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In June, the Foundation held a workshop to assess the state of the field and develop a new roadmap for using focused ultrasound to treat Alzheimer’s disease. 

Key Points The American Society of Gene & Cell Therapy meeting was held May 16–20 in Los Angeles and virtually.  Leaders in the focused ultrasound field presented their work, and several scientific abstracts may be of interest to the community. Frédéric Padilla, PhD, the Foundation’s research director for applied physics, attended the meeting.  The 26th annual meeting of the American Society of Gene & Cell Therapy (ASGCT) was held May 16–20, 2023, in Los Angeles, CA, and virtually. With approximately 8,000 attendees, ASGCT is touted as the premier event for professionals in gene and cell therapy to learn about the latest scientific research and new technologies. Beyond the educational offerings and keynote addresses, ASGCT provides networking opportunities through its workshops, career fair, luncheons, and receptions. The conference is attended by academic researchers, clinicians, bio-industry development, regulatory agencies, equipment manufacturers, patient advocates, and others.  Frédéric Padilla, PhD, the Foundation’s research director for applied physics, attended the meeting and said, “Among the hot topics this year were capsid design and brain penetrating capsids, CRISPR gene editing, and CAR-T cells for the treatment of diffuse intrinsic pontine gliomas.” He added that he made several new contacts within the pharmaceutical industry as well as with academic and clinical researchers.  During the scientific symposium, leaders in the field of focused ultrasound presented oral summaries of their gene therapy work. On May 17, Isabelle Aubert, PhD, a senior scientist at Sunnybrook Research Institute, professor of Laboratory Medicine and Pathobiology at the University of Toronto, and the Tier 1 Canada Research Chair in Brain Repair and Regeneration, presented “Delivery of AAVs to the Central Nervous System Using MRI-Guided Focused Ultrasound and Immunological Considerations.” On May 19, Katherine Ferrara, PhD, a professor of radiology and the division chief for the molecular imaging program at Stanford University, presented “AAV In Vivo Tracking and Microscopy.”  “Dr. Aubert and Dr. Ferrara delivered exceptional presentations,” said Dr. Padilla. “Dr. Aubert underscored the enhanced therapeutic efficacy achieved by using focused ultrasound to deliver AAV-based gene therapy to the brain, resulting in improved therapeutic efficacy in various preclinical models, and showing reductions in side effects at untargeted sites. Meanwhile, Dr. Ferrara introduced cutting-edge imaging techniques to trace the distribution of gene therapy vectors and address intricate concerns related to off-targeting and transduction. He added, “Last year, the Foundation started a specialized program on gene and cell therapies. It was invigorating to witness the enthusiasm of the ASGCT community toward this technology.” Beyond the symposium presentations, six scientific abstracts (including two oral presentations) were also about the use of focused ultrasound in gene therapy and will be of interest to the focused ultrasound community. Click on each title below to see the full text of the abstract. 25. Focused Ultrasound for Improved Delivery of AAV Vectors to the Brain for the Treatment of Huntington’s Disease by Nick Todd, Yongzhi Zhang, Lilyan Garrett, Alvin Yao, Kai Shing, and Kimberly Kegel-Gleason from Brigham and Women’s Hospital, Boston, MA; Northeastern University, Boston, MA; Harvard University, Boston, MA; and Massachusetts General Hospital, Boston, MA (Oral Abstract Session)  31. NHEJ Gene Editing of Hemophilia A Mice Show Therapeutic Levels of FVIII Following Ultrasound-Mediated Gene Delivery of CRISPR/Cas9 Plasmid by Savannah M. Lawton, Meng-Ni Fan, Ting-Yen Chao, Chun-Yu Chen, Ellen Xu, Xiaohe Cai, and Carol H. Miao from Seattle Children’s Research Institute, Seattle, WA, and Seattle Children’s Research Institute, Seattle, WA (Oral Abstract Session)  565. Use of Low-Intensity Transcranial Focused Ultrasound for Targeted Delivery of Exosomes to the Brain by Jacqueline Lara, Jon Haroon, Linda Flores, Marisa McDonald, Kennedy Mahdavi, Victoria Venkatraman, Hoi Wa Ngai, Samantha Schafer, Mark Schafer, Mohamed Hammad, Isaac Nardi, Taylor Kuhn, Duncan Ross, Sheldon Jordan, and Karen S. Aboody from Beckman Research Institute of the City of Hope, Duarte, CA; The Regenesis Project, Santa Monica, CA; Drexel University, Philadelphia, PA; Kimera Labs Inc., Miramar, FL; University of California Los Angeles, Los Angeles, CA; and Neurosomes Bio, Miramar, FL (Poster Session)  875. Focused Ultrasound–Mediated Intranasal Delivery of AAV to Targeted Brain Regions with Minimal Systemic Exposure by Dezhuang Ye, Jinyun Yuan, Yaoheng Yang, Yimei Yue, Zhongtao Hu, Siaka Fadera, and Hong Chen from Biomedical Engineering, Washington University in Saint Louis, Saint Louis, MO (Poster Session)  1301. Non-Viral Gene Therapy Utilizing Transcutaneous Ultrasound-Mediated Gene Delivery by Barry R. Campbell, Surabi Rao, Bert Frederich, Adel Alonso, Brit Ventura, David Kim, Katie Benthall, Brian Christie, Julia Poniatowski, Ivan Krivega, Ken Greenberg, and Steve Feinstein from Platform Technology, SonoThera, South San Francisco, CA; Gene Therapy, SonoThera, South San Francisco, CA; and SonoThera, South San Francisco, CA (Poster Session)  1381. Increasing Feasibility and Safety of Transcutaneous Ultrasound–Mediated Gene Delivery via Intravenous Injection of Plasmids and Microbubbles by Ting-Yen Chao and Carol H. Miao from Seattle Children’s Research Institute, Seattle, WA, and the Department of Pediatrics, University of Washington, Seattle, WA (Poster Session)  Content from the 26th Annual Meeting will be available to ASGCT members in late 2023.  See the Meeting Website 

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