Key Points
- The American Society of Gene and Cell Therapy meeting was held May 13–17 in New Orleans.
- The New York Times wrote a feature article to highlight the presented research that cured a rare disease in a baby.
ASCGT’s 28th Annual Meeting, held May 13–17, 2025, in New Orleans, Louisiana, brought together several thousand researchers, clinicians, and industry leaders to present and discuss the latest advances in gene and cell therapy.
The conference showcased major strides in translational science, with growing attention to both rare diseases and individual genetic conditions—as highlighted in The New York Times on the day the study was presented at ASGCT—for its transformative potential in medicine. Clinical trials involving in vivo gene editing were also featured, underscoring the field’s rapid move toward precision therapeutics.
The Foundation’s partnership with ASGCT was recognized during the Presidential Address, and Sarah Fong, PhD, a postdoctoral scholar at the University of California–San Francisco, was honored during the Outstanding New Investigator Symposium as a recipient of the ASGCT Career Development Award, which was funded by the Foundation.
The director of the Foundation’s Gene Therapy Program, Frédéric Padilla, PhD, and Stefan Radtke, PhD, senior staff scientist at Fred Hutchison Cancer Center, co-chaired a scientific session on “Improved Therapeutic Delivery of Nanoparticles,” which was organized by ASCGT’s Nonviral Therapeutic Delivery Committee. Richard Price, PhD, professor of biomedical engineering at the University of Virginia, presented “Focused Ultrasound Delivery of Non-Viral Gene Therapies to the Blood-Tumor Barrier…and Beyond.”
Notably, Gabriel Kwong, PhD, professor of biomedical engineering at Georgia Institute of Technology, presented his Focused Ultrasound Foundation–funded work performed in collaboration with Costas Arvanitis, PhD (also from Georgia Tech), “Sonothermogenetic Control of CAR-T Cells for Brain Tumor Immunotherapy,” in a session on Current Advancements in the Development of Cell Therapy for Cancer.
“It was a superb meeting, with several exciting presentations on focused ultrasound—both for enhancing the delivery of gene therapies and for sonogenetic control of gene expression—coming not only from academia but also from private companies,” said Dr. Padilla. “I believe that the prospect of a first-in-human clinical trial of focused ultrasound–mediated gene therapy delivery is coming closer and closer. It is great to see investigators we are currently supporting, or have supported in the past, being highlighted at this conference. We are excited to continue advancing the field to make gene therapies accessible for both rare and more prevalent diseases.”
The following oral presentations, abstracts, and posters may be of interest to the focused ultrasound community:
- The scientific symposium on Improved Therapeutic Delivery of Nanoparticles included “Focused Ultrasound Delivery of Non-Viral Gene Therapies to the Blood-Tumor Barrier…and Beyond,” a presentation by Richard Price, PhD, professor of biomedical engineering at the University of Virginia.
- The Current Advancements in the Development of Cell Therapy for Cancer Workshop included “Sonothermogenetic Control of CAR T Cells for Brain Tumor Immunotherapy,” a presentation by Gabriel Kwong, PhD, from the Georgia Institute of Technology and Emory University.*
- Abstract 48: Optimizing Focused Ultrasound Parameters for Enhanced AAV Delivery Across the Blood-Brain Barrier for the Treatment of Neurodegenerative Diseases by Bernie Owusu-Yaw, PhD, from Brigham and Women’s Hospital/Harvard Medical School.
- Abstract 49: Non-Viral Transcutaneous Fluoroscopy Guided Ultrasound-Mediated FVIII Delivery as a Promising Therapeutic Approach for Hemophilia A Treatment by Ting-Yen Chao from Seattle Children’s Research Institute.
- Abstract 106: Development of a Novel Non-Viral Genetic Therapy for Hemophilia A Utilizing Durable, Redosable, and Titratable Approach of Ultrasound-Mediated Delivery of an Oversized Episomal hFVIII DNA Vector by Ivan Krivega from SonoThera.
- Abstract 126: Focused Ultrasound Enhances Deep Brain Gene Delivery via Intra-CSF AAV Administration in Rodents and Non-Human Primates by Bradford Elmer, PhD, from Sanofi.
- Poster 543: Investigating Focused Ultrasound Enhancement of CSF-Delivered AAV to the Brain by Nicholas Slimmon from UT Southwestern Medical Center.
- Poster 626: Achieving Therapeutic Endogenous FVIII Levels in Hemophilia A Mice Using Ultrasound-Mediated Gene Delivery of CRISPR/Cas9 Plasmids by Ting-Yen Chao from Seattle Children’s Research Institute.
- Poster 1436: Focused ultrasound-mediated delivery of a novel engineered AAV capsid to the rat and non-human primate brain using a human clinical FUS system by Nick Todd from Brigham and Women’s Hospital.
- Poster 1530: Evaluating Behavioral Outcomes of Whole-Brain AAV9 Gene Therapy for Fragile X Syndrome via Focused Ultrasound and BBB Opening by Anthony Delalande, PhD, from the University of Orléans.
- Poster 1577: Non-Viral Genetic Medicine for Targeted Delivery of Full-Length Dystrophin to Skeletal, Cardiac, and Diaphragm Muscles in DMD Mouse Models and Non-Human Primates by Ivan Krivega from SonoThera.
- Poster 1683: MRI-Guided Focused Ultrasound: A New Drug Delivery Modality Addressing the Blood Brain Barrier by Michael V. Templin, from Charles River – Reno.
- Poster 1684: Scalable Softporation™ platform for intracellular delivery to primary natural killer (NK) cells by Kourtney Kostecki from OpenCell Technologies.
- Poster 1921: Focused ultrasounds enhance efficacy of AAV transduction via intracerebroventricular administration in mice by Ken Inoue, MD, PhD, from the National Center of Neurology and Psychiatry.
Search ASCGT’s Interactive Abstract Tool
*Research funded by the Focused Ultrasound Foundation