Gene therapy shot aims to fix gaucher disease in kids
NCT ID NCT06528080
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Shanghai Children's Medical Center, School of Medicine, Shanghai Jiao Tong University
Shanghai, Shanghai Municipality, China