Transforming Medicine: Strategies In Cell And Gene Therapy Development

By Erin Finot, MS, MBA, Vice President, Immuno-Oncology and CAGT, and Diego Correa, MD, PhD, Vice President and Global Head, Cell and Gene Therapy Center of Excellence

Cell and gene therapies (CAGTs) are transforming treatment for genetic disorders, with rising FDA approvals and global trials. However, rapid advancements bring regulatory challenges, requiring compliance with jurisdictional approvals, environmental regulations, documentation, and long-term safety monitoring to ensure patient safety and efficacy.

Beyond regulations, CAGT programs face operational hurdles, including innovative trial designs, patient identification, and logistical complexities in manufacturing and distribution. Applying CAGT insights to immuno-oncology underscores the need for specialized facilities, interdisciplinary collaboration, and real-time patient monitoring. CAR-T therapy exemplifies the benefits of personalized treatment, continuous assessment, and combination therapy research, offering a model for broader immuno-oncology advancements. As CAGTs evolve, adaptive strategies will be crucial to accelerating development and ensuring timely patient access.