Day Two

• Characterize the unique analytical requirements of trans-amplifying RNA systems, including multi component payload composition, RNA integrity, and encapsulation performance
• Compare targeted and untargeted LNP approaches to understand how delivery strategy influences cargo behavior and analytical requirements
• Develop analytical frameworks that connect cargo quality to potency, comparability, and translational readiness

• Learn how multi-attribute methods can consolidate multiple assays into a single analytical workflow, reducing testing burden while increasing product understanding
• Evaluate the challenges of method robustness, reliability, and validation when transitioning complex methods into GMP environments
• Determine when multi-attribute approaches are appropriate and how to balance efficiency with risk in release and control strategies

• Develop quantitative and qualitative assays to accurately characterize targeted LNP composition and surface modifications
• Assess protein quality, conjugation efficiency, and surface integrity to ensure consistent targeting performance
• Link analytical characterization to biological function to support robust development, comparability, and translational success

• Evaluate total and free RNA assays to identify root causes of variability in EE% measurement
• Identify that nanoparticle-mediated fluorescence attenuation results in free assay imprecision
• Demonstrate that matrix-matched calibration restores EE% accuracy in both LNP and targeted LNP

• Identify the analytical attributes that influence targeting efficiency and in vivo performance of tLNP systems
• Explore characterization strategies to assess surface properties, composition, heterogeneity, and structure–function relationships across CAR-T and SCD programs
• Demonstrate how analytical insights support scale-up, comparability, and translational readiness from discovery through development

Collaborative problem-solving session where participants from companies developing in vivo CAR-T therapies share challenges and brainstorm solutions for T-cell targeting, biodistribution assessment, and potency measurement

• Discuss the toughest characterization challenges facing in vivo CAR-T developers today
• Benchmark analytical strategies for targeted delivery, potency, and product understanding
• Generate practical solutions through facilitated peer discussion and shared experiences

• Measure co-encapsulation efficiency of mRNA and guide RNA, quantify payload ratios, ensure consistent product composition
• Assess stability of multi-component systems, identify degradation pathways for each cargo, develop orthogonal methods
• Correlate analytical profiles to editing efficiency, optimize formulations based on data, accelerate development timelines

• Understand the key challenges limiting T-cell targeting and in vivo delivery
• Explore emerging LNP designs and targeting strategies for improving delivery efficiency
• Learn how next-generation delivery platforms are enabling new gene editing and immunotherapy applications