Closing Panel, • 9/22/25 Closing Panel: Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials You Might Also Like How To Prevent Failure in Rare Disease Trials? Beyond the Feasibility Survey: Strategic Site Selection to Accelerate Trials and Optimize Resources Case Studies in Rare Disease Commercialization: Lessons Learned Designing Trials with the End in Mind: Building Market Access into Rare Disease Clinical Development Purpose-Driven Metrics: Running Gene Therapy Trials with KPIs That Matter
Closing Panel, • 9/22/25 Closing Panel: Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials You Might Also Like How To Prevent Failure in Rare Disease Trials? Beyond the Feasibility Survey: Strategic Site Selection to Accelerate Trials and Optimize Resources Case Studies in Rare Disease Commercialization: Lessons Learned Designing Trials with the End in Mind: Building Market Access into Rare Disease Clinical Development Purpose-Driven Metrics: Running Gene Therapy Trials with KPIs That Matter
