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Rebecca Ahrens-nicklas

Clinical trials sponsored by Rebecca Ahrens-nicklas, explained in plain language.

Trials to join now! 1

One-Time gene fix could free kids from lifelong diet and meds

Disease control Recruiting now

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 1/2 • Sponsor: Rebecca Ahrens-Nicklas • Aim: Disease control

Last updated Aug 28, 2026 00:00 UTC