Gene therapy trial hopes to ease Parkinson's symptoms

NCT ID NCT04167540

Ongoing Disease control Sponsor: Brain Neurotherapy Bio, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This early-stage trial tests a single dose of a gene therapy called AAV2-GDNF, delivered directly into the brain, for people with Parkinson's disease. The main goal is to check safety in 11 adults aged 35-75. Researchers will also look at whether it improves motor symptoms.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: AAV2-GDNF (a gene therapy that delivers GDNF protein to brain cells)
What this could lead to: If safe and effective, this could point toward a new way to slow or improve motor symptoms in Parkinson's disease.
What could go wrong: This is a very early Phase 1 trial with only 11 people, focused on safety. It may not show clear benefit, and brain infusion carries risks like bleeding or infection.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Parkinson disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PARKINSON'S DISEASE

Contacts and locations

Locations

The Ohio State University Medical Center

Columbus, Ohio, 43210, United States
University of California Irvine

Irvine, California, 92697, United States
University of California San Francisco

San Francisco, California, 94103, United States

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