Karsten Peppel
Associate Director - Gene Therapy CSL
Seminars
Thursday 26th March 2026
Outlining the Journey from Adult Approval to Pediatric Approval
2:00 pm
- Designing preclinical studies that demonstrate efficacy and durability in young animal models
- Building the regulatory and clinical evidence needed to progress safely and confidently from adult to younger patient populations
- Understanding challenges including toxicity, immunology, and redosing capabilities
