Safety Concerns With Gene Therapies: Barry Byrne, MD, PhD

“The field is very focused right now on some of the early safety events because of adverse events in some studies. We think we've learned some important mechanistic underpinnings of those safety events and are hoping that we can employ strategies that block those aspects of systemic gene transfer that may be the cause of safety problems in gene therapy. We need these therapies to be safe and effective because of the need to have these types of therapies available to the broad community.”

Gene therapies are being investigated as potential treatments for many previously incurable diseases, including Pompe disease, but their development is not without its challenges. As more therapies are evaluated, serious adverse events (AEs) that arise from gene therapies are gaining attention. The FDA Cellular, Tissue, and Gene Therapies Advisory Committee met last month to address the unmet need of AEs in gene therapies.

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 associated with gene therapies, including dose limiting toxicities and the need to mitigate adverse events to advance the availability of gene therapies.

REFERENCE

Spark Therapeutics announces first participant dosed in phase 1/2 study of investigational gene therapy for late-onset Pompe disease. News release. Spark Therapeutics. February 1, 2021. https://sparktx.com/press_releases/first-patient-dosed-pompe/