UPDATE: The proceedings from this meeting have now been summarized in a white paper.
Key Points
- A 1.5-day workshop was held this week to discuss challenges, opportunities, and the role of focused ultrasound in cell and gene therapy.
- A white paper summarizing the event is now available.
On October 27–28, 2025, the Foundation hosted a workshop on the role of focused ultrasound for cell and gene therapy. Nearly 50 in-person attendees convened at the University of Virginia Darden School of Business.
The conversation centered on gene therapy for neurodegenerative diseases and large-organ applications and on cell therapies for brain tumors with the goal of fostering cross-pollination across these domains.
Focused ultrasound device manufacturers Insightec and NaviFUS sponsored the meeting.
The program included presentations, flash talks, breakout sessions, and panel discussions.
Day 1 presentations covered the state of the field for using cell and gene therapy to treat diseases of the brain and large organs. The following speakers and topics were included:
- Focused Ultrasound Foundation Perspective: A Framework for Program Selection by Alec Batts
- Large Organ Gene Therapy: Challenges, Opportunities, and Recent Focused Ultrasound Data by Jonathan Lindner
- Focused Ultrasound for Brain Gene Therapy: Challenges, Opportunities, and Recent Focused Ultrasound Data by Elisa Konofagou
- Thermogenetic Control of CAR T Cells to Potentiate Brain Tumor Therapy by Lena Gamboa
- AAV Capsid Engineering for Liver Detargeting and Redirecting Cellular and Tissue Tropism by Jared Smith
- Focused Ultrasound–Mediated Gene Replacement Therapy and Gene Editing for the Treatment of Hemophilia by Carol Miao
- Dissecting Routes to the Brain: CSF Administration vs. Systemic AAV Delivery in NHPs by Michal Fortuna
- Cell Therapy of Brain Tumors by Catherine Bollard
- Routes of Administration & Platform Approach for Gene Therapy by Bryan Pukenas
The flash talks on Day 1 were:
- Delivery of ASO for a Rare Pediatric Neurodegenerative Disease by Laura Owlett
- Noninvasive Focal Gene Transfer of Chemogenetic Proteins in the Primate Brain by David Schaeffer
- Turning Resistance into Remission: Ultrasound-Enhanced CAR T Therapy for Childhood Brain Cancer by Dalia Haydar
The four presentations on Day 2 included:
- Novel Non-Viral DNA Delivery System by Jeff Bartlett
- Focused Ultrasound–Mediated Delivery of Non-Viral Gene Editing and Gene Therapy in the Brain by Rich Price
- Regulatory Development in Gene and Cell Therapy by Michael Werner
- Current Clinical Trials and Vector Usage in Gene Therapy by Michael Lehmicke
There were two panel discussions on Day 2:
- What are the Regulatory Pathways for Focused Ultrasound Cell and Gene Therapy? Panelists: Jon Lindner, Bryan Pukenas, Jared Smith, and Michael Werner
- How to select a Program for Focused Ultrasound Plus Cell and Gene Therapy? Panelists: Joe Bruder, Jennifer Johnston, Michael Kaplitt, and Bob Smith
“Building on our inaugural 2023 workshop and our Cell Therapy for Brain Tumors Roundtable earlier this year, this meeting brought together a diverse group of stakeholders, from focused ultrasound technology developers and scientists to clinicians with deep expertise in gene and cell therapy,” said Frédéric Padilla, PhD, director of the Foundation’s Gene and Cell Therapy Program. “We appreciated the active, candid exchange, with additional perspective from the Alliance for Regenerative Medicine that helped extend the conversation beyond the bench toward translation. We will aim to continue funding transformational research to move focused ultrasound–mediated gene and cell therapy delivery, immunomodulation, and sonogenetics into clinical trials, in line with our mission to expedite patient access to innovative treatments.”
Dr. Padilla also extended special thanks to the Foundation’s Gene and Cell Therapy Program Scientific Advisory Board members: Isabelle Aubert, PhD, Catherine Bollard, MD, Elisa Konofagou, PhD, Jonathan Lindner, MD, Mikhail Shapiro, PhD, Bob Smith, Ramasamy Paulmurugan, PhD, and Michael Werner, for assistance in planning the workshop.