CombiGene´s lead project CG01 has received funding from the European Union´s Horizon 2020 research and innovation programme

Market for gene therapy


Commercially, gene therapy is still at the starting line. There are a few approved gene therapy products, but 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 resolve issues concerning remuneration systems and regulatory frameworks.

A large number of clinical studies and significant investment
Internationally, 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 362 clinical studies being conducted in the gene therapy field, of which 120 are phase-I studies, 210 are phase-II studies and 32 are phase-III studies (Alliance for Regenerative Medicine, Q4 report 2018). Confidence in gene therapy is also demonstrated by the massive flow of capital into this field. During 2018 alone, investment in gene-therapy-related research and development amounted to USD 9.7 billion (Alliance for Regenerative Medicine, Q4 report 2018).

The emphasis is in the area of oncology, but cardiovascular diseases and diseases related to the central nervous system are also very common.

Historical breakthrough in the USA
On January 30th 2017 the US Food and Drug Administration (FDA) approved the first gene therapy for the US market. Kymriah, from the pharmaceutical company Novartis, has been developed for treatment of paediatric patients and young people with acute lymphoblastic leukaemia. FDA approval demonstrates the administration’s confidence in the safety and effectiveness of a gene therapy product, and a range of other gene therapies will be approved in the USA and Europe in the coming years.

Big interest from Big Pharma
Interest in gene therapy has in recent years increased markedly among the major pharmaceutical companies. During the first quarter of 2019 alone more than ten acquisitions or inlicensing deals between gene therapy companies and Big Pharma were realized. One example is Roche’s acquisition of Spark Therapeutics, a company that is developing a gene therapy treatment for haemophilia. The price was USD 4.3 billion, as compared with Spark’s market capitalization, which at the time amounted to USD 2 billion.