Gene and Cell Therapy Program

AAV Gene Therapy Landscape for Neurodegenerative Diseases

Explore our new adeno-associated virus (AAV) gene therapy landscape analysis for neurodegenerative diseases and a searchable database of program attributes, highlighting indications where focused ultrasound may add value.

Public comments are encouraged until February 27, 2026.

Focused ultrasound for delivery and control of gene and cell therapies.

The Focused Ultrasound Foundation’s Gene and Cell Therapy Program is building an ecosystem to accelerate the clinical translation of focused ultrasound–mediated gene and cell therapies. We fund external projects that support researchers using focused ultrasound to deliver genetic and cellular therapies, and to remotely control their activity via sonogenetics. By convening experts and serving as a hub for academic and clinical teams, biotech and pharma companies, focused ultrasound system manufacturers, and regulatory stakeholders, we aim to turn promising concepts into real treatment options for patients.

Use the links below to navigate this page

• Program Goals
• Key Focus Areas
• Pathways to Clinical Translation
• Workshops and Webinars
• Partnerships
• Leadership and How to Get Involved

Why combine focused ultrasound with gene and cell therapies?

Gene and cell therapies are already changing how we treat cancer, neurodegenerative diseases, and rare disorders, among others. Their impact, however, is often limited by two core challenges:

1. Reaching the right cells and tissues efficiently
2. Controlling where and when therapeutic activity occurs

Focused ultrasound offers a noninvasive way to address both:

• As a delivery platform, focused ultrasound can temporarily increase vascular and barrier permeability (e.g., blood-brain barrier opening) and enhance local uptake of viral vectors, nanoparticles, genetic payloads, or cellular therapies, while reducing off-target exposure.
• As a control modality, focused ultrasound can regulate expression of engineered gene circuits in vivo—an emerging area known as sonogenetics—so that potent therapies such as CAR T cells or other engineered cells are activated on demand, only at the treated site.

Our program is built around these two complementary facets: focused ultrasound–enabled delivery and sonogenetic control of gene and cell therapies.

Program goals

The Gene and Cell Therapy Program is designed to build a translational pipeline for focused ultrasound-mediated gene and cell therapies, from preclinical proof-of-concept through early clinical trials. We support and connect teams across institutions and disciplines.

Our goals are to:

• De-risk focused ultrasound–enabled delivery of gene and cell therapies in the brain and in large organs, from small animals to nonhuman primates and humans. 
• Advance sonogenetic strategies that use focused ultrasound to control engineered gene circuits and cell therapies in vivo. 
• Bridge communities working on brain and large organ applications, and on gene therapy and cell therapy, to stimulate true cross-pollination. 
• Clarify translational and regulatory pathways for focused ultrasound + gene and cell therapy combinations, including routes of administration, vector and capsid choice, and device–drug co-development. 
• Identify and prioritize programs with the highest potential to reach first-in-human trials, using structured program-selection discussions with our advisory board and external experts. 

Key focus areas

The program concentrates on four interconnected focus areas:

1. Brain and Central Nervous System (CNS) Gene Therapy
   • Focused ultrasound–mediated blood-brain barrier opening
   • Routes of administration (systemic vs cerebral spinal fluid) and scaling from rodent to non-human primate to human
   • Vector and capsid selection tailored for focused ultrasound delivery to the CNS 
2. Cell Therapy and Sonogenetics for Solid and Brain Tumors
   • Focused ultrasound to improve homing and persistence of adoptive cell therapies (e.g., engineered T cells, NK cells, or stem cells) in the brain and in solid tumors
   • Thermogenetic/sonogenetic control of CAR T and other engineered cells to confine activity to the treated site, improve efficacy, or to control cell differentiation
   • Integration of focused ultrasound, cell therapy, and immune modulators to reshape the tumor microenvironment and improve antitumor responses.
3. Large Organ Gene Therapy in Priority Indications
   • Focused ultrasound–mediated delivery for solid tumors and systemic targets that are directly connected to our priority areas in oncology and neurodegenerative disease (see Foundation priorities)
   • Exploring additional priority indications where focused ultrasound, with or without engineered vectors, can be used to detarget and redirect vector tropism in a clinically meaningful way
4. Pathways to Clinical Trials
   • Understanding regulatory pathways for combined focused ultrasound + gene and cell therapy products
   • Propose program-selection frameworks

Pathways to Clinical Translation

Beyond funding individual projects, the Foundation leads an internal effort to map realistic pathways to first-in-human trials for focused ultrasound-mediated gene and cell therapies.

As a first step, we have conducted a landscape analysis of AAV-based gene therapies for neurodegenerative diseases, focusing on programs that are already in or near the clinic. Building on this analysis, we are developing a selection framework to identify indications and programs where focused ultrasound could meaningfully improve delivery or safety and realistically be integrated into future clinical trial designs.

This framework helps us prioritize which concepts and partnerships the Foundation could support, for example by highlighting specific indications, vectors, and routes of administration that could be strong candidates for focused ultrasound, with a clear path to clinical implementation.

It is an in-house, strategy-driven initiative that complements our external funding, ensuring that our Gene and Cell Therapy Program is always oriented toward realistic translational opportunities.

The initial landscape analysis version can be accessed here. This is open for public comments and we strongly ask for your expertise.

We also created a database, exploring several attributes for gene therapy neurological diseases programs. The database is open for public comments.

The open database aggregates and processes public information on AAV-based gene therapy trials for neurodegenerative diseases, while the landscape analysis applies a structured selection framework to this dataset, so together they both guide our internal program choices and serve as a standalone tool to explore current trials, AAV use, and routes of administration.

Workshops and Webinars

A core part of the Gene and Cell Therapy Program is bringing the ecosystem together.

Partnerships

The Gene and Cell Therapy Program is deliberately embedded in the broader cell and gene therapy community through strategic partnerships and memberships.

Leadership and How to Get Involved