Affiliation:
1. Gene Therapy Research Unit, Children's Medical Research Institute The University of Sydney and The Sydney Children's Hospitals Network Westmead NSW Australia
2. Discipline of Child and Adolescent Health The University of Sydney Westmead NSW Australia
3. Azrieli Faculty of Medicine Bar‐Ilan University Safed Israel
4. Section for Clinical Immunology and Transfusion Medicine Unilabs Laboratory Diagnostic Services Stockholm Sweden
Abstract
AbstractTo 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.
Funder
Children's Medical Research Institute