Since the first gene therapy trial in 1990, the regulatory landscape has shifted from broad, loosely defined guidance with little variation between different cell and gene therapies to a more robust collection of guidelines and directives focused on testing and manufacturing considerations for these products in specific disease states and therapeutic settings. This article will explore the importance of new factors in gene therapy clinical study start-up, including environmental risk assessment and biosafety, as well as how guidances from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) continue to evolve in a rapidly changing regulatory landscape.
U.S. and EU regulatory guidelines: catching up with the research
In the U.S., the FDA appears intent on bringing regulatory guidance up to speed with recent advances in cell and gene therapy research. Indeed, the FDA has issued nine separate guidances since January 2020, specifically focusing on cell and gene therapy.1 The National Institutes of Health (NIH) likewise recently released guidelines specifying biosafety practices and containment strategies for constructing and handling gene therapy products.2