Global Clinical Program Design for First-in-Human Gene Therapy

Situation

• Clinical development strategy that could safely and credibly reach the target pediatric population despite regulatory constraints.
• FDA initially did not permit a pediatric trial, requiring an alternative pathway to progress development.

Approach

• Designed E2E first-in-human program with age-descending strategy.
• Established the initial development pathway through Health Canada with enrollment descending from 24+ to 18–24 followed by MHRA acceptance of further age descent from 18 to 15.
• Structured the overall program to enable progressive expansion into the intended pediatric population while maintaining clinical rigor and regulatory credibility.

Outcome

• Created a viable global clinical development pathway for a first-in-class gene therapy.
• Enabled staged progression from adults to adolescents as part of a broader age-descending strategy.
• Positioned the program to reach the target pediatric population through a structured, regulator-informed development plan.