Gene Therapy Research Unit, Children's Medical Research Institute, The University of Sydney and The Sydney Children's Hospitals Network, Westmead, NSW, Australia.
Discipline of Child and Adolescent Health, The University of Sydney, Westmead, NSW, Australia.
J Gene Med. 2024 Aug;26(8):e3721. doi: 10.1002/jgm.3721.
To date, 3,900 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. Our database brings together global information on gene therapy clinical activity from trial databases, official agency sources, published literature, conference presentations and posters kindly provided to us by individual investigators or trial sponsors. This review presents our analysis of clinical trials that, to the best of our knowledge, have been or are being performed worldwide. As of our March 2023 update, we have entries on 3,900 trials undertaken in 46 countries. We have analyzed the geographical distribution of trials, the disease indications (or other reasons) for trials, the proportions to which different vector types are used, and which genes have been transferred. Details of the analyses presented, and our searchable database are on The Journal of Gene Medicine Gene Therapy Clinical Trials Worldwide website at https://a873679.fmphost.com/fmi/webd/GTCT. We also provide an overview of the progress being made around the world, and discuss key trends since the previous review, namely the unprecedented increase in gene therapy clinical trial activity, including the implementation of genome editing technology with the potential to transform the field moving forward.
截至目前，全世界已完成、正在进行或已批准的基因治疗临床试验有 3900 项。我们的数据库汇集了来自临床试验数据库、官方机构来源、已发表文献、会议演讲和海报的全球基因治疗临床活动信息，这些信息是由个别研究人员或试验赞助商友好提供给我们的。本综述介绍了我们对全世界已完成或正在进行的临床试验的分析。截至 2023 年 3 月的更新，我们有来自 46 个国家的 3900 项试验记录。我们分析了试验的地理分布、试验的适应症（或其他原因）、不同载体类型的使用比例以及已转移的基因。在 https://a873679.fmphost.com/fmi/webd/GTCT 上的《基因治疗杂志》基因治疗临床试验全球网站上，可以查看分析详情和我们可搜索的数据库。我们还概述了全世界正在取得的进展，并讨论了自上次综述以来的关键趋势，即基因治疗临床试验活动空前增加，包括具有潜在改变该领域未来的基因组编辑技术的实施。
