Day Two

Coffee & Tea Served

• Detecting subtle product heterogeneity and post-translational modifications across upstream and downstream process steps
• Leveraging CE readouts to diagnose purification bottlenecks, improve capsid quality during downstream steps, and link analytical findings to process parameters
• Using practical case studies to show when CE outperforms other methods for resolution, turnaround time, and cost, and where hybrid analytical panels are still required

• Discussing opportunities for in-line characterisation as a low-cost, high throughput analytical method
• Establishing a GMP QC assay of percentage empty/partial/full capsids
• Defining fit-for-purpose reporting, through understanding why percentage full readings may be sufficient for regulatory submissions
• Interpreting percentage full and partial capsid data in the absence of defined thresholds, exploring what different distributions may indicate, where uncertainty remains, and how teams are currently contextualising results without clear industry benchmarks

Light snacks and refreshments served

• Explore how analytical developers can protect method intent when transferring into a platform environment
• Diagnose performance changes post-transfer and deciding between retraining, re-optimisation, or full method redevelopment
• How to interpret shifts in potency signal, vector titre, or impurity profiles across sites

• Exploring what “potency assurance” will mean in practice over the next five years, moving from individual assays toward integrated strategies that combine starting material control, process understanding, and functional readouts
• Debating the future role of platform analytics versus product-specific methods
• Exploring where digital workflows, standardised data pipelines, and analytical platforms can realistically reduce turnaround time without overselling AI or full end-to-end automation

Across gene therapy, companies are being forced to fundamentally rethink QC, validation and analytical strategies under unprecedented cost pressure, while still preparing for late-stage development, MAA/BLA submissions and inspections.

This session uses a hypothetical gene therapy company transitioning from Phase II into Phase III to explore:

• How far analytical and QC innovation can go without triggering regulatory pushback
• Where regulators are genuinely open to new statistical and efficiency-driven approaches
• Where traditional expectations still apply and why

• Navigating evolving global regulatory frameworks, differences in CMC, and requirements across regions
• Discussing ongoing initiatives and collaborations aimed at standardising CMC standards
• Strategies for overcoming them to ensure smoother global product development

• Understanding emerging regulatory frameworks for genome editing products and their unique analytical burdens
• Reviewing regulatory priorities and recent developments across ATMPs
• Exploring the latest advances in regulatory thinking for rare and ultra-rare disease therapies, including how risk-benefit, evidentiary expectations and analytical robustness are assessed when patient populations are small