Canavan Disease clinical trials at UCSF

1 research study open to eligible people

Canavan disease is a rare brain disorder. UCSF is studying a new treatment called BBP-812, which uses AAV9 gene therapy. The main goal of this trial is to test the safety and effects of this new therapy.

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  • AAV9 Gene Therapy in Participants With Canavan Disease

    open to eligible people ages up to 30 months

    The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.

    Oakland, California and other locations

Our lead scientists for Canavan Disease research studies include .

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