One-Time gene therapy could change gaucher treatment forever
NCT ID NCT06818838
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new gene therapy called LY-M001 for adults with Gaucher disease type 1, a genetic disorder that causes organ damage and other health problems. The therapy uses a harmless virus to deliver a working copy of the GBA1 gene to liver cells, aiming to restore the missing enzyme. Twelve participants will receive a single intravenous dose, and researchers will monitor safety and effectiveness over a year.
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 (a gene therapy using a modified virus to deliver a working GBA1 gene to liver cells)
What this could lead to
If successful, this could provide a long-lasting treatment that reduces or eliminates the need for regular enzyme replacement therapy for Gaucher disease type 1.
What could go wrong
This is an early-phase trial with only 12 participants, so results may not apply to everyone. Gene therapies can cause immune reactions or liver problems, and long-term effects are unknown.
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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Guangzhou First People's Hospital
RECRUITINGGuangzhou, Guangdong, 510000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Hematology Hospital, Chinese Academy of Medical Sciences
RECRUITINGTianjin, Tianjin Municipality, 300011, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Shanxi Bethune Hospital
RECRUITINGTaiyuan, Shanxi, 030000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact