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