Gene therapy shot aims to fix gaucher disease in kids

NCT ID NCT06528080

Ongoing Disease control Sponsor: Shanghai Jiao Tong University School of Medicine Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Aug 11, 2026 · Updated 3 times

Summary

This early-phase trial tests a single intravenous dose of LY-M001 gene therapy in 9 children (ages 6 to 17) with type 1 Gaucher disease. The goal is to see if it is safe and can improve key symptoms like liver size and blood markers. Researchers will monitor participants for side effects and measure how well the therapy works over 52 weeks, with long-term follow-up.

What this could mean

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

Active substance: LY-M001 (gene therapy)
What this could lead to: If it works, this could point toward a one-time gene therapy that reduces or eliminates the need for regular enzyme replacement in children with Gaucher disease.
What could go wrong: This is a very early, small trial (9 participants) focused on safety. Gene therapies can have unexpected side effects, and it is too soon to know if it will improve symptoms long-term.

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.

Gaucher disease Gaucher disease type I

As listed by the trial registrant

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

GAUCHER DISEASE

Contacts and locations

Locations

Shanghai Children's Medical Center, School of Medicine, Shanghai Jiao Tong University

Shanghai, Shanghai Municipality, China

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