Plenary Session 12: Updates in the Regulatory Landscape for Cell & Gene Therapy Products

Several cell and gene therapy products have received marketing approval from health authorities and, with that, a more complete picture is now emerging about the regulatory CMC requirements to commercialize these products. In the past couple of years, AAV-based gene therapies, autologous cell-based gene therapies (e.g., CAR-T cell products), and tissue-based products have received marketing authorization. During this period, many guidelines were issued by regulatory agencies to address CMC and clinical considerations for development of cell and gene therapies and additional stakeholder support mechanisms were introduced to provide early feedback for product development and manufacturing, such as FDA-CBER’s INTERACT and CATT meetings, respectively, and EMA’s PRIME designation.




In this session, we will engage regulators and hear their perspectives on how these initiatives can be leveraged towards successful clinical development of cell and gene therapy products. The session will provide a forum for regulators and developers to highlight the CMC challenges and to share in the learnings from successful development of this diverse array of innovative medicines; specifically, in the strategies that can be applied going forward in a pragmatic and risk-based manner for manufacturing high quality, potent and safe CGT products.




Panelists:




Jounghee Baek, NIFDS, South Korea MFDS

Andrew Byrnes, CBER, FDA

Ilona Reischl, Austrian Medicines and Medical Devices Agency