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.
Belief in gene therapy is at a very high level. This is exemplified by, among other things, the large number of clinical studies that are now under way throughout the world. There are currently 372 clinical studies being conducted in the gene therapy field (Alliance for Regenerative Medicine, H1 report 2022).
Confidence in gene therapy is also demonstrated by the massive flow of capital into this field. During 2021, investment in gene-therapy-related research and development amounted to USD 10,2 billion (Alliance for Regenerative Medicine, report 2021). The emphasis is in the area of oncology, but cardiovascular diseases and diseases related to the central nervous system are also very common.
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.
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). 
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.
 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
Partial lipodystrophy is a very rare disorder. Estimates place the number of patients in the USA and Europe at about 2,000. Therefore, according to CombiGene’s assessment, there are good prospects for obtaining orphan drug designation for the lipodystrophy project’s candidate drug. If orphan drug designation is granted for the candidate drug, CombiGene will have several significant advantages in the form of regulatory advice, tax relief, public funding, reduced fees and longer market exclusivity.
Given the advantages of an orphan drug designation, CombiGene may potentially develop this project all the way to market under its own management.
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.
CombiGene’s lead project CG01 has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 823282