Nathan Yozwiak, PhD, on Researching AAV Gene Therapy Delivery to the Brain

“Gene therapy is enormously promising for a lot of neurological indications. For many of them, the genetics are well known, you know what you'd want to change, if you could, but now [to figure out] delivery. How do you get those genes into that cell, make that edit, make that correction, that’s what really hangs over the field. And if those technologies can be unlocked, there are many diseases and thousands of patients that could potentially be treated.”

A number of researchers from Mass General Brigham (MGB) presented work at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland. A good amount of this work centered on new technologies and new methods aiming to improve gene and cell therapy and its delivery to different tissues, particularly the brain.

CGTLive® spoke with Nathan Yozwiak, PhD, head of research, Cell and Gene Therapy Institute, MGB, to learn more about research presented at the ASGCT meeting coming out of the center. He highlighted 2 presentations, one given by Casey Maguire, PhD, on encapsulated adeno-associated virus (AAV) vectors and another given by Nick Todd, PhD, on ultrasound-mediated delivery of AAV. Both presentations have potential in achieving gene therapy delivery past the blood-brain barrier for treating neurologic indications.

REFERENCES

1. 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.
2. Owusu-Yaw B, Zhang Y, Garrett L, et al. Focused Ultrasound-Mediated Delivery of AAV9-Based Gene Therapy in Aged Huntington’s Disease Mice. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract 623