Cell and gene therapy leaders discuss industrialization and AI regulation

At the Cell and Gene Meeting on the Mesa, industry leaders and regulators discussed the evolution of the cell and gene therapy (CGT) sector, focusing on the transition from commercialization to industrialization. Experts noted that traditional pharmaceutical models, designed for mass-producing pills, are ill-equipped for the personalized nature of CGT. Achieving scale requires upgrading manufacturing, distribution, and administration processes to handle individual patient needs.

Discussions also addressed the integration of artificial intelligence in the field. Steven Oh, Director of the Office of Cellular Therapy and Human Tissue at the FDA, emphasized that regulatory scrutiny will focus on the credibility and context of AI models rather than just their functionality. Due to the limited data available for rare diseases, the FDA encourages early engagement through CBER Advanced Technologies Team (CATT) meetings and Pre-IND meetings to ensure technology meets safety and documentation standards. The agency is also utilizing its own internal, secure AI tools, such as ELSA, to manage data within its firewall.