Issues and Trends in Clinical Development of Cell and Gene Therapies
All times below listed in ET
Planned by the Regulatory Affairs Committee.
Co-chairs: Daniela Drago, PhD, Aurion Biotech and Kaye Spratt, PhD, BridgeBio Gene Therapy
Register for Issues and Trends in Clinical Development of Cell and Gene Therapies
Session 1 – Baby Steps: Cell and Gene Therapy Clinical Trials in Pediatric Populations
8:00 – 9:00 a.m.
Improving Clinical Pediatric R&D For Cell and Gene Therapies
Brian Tseng, MD
Regulatory Perspective on Pediatric Studies
Elizabeth Hart, MD, Food and Drug Administration
Panel Q&A moderated by Daniela Drago, PhD, Aurion Biotech
Session 2 – When Things Go Wrong - Effectively Managing Adverse Events During Product Development
9:00 – 10:20 a.m.
Managing Unexpected Adverse Events in Global Clinical Trials
Bernhardt Zeiher, MD, Astellas Pharma
Regulatory Considerations and Developmental Impact of Unexpected Adverse Events: DMD Experience
Natalie Schmidt, Pfizer
Panel Q&A moderated by Kaye Spratt, PhD, BridgeBio Gene Therapy
Break: 10:00 – 10:30 a.m.
Session 3 – Innovation in Clinical Trial Design for Cell and Gene Therapies: What’s on the Horizon?
10:30 a.m. – 12:00 p.m.
Managing the Uncertainties of Utilizing Novel Trial Designs in Hemophilia
Alex Kuta, PhD, UniQure
Developing Innovative Clinical Trials for Rare Diseases: Danon Disease Experience
Jonathan Schwartz, MD, Rocket Pharmaceuticals
Regulatory Considerations for Novel Clinical Trial Designs
Larissa Lapteva, MD, FDA
Panel Q&A moderated by Snehal Naik, PhD, Spark Therapeutics