of gene therapy is reflected in, among other things, the considerable investments that are being made in this area, which during the first nine months of 2019 amounted to some USD 5.6 billion (ARM Q3, 2019).
As the leading Nordic gene-therapy company, CombiGene is an integrated part of a dynamic gene-therapeutic landscape. The company has two exciting projects: CG01, which is being developed for treatment of drug-resistant focal epilepsy and the project for treating the rare disorder lipodystrophy that was recently inlicensed from Lipigon.

CG01 is being developed to treat a large group of patients
The CG01 project differs from many other gene therapy projects in that the number of potential patients is very large. In the USA alone some 14,000 patients are diagnosed with drug-resistant focal epilepsy each year, patients who could be candidates for surgery. CombiGene estimates that, realistically, 10–20 percent of these patients could be treated with the company’s candidate drug, CG01. Living with untreated epilepsy entails great limitations in day-to-day life and constant worry about having to face sudden, unpredictable epileptic seizures. The large number of potential patients also means that the commercial potential of the project is very large. CombiGene has begun to carry out the concluding stages of the preclinical programme, after which the first studies in humans will commence.

With the lipodystrophy project, CombiGene is expanding into another area of therapy
The lipodystrophy project is in an early preclinical phase. As opposed to CG01, which targets a large patient population, this project refers to a small group of patients who have the rare disorder partial lipodystrophy, for which there are currently no effective treatments. Since lipodystrophy is a rare disease, there is a good chance that a future candidate drug may receive so-called orphan drug designation. Orphan drugs offer a means by which regulatory authorities, including the American FDA and the European EMA, can encourage the pharmaceutical industry to develop drugs for rare diseases. Obtaining orphan drug designation brings many great advantages. Essentially, this means that development can proceed faster and at a lower cost. An orphan drug designation also means that the chances of reaching the market are greater and the price of the approved drug is usually higher as compared to drugs for commonly occurring diseases.

“During the past year CombiGene has taken significant steps forward,” says CombiGene’s CEO, Jan Nilsson. “CG01 is a project that is targeted at a very large group of patients, which is relatively unusual for a gene therapy project. It is important to note that in the CG01 project we are not only developing a GMP-classed production method, which is a challenge for all gene therapy projects, we have also signed a Master Service Agreement with Cobra Biologics for production of CG01 for both clinical studies and commercial production of a future, approved CG01 drug. Furthermore, our lipodystrophy project has the potential to be an orphan drug, which makes our situation even more favourable. Both of our projects are also within very interesting areas. With the CG01 project we acquire great knowledge of the central nervous system and, with the lipodystrophy project, we will build up considerable knowledge surrounding gene therapy and the metabolic system. All in all, this confirms my confidence in a very positive future for CombiGene as the leading Nordic gene-therapy company.”

CombiGene’s lead project CG01 has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 823282”

About CombiGene

CombiGene’s vision is to provide patients affected by severe life-altering diseases with the prospect of a better life through novel gene therapies. 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 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 company is public and listed on the Swedish marketplace Nasdaq First North Growth Market and the company’s Certified Advisor is FNCA Sweden AB, +46 (0)852 80 03 99


Combigene AB, Medicon Village, SE-223-81 Lund, Visiting adress: Scheelevägen 2, Lund, Sweden

Linked In     Twitter      Facebook