One-Time gene therapy could change fabry disease treatment

NCT ID NCT04046224

Completed Disease control Sponsor: Sangamo Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This trial tested a new gene therapy called ST-920 for Fabry disease, a rare genetic condition. The therapy uses a harmless virus to deliver a working gene that helps the body produce an enzyme it's missing. 36 adults with Fabry disease received a single intravenous dose and were monitored for a year to check safety and tolerability. The goal is to see if this one-time treatment can provide long-term benefits.

What this could mean

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

Active substance: ST-920 (a gene therapy using a modified virus to deliver a working copy of the alpha-galactosidase A gene)
What this could lead to: If successful, this could provide a long-term treatment option for Fabry disease, potentially reducing or eliminating the need for regular enzyme replacement therapy.
What could go wrong: This is an early-phase trial (Phase 1/2) with only 36 participants, so safety and effectiveness are not yet proven. Gene therapies can have unexpected side effects, and long-term benefits are uncertain.

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Conditions

The condition(s) this trial relates to.

Fabry disease lysosomal storage disease

As listed by the trial registrant

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

FABRY DISEASE

Contacts and locations

Locations

Addenbrooke's Hospital

Cambridge, CB2 0QQ, United Kingdom
Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States
Azienda Ospedaliero-Universitaria Careggi

Florence, Tuscany, 50134, Italy
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Emory University School of Medicine

Atlanta, Georgia, 30322, United States
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)

Fairfax, Virginia, 22030, United States
M.A.G.I.C. Clinic Ltd.

Calgary, Alberta, T2E 7Z4, Canada
Mt. Sinai School of Medicine

New York, New York, 10029, United States
National Taiwan University Hospital

Taipei, Taiwan
Queen Elizabeth Hospital

Birmingham, B15 2TH, United Kingdom
Royal Free Hospital

London, United Kingdom
The Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia
University Hospital of Würzburg

Würzburg, Germany
University Medical Center Hamburg-Eppendorf

Hamburg, Germany
University of California, Irvine

Irvine, California, 92697, United States
University of Iowa Hospital and Clinics

Iowa City, Iowa, 52242, United States
University of Minnesota Medical Center

Minneapolis, Minnesota, 55455, United States
University of South Florida

Tampa, Florida, 33620, United States

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