Gene and cell therapies in Europe

Gene and Cell Therapies in Europe: Dreaming the Future explores the exciting progress in the world of Advanced Therapy Medicinal Products(s). This book highlights that, after years of research, new medical possibilities are emerging for conditions that were previously almost untreatable. It describes the successes of cell therapy (CAR T-cells) in cancer and of gene therapy in blood diseases, like sickle cell disease and haemophilia, that go beyond symptom management and offers the prospect of a cure. The book shows the great expectations of academic researchers, the pharmaceutical industry, healthcare providers, patients, caregivers and their organisations regarding gene and cell therapy in Europe. These high hopes are in stark contrast to the slow system of legislation, regulations and reimbursement, which is still far from harmonised in the European Member States. It is uncertain how the political situation will evolve in 2025. As a result, it is difficult to predict how ATMP production will develop. This could provide an extra stimulus to carry out the recommendations of the Draghi report and to profile the EU as a centre of excellence in the R&D of ATMPs. Professor Thierry Vandendriessche writes in his preface: ‘The authors adequately described some of the challenges to provide sustainable, affordable, effective and safe gene therapy treatments […] this book will become a useful reference for the many key stakeholders in this exciting field of gene therapy’.