Michael White

As gene therapy programs advance toward pivotal development and approval, analytical leaders must balance scientific rigor with practical development realities. This workshop will explore how to build fit-for-purpose analytical packages, establish meaningful potency strategies, and generate the evidence needed to support regulatory decision-making while minimizing unnecessary complexity.

This session provides a platform to delve into:

• Identifying the critical assays needed to characterize product quality, support regulatory submissions, and inform development decisions.
• Determining how and when to evolve potency approaches from early development through approval.
• Demonstrating target engagement and biological activity when clinical datasets are limited.
• Developing analytical strategies that support current milestones while reducing rework later in development.