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.