iEcure

Pioneering In Vivo Gene Editing to Treat Metabolic Disorders Involving the Liver

The first in vivo gene editing therapeutic to be tested in infants: Following clinical trial clearances in the US, UK and Australia, iECURE’s ECUR-506 is poised to be the first in vivo gene editing candidate to be tested in infants in the US.
A potentially curative therapy for a deadly disease that has long stymied therapeutic developers: For decades, genetic medicine pioneers have pursued gene therapy approaches to treat ornithine transcarbamylase (OTC) deficiency to no avail, ECUR-506 may finally solve this challenge.
Key events in 2025: Interim data readouts on safety and indicators of efficacy for ECUR-506 in neonatal onset OTC deficiency, potential advancement of other pipeline programs.