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Session 5a: Case Studies in Gene Therapy Development
Session Chair(s)
Eliana Clark, PHD
Senior Vice President Technical Operations and Quality, Intellia Therapeutics, Inc., United States
Kirsten Messmer, PHD, RAC
Owner, KM Intel LLC, United States
Gene therapies rely on new and innovative technologies aiming to modify, replace, add a missing or malfunctioning gene to treat human disease. Gene therapy products face very product specific CMC challenges due to the novelty and variety of technology employed. This session will discuss case studies for 2-3 different gene therapy technologies.
Learning Objective : At the conclusion of this session, participants should be able to:
- Understand gene therapy specific CMC challenges during product development
- Determine approaches to address the challenges to ensure regulatory compliance
- Form strategies supporting gene therapy development programs
Speaker(s)
Katherine Whitley
Group Lead, Gene Therapy Purification Development, Pfizer, Inc., United States
Speaker
Khandan Baradaran, PHD
Vice President Regulatory CMC, Ultragenyx, United States
Speaker
Michael Chang
Senior Director, Regulatory Affairs CMC, Vertex Pharmaceuticals, United States
Speaker
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