Day One

Coffee & Tea Served

• Exploring why some teams still run ddPCR, ELISA, AUC, HPLC and TEM and how to reduce redundancy
• Discussing opportunities for how early teams can converge on a smaller assay panel
• Is there a one-size-fits-all, and how close are we to establishing one?

• Outlining how early TCID₅₀ and mRNA assays were used to establish baseline infectivity and expression while planning for functional relevance as development progresses
• Developing an assay that assessed protein expression of the gene of interest, that better reflects biological effect
• Discussing planned assay modifications to enable increased robustness, lifecycle management, and eventual transition towards a bioactivity-focused potency assay for later-phase development

• Understanding vector design, transgene sequence and delivery target in the aspect of potency assay design
• Protein expression vs gene expression vs dose-response cell-based potency assays: what is needed for AAV vector characterisation/release and why?
• Development of in vivo potency assay versus in vitro potency assay for AAV vectors: at what stages of product development is in vitro testing desirable, and can one replace the other?
• Importance of selecting the product specific cell line and developing optimised, reproducible transductions in moving towards a consistent product specific potency method

Put a face to a name. This session session is the perfect opportunity to connect with KOLs, leading companies, and innovative researchers in the field of gene therapy analytics. Establish meaningful connections to build upon for the rest of the conference and gain individual insight beyond the papers and press releases into the pioneering research and technique applications.

Light snacks and refreshments will be served.

• Designing functional potency assays that reflect true mode of action, using enzyme-based or activity-driven endpoints embedded within cell-based systems
• Implementing layered control strategies to manage variability across complex cell-based potency assays to enable qualification, validation, and long-term assay stability
• Reviewing strategies for managing assay life cycle

• What is the true marker of potency for your product type?
• Should expression or function matter more? And which has been more reliable?
• Is the data interpretation from these assays accurate?

10-minute presentation followed by 30-minute roundtable discussion

• Case example of where identity looked near ‘perfect’ but was not indicative of strong potency
• Limitations of long-read sequencing and how they affect QC readiness
• Why NGS results frequently disagree with AUC, ddPCR, and functional potency assays
• How emerging automation/algorithms are improving reproducibility

Connect with peers in a relaxed atmosphere and continue to forge new and existing relationships while exploring the latest advancements in gene therapy analytical development.

To submit a poster, please contact info@hansonwade.com

• Pinpointing where HCPs, residual DNA, and process-related contaminants accumulate across upstream, downstream, and final formulation to give teams a proactive risk profile
• Evaluating when LC-MS, DIA-MS, ddPCR and immunoassays complement each other to ensure no single assay misses critical contaminants
• Linking impurity patterns to potency drift, vector quality, and safety signals

• Detecting deamination, oxidation, backbone cleavage and other chemical degradations that compromise safety even when identity appears intact
• Using HPLC-UV, LC-MS and mass photometry to identify early degradation signatures before potency drops
• Correlating degradation signatures with functional potency and clinical behaviour
• Understanding how chemical instability drives immunogenicity or unexpected clinical reactions