Key Points
- Last November, 75 experts presented research and deliberated issues related to focused ultrasound and gene and cell therapy for neurodegenerative disorders.
- The summary white paper, which includes four immediate action items, is now available.
The Foundation held its first workshop to discuss focused ultrasound and gene and cell therapy for neurodegenerative diseases in November 2023. More than 75 experts from academia, industry, government, and the not-for-profit sector attended in person in Washington, DC.
Each presentation and the lively discussion throughout the workshop have now been summarized in a white paper. The two-day program featured 26 speakers and panelists. Presentations covered diverse topics ranging from overviews of gene therapy, disease-specific applications, preclinical advances, manufacturing challenges, and regulatory considerations for translation.
Attendees discussed the following six burning questions:
- What are the needs and gaps in gene delivery or gene therapy that focused ultrasound can address?
- What are the diseases in which there is the greatest need and opportunity for less invasive gene delivery that could potentially be provided with focused ultrasound?
- Which vectors will benefit most from combination with focused ultrasound?
- How can we validate the delivery and effectiveness of gene or cell therapy to the target site?
- What obstacles must be overcome to progress to clinical trials?
- What types of partnerships should be pursued to facilitate transition to clinical trials?
Discussions emphasized that focused ultrasound offers a platform to noninvasively increase delivery efficiency of both viral and non-viral gene therapy vectors. Combining these technologies could expand therapeutic reach to larger tissue volumes over direct injections and provide more uniform distribution while requiring lower vector doses that could improve safety and manufacturing feasibility. Researchers presented data demonstrating feasibility to target diverse brain regions and cell types without overt toxicity.
Participants also raised important questions surrounding mechanisms, acoustic parameter optimization, routes of delivery, and anatomical factors.
The community advanced four action items for moving forward:
- Hold follow-up meetings to continue driving development of focused ultrasound–enhanced gene therapy, foster collaborations, and develop best practices for the field.
- Create shared resources, reference materials, and testing pipelines.
- Promote future collaborations by connecting focused ultrasound experts with gene therapy researchers.
- Explore how the Foundation can enable further technology evaluation through funding or other mechanisms.
“Our overarching mission at the Foundation is to expedite patient access to innovative focused ultrasound treatments,” said Frédéric Padilla, PhD, the Foundation’s director of the Gene and Cell Therapy Program. “This first workshop was a pivotal step toward propelling focused ultrasound–mediated gene therapy into clinical trials.”
Join the Conversation
The Foundation has established a public LinkedIn group as a platform for exchanging information and fostering discussions about focused ultrasound for gene and cell therapy. We aim to make this a hub for sharing the latest news, publications, conference updates, and more. Join the “Focused Ultrasound for Gene and Cell Therapy” LinkedIn Group