Improving animal to human translation in advanced therapy programs

Bridging the gap between animal models and human subjects remains a central challenge for cell and gene therapy developers. This discussion addresses limitations in current model systems, strategies to refine dose selection and safety margins, and approaches to integrate mechanistic data for more reliable human predictions.

Key takeaways

• Evaluate model selection criteria that influence translational fidelity and predictive validity
• Define approaches to align nonclinical PK and PD data with human study expectations
• Strengthen dose selection and safety margin frameworks for first-in-human readiness
• Identify gaps in animal models that affect biomarker strategy and clinical interpretation
• Integrate mechanistic insights to improve confidence in human study planning