About the European Society of Gene and Cell Therapy

During the last few years gene therapy has delivered convincing evidence that clinical benefit can be achieved with gene transfer techniques in inherited diseases. After several years of uncertainty, renewed optimism and excitement has filled the air. European Society of Gene and Cell Therapy (ESGCT) is one of the leading organizations in the field and is committed to offer continuous support to basic and translational research and clinical applications of gene and cell therapy for inherited and acquired diseases. Many of the recent advances have been made in Europe and ESGCT has played an active role in increasing awareness of gene and cell therapy through its Annual Congress and numerous interactions with other societies, funding organizations and regulatory bodies.

The field has matured during the last twenty years, since the very first attempts to treat human diseases with gene therapy. We have realized that development of gene drugs faces similar challenges as other biological drugs. However, there is a wide-spread feeling that gene therapy products are still even more tightly scrutinized because of various biological, environmental and ethical reasons. Therefore, it is very encouraging that successful clinical applications have been developed in inherited diseases, although we have also witnessed severe adverse events due to uncontrolled integration of vectors into the patients’ genome. In spite of these setbacks and issues around tight regulation of the manufacturing of gene therapy medicinal products, European research groups are leading the way forward and also getting support from European Union funding system and regulatory authorities at the national and EU level. One example is the Hospital Exemption Regulation that will allow academic institutions and hospitals to produce limited quantities of gene and cell therapy medicinal products for clinical testing under physician’s supervision without having a full GMP manufacturing license.

Even though clinical applications have always been the most visible area in gene and cell therapy research, progress in the field is undoubtedly based on advances in basic research revealing specific targets for gene therapy, vector development, improvement of the delivery methods and refining ex vivo gene transfer conditions. However, it is clear that much still needs to be done to achieve regulated, tissue-specific gene expression, targeted integration, precise local delivery and lower toxicity of the vectors. Also, fundamental issues related to immunological responses against both the vectors and the transgenes need to be explored.

Gene transfer techniques have become very useful research tools in several areas of basic research, and it would be very important to achieve an even more wide-spread understanding of the possibilities to use gene transfer technologies in the diverse areas of biological research from plant biology to mammalian cell biology, developmental biology and pathophysiological studies.

Stem cells and iPS cell biology is an area where rapid progress can be foreseen and where gene transfer techniques play a crucial role in both the generation of iPS cells and guiding differentiation into specific cell types. In this area advanced vector technology and cell technology are both needed to lay background for future applications. Experience from difficulties in the translation of gene therapy products to clinical arena can also be very useful for future clinical applications in cell therapy.

General role of ESGCT in supporting all these activities is an important cornerstone for future success of gene and cell therapy in Europe. ESGCT will promote basic and translational research and encourage clinical applications of gene and cell therapy. It will also facilitate education and exchange of information and technologies through its Annual Congress, Educational Sessions and other meetings and research consortia, where ESGCT is involved. Also, support to national societies and fruitful interaction with their members and national meetings will be very important for ESGCT. Increasing awareness of gene and cell therapy among researchers, clinicians and lay people will be very important for the further growth of the Society. We will aim to have more than 1000 active members in ESGCT in the next two years. Continued success of ESGCT Annual Congress as one of the leading gene therapy symposia in the world is the highlight of the Society.

I would like to invite all researchers in basic science and translational medicine, clinical colleagues and representatives from industry, regulatory agencies, public and private funding bodies and patient organizations with an interest in future novel therapies to join ESGCT as active members and to participate in our Annual Congress. As active members of the research community strongly supported by ESGCT it will be easier to develop these techniques into advanced research tools in basic science and to wide-spread clinical applications for the treatment of severe inherited and acquired diseases.

Seppo Ylä-Herttuala, MD, PhD, FESC
President
European Society of Gene and Cell Therapy