Casey Maguire, PhD, on Potential Advantages of Encapsulated AAVs

Commentary
Video

The associate professor and associate investigator of neurology at Harvard Medical School discussed research confirming proof-of-concept with EV-AAVs.

“We focused on the capsids inside the lumen and found that we could actually achieve transduction with them. So those are functional. It's been a long question that we actually were able to address. We're really happy about that. [Before now] we didn’t actually know, if we got more of that capsid packaged inside the vesicles, will that make a better gene delivery product? And that evidence suggests that yes, that would be a good strategy.”

Associated adeno-associated virus (AAV) vectors face challenges with safety and delivery of gene therapies. Combining AAV technology with viral-like particles (VLPs) may yield a product more efficacious than VLPs alone and safer than AAVs alone.

Casey Maguire, PhD, associate professor of neurology, Harvard Medical School, and associate investigator, Massachusetts General Hospital, presented research on EV-AAVs from his lab at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland.

CGTLive® spoke with Maguire to learn more about EV-AAVs and research ongoing with them. Maguire discussed his startup company revolving around EV-AAVs, Sphere Gene Therapeutics, and some collaborative research that may point to the best uses of this technology, including showing benefits in liver-targeted therapies compared to traditional AAVs. He also discussed looking deeper into intraluminal AAV packing with the EV-AAVs and confirming the efficacy of the encapsulated AAVs.

Click here to read more coverage of the 2024 ASGCT Annual Meeting.

REFERENCE
Cheng M, de la Cruz D, Ng C, et al. New Biological Insights into Extracellular Vesicle Associated AAV Help Improve Vector Yield and Provides Rational for Intraluminal AAV Packing. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract 221.
Recent Videos
Mark Hamilton, MD, PhD, a hematology-oncology and bone marrow transplant (BMT) cell therapy fellow at Stanford University
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Sarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program in the Division of Hematology/Oncology at David Geffen School of Medicine at University of California, Los Angeles (UCLA)
David Porter, MD, the director of cell therapy and transplant at Penn Medicine
David Porter, MD, the director of cell therapy and transplant at Penn Medicine
Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Related Content
© 2025 MJH Life Sciences

All rights reserved.