Clinical Research: Phase 1 - Phase 4

Achieving the Promise of Gene Therapy: New Pathways to Overcome Patient Enrollment and Safety Challenges

Meeting shifting operational and regulatory demands in precision medicine trials

This webinar examines the evolving science of gene therapy, covering current approaches such as gene transfer and gene silencing, safety considerations, and delayed adverse events. Learn about the current regulatory landscape, identify opportunities to more closely engage with regulators, and more.

Topics in this presentation include:

  • Understanding the disease burden and patients’ treatment goals
  • Designing studies that identify relevant endpoints and meet regulatory and safety requirements
  • Discussing unique case studies that address operational challenges in gene therapy trials

Presented by:
Allan Valmonte, MBA, Executive Director, Program Strategy – Rare Disease & Pediatrics, Premier Research
Mesaki Kenneth “Ken” Ndugga-Kabuye, MD, FACMG, Medical Director, Rare Disease/Pediatrics, Premier Research
Naomi Kautz, Director, Regulatory Affairs, Regulatory Professionals