Mycoplasma testing is essential for ensuring the quality of biotechnological products and cell-based materials, yet traditional methods are constrained by long turnaround times, testing volume requirements, and reliance on specialized laboratories. USP <77> introduces nucleic acid amplification tests (NATs) for the rapid, qualitative detection of mycoplasma nucleic acid and provides a risk-based framework for their implementation. This presentation outlines key elements of GC <77>, including assay controls, validation parameters, and comparability considerations, and highlights USP’s evolving position on Modern Microbiological Methods (M3).

This presentation will explore the growing need for rapid sterility testing strategies tailored to cell-therapy development and manufacturing. It will highlight the unique time, logistics, and product release pressures associated with living medicines, and consider how faster microbial detection approaches may support quality, safety, and timely patient access. Broader themes may include method selection, implementation challenges, regulatory considerations, and the evolving role of rapid testing within advanced-therapy control strategies.

Cell characterization and testing are critical for bioprocess monitoring in biopharmaceuticals and cell-based therapies. Standards are urgently needed to establish common practices, interoperability, and translation in cell-based biotechnologies. Here, we will describe the current standards infrastructure for cell characterization. In addition, we will discuss recent trends in process analytical technologies for cell-therapies, including label-free, real-time monitoring, and metabolomic approaches.

To accelerate cell therapy development and scalability, there is an urgent need to accelerate processing times, increase throughput, reduce cell material requirements for testing, and enhance assay robustness. This presentation will review BlueRock’s approach to achieve this goal: the development of a suite of plate-based assays to characterize our cell therapy products, adaptation to liquid handling platforms, and the development of automated workflows for hands-free operability.

Potency is a critical quality attribute for cell- and gene-therapy products and is essential for product release and licensure. Establishing scientifically sound, mechanism-based, and phase-appropriate potency strategies remains a key consideration through clinical development and commercialization. We present a structured, risk-based approach to potency-assay development and validation to ensure functional consistency, support pivotal clinical trials, and enable successful licensure of cell-therapy products.

As Bayer and BlueRock advance towards Commercial Readiness following the RMAT designation from the FDA, the concept of phase-appropriateness becomes increasingly important. We are transitioning into late-phase development and ensure process comparability, it is essential to ensure that our assays effectively capture the Critical Quality Attributes (CQAs), whether during release or through extended characterization. Collectively, all assays provide a comprehensive view of product quality, allowing us to maintain high standards throughout the development process.

This presentation will showcase a novel single-cell digital droplet PCR (SC-ddPCR) method to characterize an autologous mRNA-modified macrophage therapy for the treatment of end-stage liver disease. Offering single-molecule sensitivity, the assay accurately quantifies transfected subpopulations within the drug products. The workflow’s robustness and precision ensure it is highly effective for routine batch release within a GMP-QC environment, bridging the gap between high-resolution analysis and, manufacturing scalability.

Developing a robust potency strategy for cell therapy products requires a deep, integrated understanding of product characteristics and MoA. By aligning these insights with MoA-driven hypotheses, teams can more effectively identify, prioritize, and refine potency assays that are both scientifically meaningful and phase-appropriate. The session will highlight practical considerations for early-stage programs, including how to balance exploratory analytics with regulatory expectations, how to use characterization data to select surrogate or functional readouts, and how to evolve potency strategies as products advance toward clinical development.