Gene-Editing shot aims to tame deadly fat levels in kids
NCT ID NCT07371767
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a new gene-editing treatment called CS-121 in 15 children and teens (ages 4-18) with a rare condition causing extremely high triglycerides. The therapy uses lipid nanoparticles to deliver a base editor that targets the APOC3 gene in the liver. The main goals are to check safety and see if it lowers triglyceride levels.
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 to edit the APOC3 gene)
What this could lead to
If it works, this could point toward a one-time treatment to control severe high triglycerides in children, reducing risk of pancreatitis.
What could go wrong
This is a very early, small trial (15 people) testing safety first. Gene editing is new and may have unknown risks or fail to lower triglycerides enough.
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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Shanghai Children's Medical Center
RECRUITINGShanghai, Shanghai Municipality, China
Contact Phone: •••-•••-•••• Email: •••••@•••••