Ensuring Quality in Cell-Based Therapies: CMC Principles and a Regulatory Science Perspective

Abstract: TCell-based products present new therapeutic opportunities in medicine, but their inherent complexity as living drugs introduces unique scientific and manufacturing challenges. This presentation will explore the critical Chemistry, Manufacturing, and Controls (CMC) considerations that support a successful cell therapy program, emphasizing how these requirements evolve from early to late clinical development. Key topics include the scientific challenges of demonstrating comparability following manufacturing changes and developing biologically relevant potency assays that link critical quality attributes to therapeutic function and clinical performance. In addition, the talk will provide an overview of the regulatory process for cell-based products and include regulatory science project examples that illustrate approaches to addressing key challenges in evaluating and supporting these innovative therapies, underscoring the importance of a proactive, science-driven CMC strategy in delivering safe, consistent, and effective cell therapies to patients.

Learning Objectives:
1. Understand how CMC expectations for cell-based therapies evolve throughout clinical development and the scientific principles that guide these expectations.
22. Recognize the challenges associated with manufacturing changes, comparability assessments, and potency assay development for complex cellular products.
3. Gain insight into regulatory processes and regulatory science initiatives that inform the evaluation, consistency, and safety of emerging cell-based therapies.