The human body is a remarkable structure composed of countless minuscule cells—the basic building blocks of life. Like bricks used to build a house, the intricate organization of these cells forms the complex structure of the body. However, like any structure, the body is not invulnerable and can suffer from structural damage and dysfunction like injuries, diseases, and wear and tear. Cell and gene therapies (CGTs) provide groundbreaking approaches to repairing or replacing dysfunctional cells and enabling the smooth functioning of the human body.

Although still in their early days, these therapies could change the way we tackle previously untreatable diseases. There is no better illustration of their potential than in the recent case of a 19-month-old baby girl in the UK diagnosed with a rare genetic disorder (metachromatic leukodystrophy [MLD]), who showed no signs of it after being treated with a gene therapy called Libmeldy developed by Orchard Therapeutics. The treatment involved extracting stem cells from the child, replacing the faulty gene with a healthy one capable of producing a crucial enzyme, and administering the modified cells back into the body. 

As of August 2023, more than 30 CGTs have been approved by the US FDA, with several other therapies awaiting approvals as of the same date. The number of clinical trials being conducted for CGTs totaled 1,600+ across the North American, European, and Asia-Pacific regions.