Gene therapy clinical trials, where do we go? An overview

Abstract

There have been many ups and downs since the introduction of gene therapy as a therapeutic modality for diseases. However, the journey of gene therapy has reached a fundamental milestone, as evidenced by the increasing number of gene therapy products on the market. Looking at the currently approved and under-approval products, as well as the numerous clinical trials in this field, gene therapy has a promising future. Trend of changes in gene therapy strategies, vectors, and targets could be insightful for pharmaceutical companies, policymakers, and researchers. In this paper, following a brief history of gene therapy, we reviewed current gene therapy products as well as gene therapies that may be approved in the near future. We also looked at ten-year changes in gene therapy clinical trials strategies, such as the use of vectors, target cells, transferred genes, and ex-vivo/in-vivo methods, as well as the major fields that gene therapy has entered. Although gene therapy was initially used to treat genetic diseases, cancer now has the greatest number of gene therapy clinical trials. Changes in gene therapy strategies, particularly in pioneering countries in this field, may point to the direction of future clinical products.

Keywords: Cancers; Clinical trials; Gene therapy; Genetic diseases; Genetic vectors; Trends.