Aug. 18 at 5:56 PM
$DTIL ... IECure Reminder: trial results for OTC-HOPE trial...
"1. OTC-HOPE Trial Results (ECUR-506 for Neonatal-Onset OTC Deficiency)"
"The OTC-HOPE trial is a first-in-human clinical study evaluating ECUR-506, a targeted gene insertion therapy, in male infants with neonatal-onset ornithine transcarbamylase (OTC) deficiency. Key outcomes as of 2026 include:"
"Participants: Seven infants treated across three dose cohorts."
Efficacy:
"In the low-dose cohort (three infants), there was:
57% reduction in annualized hyperammonemic events
65% reduction in hyperammonemic crises
The first treated infant experienced no hyperammonemic events for 18 months post-treatment and discontinued standard-of-care therapies.
Two of the three infants in the low-dose group experienced no hyperammonemic events or crises post-treatment."
