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

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April 28, 2026

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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

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March 18, 2026

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

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January 7, 2026

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

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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

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October 2, 2025

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Initial Findings from the First Four Patients Enrolled in OTC-HOPE Clinical Trial: No Hyperammonemic Events in First Participant to Complete 24-week Study

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https://iecure.com/wp-content/uploads/ICIEM-poster_web.pdf

iECURE to Present Initial OTC-HOPE Clinical Trial Data of ECUR-506 in Ornithine Transcarbamylase Deficiency at 6th International Symposium on UCDs and 15th International Congress of Inborn Errors of Metabolism

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August 25, 2025

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Initial clinical results from OTC-HOPE, the first in vivo, liver directed, AAV-mediated gene insertion study in neonatal OTC deficiency: complete clinical response observed in first male infant to receive ECUR-506 and complete 24-week Phase 1/2 study

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https://iecure.com/wp-content/uploads/iECURE_ASGCT_Poster-852_Final.pdf