Janet Glassford

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

• 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