Back to: FDA Cell and Gene Therapy Roundtable (June 2025)
Abstract
This extensive discussion revolves around the advancement and regulation of cell and gene therapies, particularly for rare and ultra-rare diseases, with a strong emphasis on pediatric patients. Experts, including researchers, physicians, patient advocates, and government officials, highlight the transformative potential of these therapies, citing successful treatments for conditions previously considered incurable. A recurring theme is the urgent need for regulatory flexibility and efficiency from the FDA to accelerate development and approval, especially for therapies that may not fit traditional commercial models due to small patient populations. Speakers also address the importance of reducing development costs, fostering collaboration between academia and industry, ensuring patient access and affordability, and maintaining U.S. leadership in this rapidly evolving field amidst international competition. The conversation underscores the critical balance between scientific innovation, patient safety, and practical pathways to bring these life-changing treatments to those in need.