Outlining the Journey from Adult Approval to Pediatric Approval

11:15 am - Thursday 26th March 2026

  • 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

Speakers:

Associate Director - Gene Therapy
CSL