New gene therapy aims to tackle fabry disease in early trial

NCT ID NCT06539624

Recruiting now Disease control Sponsor: The Children's Hospital of Zhejiang University School of Medicine Source: ClinicalTrials.gov ↗

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

Summary

This study tests a gene therapy called EXG110 in 12 people with Fabry disease. The therapy uses a harmless virus to deliver a working gene that may help the body produce a missing enzyme. The main goal is to check safety and find the right dose, while also looking at how it affects kidney, heart, and skin symptoms.

What this could mean

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

Active substance: EXG110 (a gene therapy using a modified virus to deliver a working gene)
What this could lead to: If successful, this could point toward a one-time treatment that helps control Fabry disease symptoms and organ damage.
What could go wrong: This is a very early, small trial with only 12 people, so results may not apply widely. Gene therapies can have side effects like immune reactions or liver issues.

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

Locations

Children's Hospital, Zhejiang University School of Medicine
RECRUITING

Hangzhou, Zhejiang, China
Shanghai Children's Medical Center
RECRUITING

Shanghai, Shanghai Municipality, China

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