PHILADELPHIA, PA — iECURE, Inc. has announced that early data from its Phase 1/2 OTC-HOPE clinical trial, evaluating the gene-editing candidate ECUR-506 for neonatal onset ornithine transcarbamylase (OTC) deficiency, will be presented at the upcoming 2025 ACMG Annual Clinical Genetics Meeting in Los Angeles. The presentation represents a critical step forward in developing advanced gene editing therapies for rare genetic disorders.
Preliminary results reported in January 2025 revealed a complete clinical response in the first infant dosed with ECUR-506, setting an encouraging foundation for the ongoing trial. The detailed findings will be shared on Friday, March 21, during a poster presentation led by Dr. Julien Baruteau of Great Ormond Street Hospital for Children, London.
The OTC-HOPE Study
The OTC-HOPE clinical trial is the first of its kind to evaluate ECUR-506, an in vivo gene-editing candidate, in neonatal patients suffering from genetically confirmed OTC deficiency, a rare life-threatening metabolic disorder. The condition presents soon after birth and severely impacts the body’s ability to metabolize nitrogen, often leading to toxic levels of ammonia in the bloodstream.
The trial, designed as a single-arm, open-label Phase 1/2 study, focuses on male infants up to seven months of age. The primary goal is to assess the safety and tolerability of a single intravenous dose of ECUR-506. Secondary objectives include pharmacokinetics, efficacy, and potential improvements in patients’ developmental milestones and overall quality of life.
The trial consists of multiple stages, including screening, stabilization, dosing, and follow-up over six months. Participants will then transition into a 14.5-year long-term follow-up study (ECUR-LTFU) to monitor the enduring effects of the treatment.
Significance of ECUR-506
This groundbreaking clinical study highlights the potential of in vivo gene editing to address severe rare diseases at their source. By targeting neonatal onset OTC deficiency, iECURE aims to provide a much-needed therapeutic option that could dramatically improve clinical outcomes and quality of life for affected infants.
The upcoming presentation at ACMG offers a unique opportunity for specialists to gain deeper insights into the initial data, further validating the promise of gene editing technologies in addressing genetic conditions that currently lack viable treatments.
With the OTC-HOPE trial successfully underway, iECURE continues to push the boundaries of medical innovation in genetic therapies, paving the way for future breakthroughs in rare disease treatment.
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