One-Time gene fix could free kids from lifelong diet and meds
NCT ID NCT07667387
First seen Jun 27, 2026 · Last updated Jun 30, 2026 · Updated 2 times
Summary
This early-phase trial tests a single intravenous dose of a gene editing therapy called LNP.UCD.ABE in 7 children with severe urea cycle disorders. The therapy aims to correct the genetic defect using a lipid nanoparticle to deliver a base editor. The main goal is to check safety, but researchers will also see if it allows children to eat more protein and take less medication.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
LNP.UCD.ABE (a lipid nanoparticle-delivered gene editing therapy)
What this could lead to
If successful, this could point toward a one-time treatment that corrects the genetic defect, potentially reducing or eliminating the need for lifelong medication and dietary restrictions.
What could go wrong
This is a very early, small trial (only 7 children) testing a personalized therapy. It may not work for all patients, and there are unknown risks from the gene editing process, including off-target effects.
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: •••••@•••••
Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States