Medical and Regulatory Affairs

Applied Clinical Trials: The Rise of Gene Therapy: Advanced Regulatory, Site, and Enrollment Considerations

Gene therapy development is gaining momentum. In 2020 alone, gene therapy attracted $12 billion in global financing through just the first three quarters with more than 370 gene therapy clinical trials underway worldwide.1 The U.S. Food and Drug Administration (FDA) itself predicted it may be approving 10 to 20 gene therapies per year by the year 2025.2As gene therapy research expands across modalities and therapeutic areas, sponsors are tasked with designing and executing safe, ethical trials in a fast-changing regulatory environment.

In this article, we explore key regulatory, site, and enrollment considerations for gene therapy trials, offering tips on successfully navigating the development process based on evolving best practices and our own experience in this area.