One-Time gene fix could free kids from lifelong diet and meds
Disease control
Not yet recruiting
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…
Phase: PHASE1, PHASE2 • Sponsor: Rebecca Ahrens-Nicklas • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC