AAV Gene Therapy Landscape for Neurodegenerative Diseases

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.

Beyond funding individual projects, the Foundation is pursuing an internal, strategy-driven effort to map realistic pathways to first-in-human trials for focused ultrasound–mediated gene and cell therapies. As an initial step, we conducted a landscape analysis of AAV-based gene therapies for neurodegenerative diseases that are in or near the clinic, and are using these insights to develop a framework for identifying indications and programs where focused ultrasound could be feasibly and meaningfully integrated into future clinical trial designs. This approach helps ensure the Gene and Cell Therapy Program remains focused on high-priority, translational opportunities that complement the Foundation’s external funding.

Explore our landscape analysis and a searchable database of program attributes, highlighting indications where focused ultrasound may add value.

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Public comment period: open until February 27, 2026.

You can submit feedback using the form embedded below. If you prefer, you can also email comments to Fred Padilla, PhD, at [email protected]. If you choose to annotate the Word or PDF document, please attach your marked-up file in your email.