Gene and Cell Therapy – An Overview of the Current and Changing Regulatory Requirements and Study Design for Nonclinical Safety Evaluation
Presenters: Mark D. Johnson, M.S. and Scott Boley, Ph.D., DABT
Session description: Nonclinical development of gene and cell therapies has been ongoing for more than three decades, and has been increasing in recent years. In the early days of these efforts, these therapies shared the fate of many novel experimental medicine approaches with progress impeded by severe side effects in treated patients or a failure to demonstrate efficacy in the target patient population. Research over the past decade has resulted in the development of highly sophisticated gene transfer tools with improved safety and therapeutic efficacy. As these tools have been developed, regulatory agencies worldwide have adapted new review processes and developed a wide range of guidance documents to aide researchers in designing rigorous nonclinical studies to ensure the safety, biodistribution and potential benefit of these advanced therapeutics. This presentation will provide an overview of the regulatory guidelines, provide some key considerations in the nonclinical development and testing of cellular and gene therapies, and detail several case studies of these therapeutics.