Accelerating Innovative Cell Therapy Development Through Phase-Appropriate Potency Control

By Deb Bhattacharya, Ph.D. and Cindy Riggins, Ph.D.

Cell and gene therapies are revolutionizing medicine by offering targeted treatments that address disease at a genetic and cellular level. These therapies rely on gene delivery technologies such as viral vectors and non-viral methods to introduce or modify genetic material, equipping cells with therapeutic properties. A critical component of their development is potency assays, which measure the biological activity of a product to ensure consistency, efficacy, and safety. Potency assays must reflect the therapy’s mechanism of action and evolve throughout the development lifecycle, from early research phases to pivotal clinical trials and commercialization.

For CAR and TCR T cell therapies, potency assays assess gene integration, protein expression, cytokine release, and cell-killing activity, using techniques like qPCR, flow cytometry, and ELISA. Developing these assays requires extensive optimization, including selection of critical reagents, establishment of reference materials, and validation of analytical methods. Regulatory expectations emphasize a lifecycle approach to potency, ensuring that assays align with evolving knowledge of the therapy’s function. Many companies, particularly smaller biotech firms, face challenges in developing robust potency strategies due to limited resources and expertise. Establishing a well-defined potency control strategy early in development is crucial for regulatory compliance and the successful commercialization of cell and gene therapies.