Abstract

A
Over this time, European gene therapy investigators have led the world with numerous stunning achievements. Most notable among these have been achieving clinical efficacy in treating previously untreatable genetic disorders, leading to the first clinically approved adeno-associated virus and gammaretrovirus (gRV) vectors in the world in the form of Glybera® (alipogene tiparvovec) and Stimvelis® (autologous CD34+ cells transduced with gRV-ADA vector). These two approvals are not isolated examples, but rather are indicative of the steady progress that has been made in the fields of gene and cell therapy in Europe in recent decades.
These accomplishments have been enabled by the steady approach taken by the European Union in its funding of biomedical research, as outlined by Dr. David Gancberg in his perspective piece in this issue. 1 This predictable pace of funding stands in contrast to the erratic swings in funding in the United States, where investigators experienced a surge in National Institutes of Health funding associated with the American Recovery and Reinvestment Act of 2009 followed by a sharp decrease during the budget sequestration of 2013. In addition, the European Medicines Agency has enabled these developments, overcoming potential complications of national boundaries, particularly for the class of advanced therapy medicinal products to which gene and cell therapies belong. This environment, populated by the gifted European researchers who comprise the ESGCT, bodes well for ever-accelerating progress and ongoing success.
In order to provide some insight into just how broad and impactful the work of the ESGCT is, this issue includes invited reviews and original contributions from major European leaders in the field, all of whom participated first-hand in these achievements. We wish to thank the authors for these contributions. We also wish to thank the ESGCT Board of Directors and all of its members for their dedication to bettering the lives of the patients helped by these therapies. We most especially want to thank our Guest Editor, Professor Hildegard Büning, for her tireless efforts in assembling this important special issue.
