The market for gene therapy

Commercially, gene therapy is still at the starting line. There are some approved gene therapy products and a range of new drugs are expected to be approved by the relevant authorities in the coming years. As research and development continue, so do intensive efforts to find solutions concerning remuneration systems.

A large number of clinical studies and significant investment

Confidence in gene therapy is at a very high level, which is illustrated by the large number of clinical studies that are ongoing worldwide. There are currently over 500 clinical trials in the gene therapy field (Alliance for Regenerative Medicine, Q2 2023 Data). The focus of the studies is in the field of oncology, but cardiovascular diseases and diseases related to the central nervous system are also common.

Major financial investments are being made in the gene therapy field with several billion USD invested annually in research and development, and the long-term trends in the sector indicate growing support and interest.

Historic breakthrough in the USA

On August 30, 2017, the US Food and Drug Administration (FDA) approved the first gene therapy for the US market. The approved therapy, Kymriah, from the pharmaceutical company Novartis, has been developed for treatment of pediatric patients and young people with acute lymphoblastic leukemia. Since then, a number of gene therapies have been approved in both the US and Europe, including Luxturna from Spark Therapeutics.

The market for CombiGene’s pain program

It is too early to define any exact market potential for CombiGene’s pain program. It is, however, apparent that there is a great medical need for new and effective treatments. About 20 percent of the world’s adult population suffers from some form of chronic pain. In the United States, between four and eight percent of the population is estimated to be affected by high impact chronic pain. Conventional treatment of severe pain consists primarily of anti-inflammatory drugs, antidepressants, antispasmodic drugs, and opioids (a group of substances with a morphine-like mechanism of action). [1]

The problem with these treatments is that they are not specifically developed to treat chronic pain. The pain relief that is achieved therefore often has a number of disabling side effects such as addiction problems, depression, anxiety, fatigue, impaired physical and mental ability. In the United States, an estimated 700,000 people have died due to opioid abuse in the past 20 years.

[1] Prevalence of Chronic Pain and High-Impact Chronic Pain Among Adults — United States, 2016; CDC; Morbidity and Mortality Weekly Report Weekly / Vol. 67 / No. 36 September 14, 2018

The market for CombiGene's epilepsy project

Epilepsy is a global problem. The disease affects an estimated 0.6 to 0.8 percent of the world’s population. In 2016 there were 5.7 million diagnosed epilepsy patients in the USA, the EU4*, the UK, and Japan. About one-third of these patients do not respond to conventional medical treatment. In addition, there are many thousands of new patients with focal drug-resistant epilepsy every year. It is this patient group CombiGene intends to help with its candidate drug, CG01.

* EU4 = France, Germany, Italy, and Spain

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 candidate drug CG01. 

European Union’s Horizon 2020

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