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.

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.

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.

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.

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 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).