The state of cell and gene therapy in 2023

Abstract

Progress in the understanding of human diseases has coincided with the advent of precision medicine, whereby the underlying genetic and molecular contributors can be used as diagnostic and therapeutic biomarkers. To address these, drug developers have designed a range of different treatment strategies, including gene therapy, which the American Society of Gene and Cell Therapy defines as the use of genetic material to treat or prevent disease. A number of approaches exist, including the delivery of genetic material in vivo or ex vivo, as well as the use of RNA species to alter gene expression in particular disease states. Through the end of the first quarter of 2023, there were more than 100 different approved gene, cell, and RNA therapies throughout the world, with over 3,700 more in clinical and preclinical development. This review comprehensively captures the landscape for such advanced therapies, including the different genetic technologies used and diseases targeted in clinical trials.

Keywords: RNA therapy; approved products; cell therapy; clinical trials; gene therapy; genetically modified cell therapy.

Graphical abstract

Figure 1

Current universe of advanced therapies, including areas of overlap.

Figure 2

Biopharmaceutical industry pipeline of gene, cell, and RNA-based drugs under active development.

Figure 3

Divisions of gene, cell, and RNA therapy pipeline by technology.

Figure 4

Number of clinical trials initiated each year for gene, cell, and RNA therapies.

Figure 5

Breakdown of active gene, cell, and RNA therapy clinical trials by status and therapy area (TA).

Figure 6

Therapy area concentration for active clinical trials of gene, cell, and RNA therapies Please note that therapeutic areas may overlap.