One-Time gene shot aims to tame rare enzyme disorder
NCT ID NCT06818838
First seen Jan 23, 2026 · Last updated May 14, 2026 · Updated 12 times
Summary
This early-phase trial tests a single injection of gene therapy (LY-M001) in 12 adults with Gaucher disease type 1, a genetic condition causing enzyme deficiency and organ damage. The therapy delivers a working gene to the liver to produce the missing enzyme. The main goals are safety and checking if the treatment can improve enzyme levels and reduce liver and spleen swelling.
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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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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
Conditions
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