Gene and cell therapy refer to introduction, removal, or change in the content of a person’s genetic code (gene therapy) or administration of living whole cells (cell therapy) to patients to achieve desired therapeutic effects.
Gene and cell therapies are currently being intensively developed to tackle diseases such as cancer, cardiovascular and autoimmune disorders. These new approaches have shown great potential, and during recent years several important milestones within the field of gene and cell therapy have been achieved.
To demonstrate the progress, the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have approved chimeric antigen receptor T-cell (CAR-T) therapy as a treatment for blood diseases, such as large B-cell lymphoma and acute lymphoblastic leukemia [1,2]. CAR-T therapy was also recently introduced in clinical practice in Finland (Helsinki University Hospital) . Clinical evidence of CAR-T-based and other gene and cell therapies have been highly encouraging and sheds light to diseases that have been earlier considered untreatable.
Development of new and safe gene and cell therapies requires active, multidisciplinary research. Finland has a long history of being in the forefront of gene and cell therapy research and Finland is highly recognized also internationally. New infrastructures, such as the Kuopio Center for Gene and Cell Therapy (KCT), further facilitate these efforts.
Kuopio Center for Gene and Cell Therapy
KCT is a newly formed research center located in the Northern Savonia of Finland, in the city of Kuopio. KCT was founded and is owned by the Frederik Paulsen Foundation and currently it employs approximately 30 researchers. KCT’s focus areas include applied and translational research emphasizing particularly the development of viral vectors for novel gene therapy products and engineering of primary cells for cell therapy products. The new research center offers services related to the lifespan of the product development, starting from the basic biological research to the first phase clinical trials and manufacturing procedures. Current operations are mostly connected with the initial sponsor’s projects, but the center is fully open for new partnerships and innovations. KCT is also actively searching for new research ideas, providing also possible funding channel for new innovative approaches. KCT is a unique operator in Finland positioning itself between academic research and the pharmaceutical industry.
Medaffcon met with Vesa Turkki, PhD, Head of KCT’s Analytical Unit, to discuss the current operations of the KCT and visions of the future possibilities of gene and cell therapies.
“We try to keep the research environment as flexible and innovative as in academia, but with a translational capability to enable innovative potential therapies to reach the clinic. We envision that the center will expand up to 50 researchers. This will keep the organization dynamic and swift”, Vesa Turkki explains.
KCT has divided its operations into three units; the cell therapy and gene therapy main units, supported by a smaller analytical core unit.
The cell therapy unit focuses on both non-genetical modified cells and genetically modified cell therapies. KCT is currently developing industrial level manufacturing processes, where healthy donor blood cells are enriched and transformed into a regenerative therapeutic product. Collaboration with national and international partners is seen essential to advance these activities.
Gene therapy unit focuses on the manufacture and development of the viral vectors needed in gene therapies. Vectors are important elements in gene and some cell therapies due to being critical in the transfer of genetic material to specific targets. In addition to having state-of-the-art equipment, personnel of the center have decades of expertise in the development of viral vectors.
The analytical unit supports these two main units and is responsible for maintaining and developing everyday analytics. The analytical core is important for the fluency of operations and reproducibility.
The overall goal of collaboration between the units is to research and develop new safer and more effective Advanced Therapy Medicinal Products (ATMPs). Scientific knowledge is gained every day, however, developing an effective therapy is a result of several years’ work.
“It is vital to acknowledge the extent of both innovation and experience needed to work effectively in the gene and cell therapy. Many startups and pharma companies have recently entered the space. Our team members have acknowledged scientific track records and vast experience and therefore we strongly believe that KCT has a bright future in the field.”, Vesa Turkki points out.
The Kuopio health campus, residing KCT, includes the University of Eastern Finland, A.I. Virtanen Institute, Kuopio University Hospital (KUH), Lab Animal Center, Finnish Medicines Agency (FIMEA) and several other pharma/biotech companies. This makes flexible collaboration between institutions and companies possible. KCT is in tight collaboration with several different operators of the campus.
“Several of the important operators that we collaborate with are just a few minutes’ walk away. Furthermore, Kuopio is a research and innovation city, and has been steadily building its reputation within the research community”, Vesa Turkki concludes.
There is no doubt that gene and cell therapies are an important landmark towards more effective and personal treatments. However, these kinds of never treatments needs years of disciplined research, enormous resources and tight collaboration. Gene and cell therapy products will have an increasing presence in the near future and this will lead to therapeutic solutions to treat diseases in areas of high unmet clinical need where traditional small molecules have failed.
More about KCT