Navigating early clinical milestones in cell and gene therapy programs

Early clinical development in cell and gene therapy requires careful planning to ensure safety, translational fidelity, and clear decision points. This spotlight session reviews how teams can structure first-in-human and early phase studies, manage dose escalation, and use emerging PK and PD signals to guide next steps.

Key takeaways

• Establish criteria for early phase study design in cell and gene therapy
• Guide dose escalation strategies that balance safety margins and translational read-through
• Interpret initial biomarker, PK, and PD data to refine clinical decision making
• Align early milestones with manufacturing, regulatory, and program readiness needs
• Apply frameworks that support predictive validity for human subjects