# Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders

Clinical trial · Rebecca Ahrens-Nicklas

- Archive id: nct:NCT07667387
- Published: 2026-06-25
- Venue: ClinicalTrials.gov
- ClinicalTrials.gov: https://clinicaltrials.gov/study/NCT07667387
- Source URL: https://clinicaltrials.gov/study/NCT07667387
- Provenance: clinicaltrials

## Abstract

This is a single-site Phase 1/2 open-label umbrella clinical trial designed to evaluate the safety, tolerability, and efficacy of a single intravenous dose of LNP.UCD.ABE in 5 pediatric subjects with severe infantile-onset UCDs. This is a master clinical protocol in which subjects with a variant in a urea cycle disorder (UCD) gene (CPS1, OTC, ASS1, ASL, ARG, NAGS, or SLC25A15) that is demonstrated to be amenable to corrective editing by an adenine base editor (ABE) would be eligible for enrollment.

_Abstract reproduced under ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters._

## Trial details

- status: RECRUITING
- phase: PHASE1, PHASE2
- study type: INTERVENTIONAL
- enrollment: 7
- sponsor: Rebecca Ahrens-Nicklas
- conditions: Urea Cycle Disorders; Carbamoyl-Phosphate Synthase I Deficiency
- interventions: BIOLOGICAL: LNP.UCD.ABE
- start date: 2026-08-07
- start date type: ACTUAL
- primary completion date: 2028-09-01
- has results: false
- data source: ClinicalTrials.gov
- data processed: 2026-10-29T09:00:05
- modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters

Topics: [CRISPR & Gene-Editing Therapies](https://space-kitty.com/topics/gene-editing-therapies)

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