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Clinical trial

Master Protocol for a Phase I/II Open-label Safety and Efficacy Study of LNP.UCD.ABE, a Lipid Nanoparticle-delivered Base Editing Therapy, in Patients With Urea Cycle Disorders Due to Variants Amenable to Corrective Editing by LNP.UCD.ABE

NCT07667387 is registered in ClinicalTrials.gov with Rebecca Ahrens-Nicklas as lead sponsor.

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Registry record

As filed in ClinicalTrials.gov.

Title
Master Protocol for a Phase I/II Open-label Safety and Efficacy Study of LNP.UCD.ABE, a Lipid Nanoparticle-delivered Base Editing Therapy, in Patients With Urea Cycle Disorders Due to Variants Amenable to Corrective Editing by LNP.UCD.ABE
Lead sponsor
Rebecca Ahrens-Nicklas
Phase
Phase 1/2
Overall status
Recruiting
Start date
August 7, 2026
Completion date
September 30, 2028
Enrollment
7 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

carbamoyl-phosphate synthase i deficiency, urea cycle disorders