The director of the Powell Gene Therapy Center at the University of Florida discussed the recent meeting of the FDA Cellular, Tissue, and Gene Therapies Advisory Committee.
“A very significant problem that we've focused a lot of attention on, because of our interest in the immune responses to AAV vectors of both the capsid proteins and the transgene itself, is thrombotic microangiopathy... which contributes to safety events in many studies now. We know some mechanistic underpinnings of that issue and have strategies to monitor early laboratory values that help guide clinicians to manage those events, understand the clinical significance, and potentially prevent those events to improve on the safety of the treatment paradigm.”
While gene therapies represent a revolutionary step forward in the potential treatment of many previously incurable diseases, including Pompe disease, the therapies have come with a host of challenges. Namely, serious adverse events (AEs) associated with these therapies are garnering more attention and the mitigation of these AEs represent an unmet need in this emerging field. To address these concerns and identify steps forward, the FDA Cellular, Tissue, and Gene Therapies Advisory Committee met last month to discuss these topics.
GeneTherapyLive spoke with Barry Byrne, MD, PhD, director of the University of Florida’s Powell Center for Gene Therapy and advisory committee member, to learn more about adverse events typically observed with gene therapies and takeaways from the FDA meeting.