Abstract
I vividly remember attending the first annual meeting of the European Society of Gene Therapy (later renamed to include also Cell Therapy) in Tampere, Finland, in 2004. With about 500 attendees and 60 oral presentations, the meeting impressed me due to its small size, which facilitated exchanges, and the very active, passionate, and innovative community of mostly European scientists. At that time, there were few clinical trials, the majority ex vivo, and industry attendance was very limited. Fast forward 20 years, and there are now over a dozen gene therapy products approved on the market, including one based on genome editing, with thousands of patients treated successfully worldwide. Biotech and industry in addition to academia have significantly contributed to technological and clinical progress. Gene and cell therapy, which Naldini and colleagues defined as a “renaissance” in 2016, now seem to have transitioned to a full “revolution”, if we continue with the historical analogy.
The 31st ESGCT annual meeting, which will be held in Rome at the end of October 2024, will reflect the ongoing revolution in the field. On one side, there is an increased possibility to translate research from bench to bedside due to advances in vector design, manufacturing, and regulatory paths. These will need to evolve continuously to increase sustainability by exploring innovative models that do not necessarily see industry as the sole depositary of clinical development and post-market distribution. On the other side, the continuous development of novel editing approaches allows for single nucleotide substitutions as well as the precise integration of large therapeutic DNA, while novel potent and safe vectors, both viral and non-viral, target compartments such as whole body muscle or the brain from the bloodstream. New gene therapy platforms, which appeared revolutionary in their design and conception just a few years ago, are now being used successfully in humans solving previously unsolvable problems.
The current issue of Human Gene Therapy celebrates the 31st ESGCT annual meeting and the (r)evolution in our field. In this issue, you will find reviews authored by international experts covering a range of topics from in and ex-vivo gene therapy to genome editing, viral and nonviral vectors as well as gene-specific and gene-agnostic approaches. A similar variety of topics are addressed by high-impact original research papers which are also included in this issue. The future ESGCT congress, along with this special issue, marks a new era in health care with lifesaving nucleic acid-based drugs as well as new technological developments and challenges that lie ahead. The continuous cycle from bench to bedside and back to bench remains crucial for improving our knowledge and designing novel and better therapies. The (r)evolution does not stop.