Brain gene therapy aims to silence Huntington's disease

NCT ID NCT04120493

Ongoing Disease control Sponsor: UniQure Biopharma B.V. Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tests a one-time gene therapy called AMT-130, injected directly into the brain, to lower the production of the harmful huntingtin protein. It involves 43 adults with early Huntington's disease. The study is primarily checking safety, but also looks for signs that the treatment might slow the disease.

What this could mean

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

Active substance: AMT-130 (a gene therapy injected into the brain to lower the harmful huntingtin protein)
What this could lead to: If successful, this could point toward a treatment that slows or stops Huntington's disease progression by reducing the toxic protein at its source.
What could go wrong: This is an early, first-in-human study with only 43 participants, so safety and effectiveness are not yet proven. Brain surgery carries risks like infection, bleeding, or swelling.

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.

Huntington disease

As listed by the trial registrant

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

HUNTINGTON'S DISEASE

Contacts and locations

Locations

CenExel Rocky Mountain Clinical Research

Englewood, Colorado, 80113, United States
Johns Hopkins University

Baltimore, Maryland, 21287, United States
Ohio State University

Columbus, Ohio, 43210, United States
Rush University Medical Center

Chicago, Illinois, 60612, United States
The University of Texas

Houston, Texas, 77030, United States
University of Alabama at Birmingham

Birmingham, Alabama, 35294-0111, United States
University of Arizona (Surgical Site Only)

Tucson, Arizona, 85724, United States
University of California, San Francisco

San Francisco, California, 94158, United States
University of Michigan Department of Neurology

Ann Arbor, Michigan, 48105, United States
University of Washington Medical Center

Seattle, Washington, 98195, United States
Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States
Virginia Commonwealth University VCU School of Medicine, Department of Neurology

Richmond, Virginia, 23298, United States

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