Personalized LNP.UCD.ABE Gene Editing Therapy for Young Children With Severe Urea Cycle Disorders Evaluating Safety and Efficacy

What this Trial Is About

Researchers are conducting a Phase 12 open-label trial to study the safety, tolerability, and effectiveness of a single intravenous dose of LNP.UCD.ABE in five pediatric patients with severe infantile-onset urea cycle disorders UCDs. This trial targets children under 5 years old who have specific genetic variants in UCD-related genes that can be corrected by an adenine base editor ABE. Each participant will receive a personalized version of LNP.UCD.ABE, which is a gene editing therapy delivered via a lipid nanoparticle containing messenger RNA for the adenine base editor and guide RNA. Before treatment, each childs personalized drug will be developed and evaluated during a screening period lasting up to eight months. After a lead-in period to establish a stable diet, the child will receive a single intravenous infusion of the therapy and then be monitored. During the study, participants will be followed for 52 weeks after treatment to assess safety and tolerability. Researchers will perform various clinical evaluations, biochemical tests, and monitoring to measure the therapys effects. The main outcome is safety over 52 weeks, with secondary outcomes assessing clinical efficacy at 16 weeks. The total participation period may extend from screening through the follow-up phase lasting about one year.

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