Nicole Paulk, PhD
Assistant Adjunct Professor
UC, San Francisco

Federico Mingozzi

Federico Mingozzi, PhD
Chief Scientific Officer
Spark Therapeutics

Julianna LeMieux

Julianna LeMieux, PhD
Senior Science Writer

Alex Philippidis

Alex Philippidis
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.

• Julianna LeMieux – GEN Senior Science Writer
• Alex Philippidis – GEN Senior Business Writer

Special Guests:
Nicole Paulk (UCSF)
Federico Mingozzi (CSO, Spark Therapeutics)

Moderated by: Kevin Davies (GEN/The CRISPR Journal)


Produced with support from:

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