The 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

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 366 clinical studies being conducted in the gene therapy field, of which 117 are phase-I studies, 219 are phase-II studies and 30 are phase-III studies (Alliance for Regenerative Medicine, Q4 report 2019). 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.

Historic breakthrough in the USA

On August 30th 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 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 acquisition price was USD 4.3 billion, as compared with Spark’s market capitalization, which at the time amounted to USD 2 billion.

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 has an exclusive collaboration and licensing agreement for the CG01 project with Spark Therapeutics.

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, Agavägen 52A, SE-181 55 Lidingö


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