Nicole Paulk, PhD
Assistant Adjunct Professor
UC, San Francisco
Federico Mingozzi, PhD
Chief Scientific Officer
Julianna LeMieux, PhD
Senior Science Writer
Senior Business Writer
Originally Aired: August 12, 2020
Time: 3:00 pm ET
Gene therapy has undergone a renaissance in recent years, with several drugs offering beneficial, even life-saving treatments, now approved by the FDA and EMA. A big reason for this turnaround is the emergence of adeno-associated virus (AAV) as a generally safe and efficient vector for therapeutic gene delivery.
Earlier this summer, reports emerged of two fatalities in young boys in a clinical trial for a rare muscle disorder using AAV. This sad news has turned the spotlight on viral dosage, vector manufacturing and immune responses as factors we need to better understand to ensure the safety of gene therapy treatments in the future.
On Episode 3 of GEN Live, we discuss the state of gene therapy, the rise of AAV, and the safety issues that need to be addressed going forward.
Attendees were able to ask questions live or via chat/social media.
• Nicole Paulk (UCSF)
• Federico Mingozzi (CSO, Spark Therapeutics)
Produced with support from: