Gene therapy trial aims to fix enzyme defect in fabry disease

NCT ID NCT06270316

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

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial is testing a single-dose gene therapy called AMT-191 in 12 adult men with classic Fabry disease. The therapy uses a harmless virus to deliver a working copy of the GLA gene to the liver, so the body can produce the missing enzyme. The main goals are to check safety and see how the drug behaves in the body.

What this could mean

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

Active substance: AMT-191 (a gene therapy using a harmless virus to deliver a working copy of the GLA gene)
What this could lead to: If successful, this could lead to a one-time gene therapy that helps the body produce the missing enzyme, potentially reducing the need for regular enzyme infusions.
What could go wrong: This is an early-phase trial with only 12 participants, so results may not apply to everyone. Gene therapies can have side effects like immune reactions, and long-term effects are unknown.

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.

Fabry disease

As listed by the trial registrant

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

FABRY DISEASE

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••
Contact

Email: •••••@•••••

Locations

Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Emory University School of Medicine
RECRUITING

Atlanta, Georgia, 30322, United States
Lysosomal & Rare Disorders Research and Treatment Center, Inc
RECRUITING

Fairfax, Virginia, 22030, United States
MHealth Fairview University of Minnesota Medical Center East Bank
RECRUITING

Minneapolis, Minnesota, 55455, United States
NYC Health + Hospitals/Metropolitan
RECRUITING

New York, New York, 10029, United States
The Kirklin Clinic Of university of Alabama Birmingham Hospital
RECRUITING

Birmingham, Alabama, 35233, United States
UPMC Children's Hospital of Pittsburgh
RECRUITING

Pittsburgh, Pennsylvania, 15224, United States
University of Utah, Clinical and Translational Sciences Institute
RECRUITING

Salt Lake City, Utah, 84108, United States

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