iECURE Reports Sustained Clinical Response in the First Patient Dosed with ECUR-506 & Reductions in the Annualized Rates of Hyperammonemic Events and Crises in the First Completed Cohort of ECUR-506 in Ongoing OTC-HOPE Trial

May 18, 2026

Decreased rate of hyperammonemic crises in infants with neonatal-onset OTC deficiency post ECUR-506 administration: preliminary update from the OTC-HOPE study

https://iecure.com/wp-content/uploads/ASGCT-2026_iECURE_Presentation.pdf

iECURE Presents Preliminary Clinical Data from Ongoing Clinical Trial of ECUR-506 in Ornithine Transcarbamylase Deficiency at the ASGCT Annual Meeting

May 13, 2026

iECURE to Present Preliminary Clinical Data from Ongoing OTC-HOPE Trial of ECUR-506 in Neonatal-Onset Ornithine Transcarbamylase Deficiency at the ASGCT and SIMD Annual Meetings

April 28, 2026

iECURE Selected for U.S. FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot Program to Support Manufacturing Readiness of ECUR-506 Ahead of Planned BLA Submission

March 18, 2026

iECURE Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ECUR-506 for Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency

January 7, 2026

OTC-HOPE: The first in vivo, liver directed, AAV-mediated, gene insertion clinical trial in infants with Ornithine Transcarbamylase Deficiency

https://iecure.com/wp-content/uploads/ASHG_iEcure_72x36_poster.pdf

iECURE to Present OTC-HOPE Clinical Trial Data of ECUR-506 in Ornithine Transcarbamylase Deficiency at the European Society of Gene & Cell Therapy Annual Congress and the American Society of Human Genetics Annual Meeting

October 2, 2025