CMC Progression from Early to Late Phase – Practical Considerations for Gene Therapy

The principles for developing traditional biologics are well established, but do not always translate well to cell and gene therapy products.  Creative yet practical adaptations are necessary to advance gene therapy programs toward licensure.  During this presentation we will explore three key areas where practical adaptations are required: Building toward the commercial process: change control and managing product evolution for non-traditional development pathways, comparability challenges, and regulatory expectations Maturation of analytics – the level of product understanding expected as programs advance, method advancement from early to late phase, ensuring repeatability and robustness in method performance Finding the right partners – finding the right match in terms complementary capabilities, technical performance, phase-appropriate GMP compliance and client service infrastructure