Inaugural Focused Ultrasound Gene and Cell Therapy Workshop Convenes Leaders in Neurodegeneration Research


Key Points

  • Seventy-five experts discussed burning questions related to focused ultrasound and gene and cell therapy, specifically for neurodegenerative diseases.
  • A white paper summarizing the meeting will be available in the coming weeks. 
Focused Ultrasound for Gene and Cell Therapy

This November, the Foundation held a first-of-its-kind workshop to discuss focused ultrasound and gene and cell therapy, specifically for neurodegenerative diseases. More than 75 experts from academia, industry, government, and the not-for-profit sector attended in person in Washington, DC. The two-day program featured 26 invited speakers and panelists.  

The goal of the meeting was to evaluate the current state of the field for focused ultrasound–mediated gene therapy, identify knowledge gaps, and understand how focused ultrasound can be used to augment neurodegenerative disease treatments. Key topics that were discussed include focused ultrasound methods and routes for gene therapy delivery, vector design, clinical translation, and regulatory considerations. The meeting also served as an opportunity for attendees to network with each other between academia, focused ultrasound system manufacturers, pharmaceutical companies, and others to advance the field.  

Discussion focused on the following six burning questions: 

  1. What are the needs and gaps in gene delivery or gene therapy that focused ultrasound can address?  
  1. 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?  
  1. Which vectors will benefit most from combination with focused ultrasound?  
  1. How can we validate the delivery and effectiveness of gene or cell therapy to the target site?  
  1. What obstacles must be overcome to progress to clinical trials?  
  1. What types of partnerships should be pursued to facilitate transition to clinical trials? 

The workshop led to two important outcomes: 

  • Attendees laid the groundwork to establish a working group to foster networking and collaborations, begin comparing different blood-brain barrier opening approaches, and better understand focused ultrasound–mediated gene therapy delivery. 
  • Attendees identified strategic challenges, which the Foundation plans to work collaboratively to overcome.  

“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 inaugural workshop was a pivotal step toward propelling focused ultrasound–mediated gene therapy into clinical trials.” 

A white paper summarizing the discussion will be available in the coming weeks. 

Read More about Focused Ultrasound and Gene Therapy