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 several 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
Confidence in gene therapy is now at a very high level, as evidenced by, among other things, the large number of clinical studies currently under way throughout the world. There are now more than 500 clinical studies being done in the gene therapy field, of which 184 are Phase I studies, 286 are Phase II studies and 34 are Phase III studies (Alliance for Regenerative Medicine, Q2 report 2017). Confidence in gene therapy is also demonstrated by the massive flow of capital into this field. In the second quarter of 2017 alone, investment in gene-therapy-related research and development amounted to more than USD 1.18 billion (Alliance for Regenerative Medicine, Q2 report 2017).

The main emphasis is on studies in the oncology field, as well as cardiovascular diseases and diseases related to the central nervous system.

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. Kymriah, from the pharmaceutical company Novartis, has been developed for treatment of paediatric patients and young people with treatment-resistant acute lymphatic leukaemia. FDA approval demonstrates the administration’s confidence in the safety and effectiveness of a gene therapy product, and several other gene therapies may be expected to be approved in the USA and Europe in the coming years.