Gene therapy research and studies, like that of many new treatments, do not always produce outcomes that are beneficial or successful. This session will explore some of the areas where treatments can get stopped while en route to an approval. We'll have a panel discussion with perspectives from regulatory, industry, clinical, and preclinical research. We'll also discuss failures on the commercial side and possible program halts, regulatory holds, safety and patient enrollment issues, and off-target effects in a pre-clinical setting related to gene therapies.
Introduction
12-12:05 p.m.
Moderator: Emily McGinnis, MPH, chief patient officer and head of government affairs at Taysha Gene Therapies
Panel Discussion
12:05-12:45 p.m.
Speakers:
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Kimberly Goodspeed, MD, assistant professor of pediatrics, division of neurology, UT Southwestern Medical Center
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Jennifer Helfer, PhD, senior director, patient advocacy & engagement, corporate affairs at Viridian Therapeutics
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Tejashri Purohit-Sheth, MD, FACAAI, CQIA, director, Division of Clinical Evaluation and Pharmacology/Toxicology, Office of Tissues and Advanced Therapies Center for Biologics Evaluation and Research, U.S. FDA
- Sandhya Sanduja, PhD, acting branch chief, Office of Pharmacology/Toxicology, U.S. FDA
Q&A Session
12:45-1 p.m.
Have questions for our FDA speakers? Send them to ASGCT Patient Outreach Manager Lexi Starosta. Please note: There will be no live chat during this event.
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