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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

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 …

Urea Cycle DisordersCarbamoyl-Phosphate Synthase I Deficiency

~$10 – $7,500

Rebecca Ahrens-NicklasNCT07667387