Gene editing shot aims to tame deadly fat levels in rare disease
NCT ID NCT07176923
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This early-stage trial tests a single infusion of CS-121, a gene editing therapy, in 15 adults with familial chylomicronemia syndrome (FCS), a rare condition causing extremely high blood fat levels. The therapy targets the APOC3 gene to lower triglycerides and reduce disease burden. The main goal is to check safety and tolerability, with early signs of effectiveness also measured.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
CS-121 (a gene editing therapy delivered by lipid nanoparticles)
What this could lead to
If successful, this could lead to a one-time treatment that controls dangerously high fat levels in the blood for people with familial chylomicronemia syndrome.
What could go wrong
This is a very early, small Phase 1 trial with only 15 people, so safety and effectiveness are not yet known. The gene editing approach is new and may not work as hoped or could cause unexpected side effects.
Disclaimer
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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: •••••@•••••
Locations
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The First Affiliated Hospital of Anhui Medical University
RECRUITINGHefei, Anhui, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact