Tackling trial design challenges in advanced therapies

Designing clinical trials for cell and gene therapies requires careful consideration of safety, biomarkers, and translational predictivity. This session explores approaches to structure studies that meet regulatory expectations, inform dose selection, and maximize predictive validity for human subjects.

Key takeaways

• Define frameworks for first-in-human and early phase trial design
• Optimize dose selection, escalation, and safety margin evaluation
• Integrate biomarker strategies and PK/PD readouts to guide decisions
• Address challenges in cohort diversity and translational fidelity