Gene-Editing shot aims to tame rare 'Syrup Blood' disease
NCT ID NCT07176923
First seen Oct 01, 2025 · Last updated May 25, 2026 · Updated 25 times
Summary
This early-stage study tests CS-121, a one-time gene-editing treatment given by IV, for people with familial chylomicronemia syndrome (FCS) – a rare condition causing extremely high blood fats and risk of pancreatitis. About 15 adults aged 18-55 will receive the therapy to see if it is safe and lowers triglyceride levels. The goal is to control the disease long-term, not cure it, as ongoing monitoring is needed.
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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: •••••@•••••
Locations
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The First Affiliated Hospital of Anhui Medical University
RECRUITINGHefei, Anhui, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
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