Cell & gene therapies have emerged as revolutionary treatments for a variety of diseases, including cancer, genetic disorders, and autoimmune conditions. These therapies are designed to modify or replace a patient's genetic material or cells to treat or prevent disease. 

Cell & gene accredited contract research organizations (CROs) play an essential role in managing and conducting clinical trials for these innovative therapies, providing a range of specialist services from regulatory consultancy, drug development planning, site selection, and data management.

The clinical trial landscape for cell & gene therapy is constantly evolving, as new therapies are developed and existing ones are refined. According to GlobalData, these trials are taking place globally, with the US, China, and Europe leading the way in terms of the number of trials.

Cell and gene therapy trials in the Asia Pacific region are growing 50% faster than ROW with the majority of trials in oncology, specifically for blood cancers, viral infections, and solid tumors. After oncology, the cell and gene trials are in infectious diseases, CNS, and cardiovascular diseases. 

The region already accounts for over a third of cell & gene therapy trial activity with China showing a 15% faster growth rate than the ROW.

In addition, the Asia Pacific is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. 

One of the major challenges in conducting clinical trials for cell & gene therapy is the complexity of the treatments themselves. Unlike traditional therapeutics, which are typically small molecules or proteins, cell & gene therapies involve complex biological processes and require specialized manufacturing and delivery methods. As a result, clinical trials for these therapies require highly specialized CRO expertise and an experienced team.

Virtual clinical trials are a promising development in this clinical trial landscape, offering several advantages over traditional trials, including expanded patient access, reduced costs, improved patient recruitment and retention, and faster time to market. Virtual clinical trials involve the use of digital technologies to collect data remotely, allowing patients to participate in clinical trials from the comfort of their own homes. CROs are increasingly offering virtual clinical trial programs, including virtual site monitoring, eConsent, and telemedicine.

Despite the challenges associated with developing and conducting clinical trials for cell & gene therapy, the potential benefits of these treatments are significant. Cell and gene therapies have the potential to cure or significantly improve the lives of patients with a variety of diseases, many of which are currently incurable or difficult to treat.

An example of a successful cell and gene therapy is Kymriah, a treatment for pediatric and young adult patients with certain types of leukemia. Kymriah is a CAR-T cell therapy, which involves genetically modifying a patient's own immune cells to recognize and attack cancer cells. In clinical trials, Kymriah was found to be highly effective in treating patients with relapsed or refractory leukemia, and was approved by the FDA in 2017.

Another example is Luxturna, a gene therapy for Inherited Retinal Dystrophy caused by a gene mutation. Luxturna is the first directly administered gene therapy approved in the US that targets a disease caused by mutations in a specific gene. Luxturna was approved by the FDA in 2017.