Issues and Trends in Clinical Development of Cell and Gene Therapies
Register separately for one or more workshops on May 15

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

  • Larissa Lapteva, MD, FDA

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

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