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

Broadcast Date: 
  • Time: 

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 select 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 select UCSF)
• Federico Mingozzi select CSO, Spark Therapeutics)

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

Produced with support from:

PerkinElmer logo