The U.S. Food and Drug Administration (FDA) held a public listening session on September 18, 2025, aimed at harnessing previous knowledge to enhance the development and regulatory review of cell and gene therapy (CGT) products. This session, organized by the FDA’s Office of Therapeutic Products (OTP), brought together industry experts to discuss how existing data could streamline processes and improve patient outcomes in CGT.
Among the panelists were renowned professionals in the field, including Paris Margaritis, PhD, Chief Scientific Officer at GeneVentiv Therapeutics, and Jacob Smith, Head of Technical Development and CMC Strategy at Viralgen. Their insights highlighted the importance of leveraging prior knowledge across various aspects of CGT, including chemistry, manufacturing, controls (CMC), and clinical trials.
Key Themes from the FDA Listening Session
The discussions focused on several critical themes that could shape the future of CGT development. Panelists emphasized the necessity of identifying what types of data are most beneficial to share among stakeholders, along with the importance of setting clear boundaries on data use. This approach is vital for ensuring safety and efficacy while navigating the complexities of product development.
Additionally, the session addressed how sponsors and Contract Development and Manufacturing Organizations (CDMOs) can effectively collaborate and share data throughout the product lifecycle. The integration of knowledge across different disciplines was identified as a crucial strategy to enhance development efficiency and risk assessment.
Attendees of the accompanying webinar had the opportunity to engage directly with experts during a live Q&A session, allowing for a rich exchange of ideas and clarification of complex points.
Looking Ahead: Implications for Developers and Regulators
The insights gathered from the FDA’s listening session hold significant implications for both developers and regulators in the CGT space. By fostering a collaborative environment where knowledge sharing is prioritized, the industry can make strides towards quicker, safer, and more effective therapies.
As Paris Margaritis noted, the connection between scientific expertise and practical application is key to advancing CGT. His extensive experience, particularly in AAV gene therapy for hemophilia, adds a vital perspective to the ongoing discussions about product safety and innovation.
Similarly, Jacob Smith‘s background in manufacturing processes, including his role in developing the Pro10 manufacturing system, underscores the importance of robust technical strategies in ensuring successful therapy development.
The outcomes of this session signal a proactive approach by the FDA to engage with the CGT community, indicating a willingness to adapt regulatory frameworks to better accommodate the evolving landscape of gene therapies. As the industry progresses, the insights from this session will likely inform future strategies for both development and regulatory oversight, ultimately benefiting patients in need of innovative therapies.
Through initiatives like this, the FDA demonstrates its commitment to fostering collaboration and efficiency within the CGT sector, paving the way for advancements that could transform treatment landscapes worldwide.