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
iECURE, Inc., a genome editing company focused on the development of variant-agnostic in vivo targeted gene insertion therapies for the treatment of liver disorders with significant unmet need, today announced that additional data from the ongoing OTC-HOPE clinical trial evaluating its candidate ECUR-506 in neonatal onset ornithine transcarbamylase (OTC) deficiency will be presented at the 6th International Symposium on Urea Cycle Disorders (UCDs), taking place September 1-2, 2025 in Kyoto, Japan, and the 15th International Congress of Inborn Errors of Metabolism (ICIEM), taking place September 3-6, 2025 in Kyoto, Japan.
The poster presentations will build upon data the company originally reported in January 2025 showing that the first infant dosed in the ongoing OTC-HOPE study experienced a complete clinical response as per study protocol.
6th International Symposium on Urea Cycle Disorders Presentation Details:
- Title: 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
- Session: Poster Session
- Date/Time: Monday, September 1 – Tuesday, September 2
- Location: Hotel Okura Kyoto
- Presenter: George Diaz, M.D., Ph.D., VP, Therapeutic Area Lead, UCDs, iECURE
15th International Congress of Inborn Errors of Metabolism (ICIEM) Presentation Details:
- Title: Initial Findings from the First Four Patients Enrolled in OTC-HOPE Clinical Trial: No Hyperammonemic Events in First Participant to Complete 24-week Study (#BP-30)
- Session: Best Poster Award Presentation Session
- Date/Time: Thursday, September 4 at 2:00 p.m. JST
- Location: Event Hall, Kyoto International Conference Center
- Presenter: George Diaz, M.D., Ph.D., VP, Therapeutic Area Lead, UCDs, iECURE
iECURE will also be presenting at the ICIEM Exhibition Booth, September 3-6, 2025, at the Event Hall in the Kyoto International Conference Center, Booth #E.
About the OTC-HOPE Study
The OTC-HOPE study is a first-in-human clinical trial of ECUR-506 in baby boys with genetically confirmed neonatal onset OTC deficiency and has been cleared to evaluate ascending dose levels of ECUR-506, if necessary. The study is enrolling newborn males up to seven months of age at screening who are diagnosed with severe neonatal onset OTC deficiency and meet certain other criteria. The primary objective is to assess the safety and tolerability of intravenous administration of a single dose of ECUR-506. It will also assess the pharmacokinetics and efficacy of ECUR-506 administration and the potential effects of ECUR-506 on clinical outcome measures, disease-specific biologic markers, developmental milestones and quality of life. The main study will occur in a series of stages over a 10-month period, including screening, stabilization, dosing eligibility, study drug administration, and six-month follow-up. Upon completion of the OTC-HOPE study, participants transition to the 14.5 year long term follow up study (ECUR-LTFU). For more information, visit https://OTC-HOPE.com.
About iECURE
iECURE is a clinical-stage genome editing company focused on developing therapies that utilize variant-agnostic in vivo targeted gene insertion for the treatment of liver disorders with significant unmet need. We believe our approach has the potential to restore the function of a dysfunctional gene, regardless of variant, by knocking-in a functional copy of that gene to offer durable gene expression and long-term, potentially curative, therapeutic benefit. Our management team has extensive experience in executing global orphan drug and gene therapy clinical trials and successfully commercializing multiple products. We intend to leverage our team's core strength in research and development strategy to identify what we believe to be the most suitable target and modality for our product candidates to address particular liver diseases. For more information, visit https://iecure.com and follow on LinkedIn.
About Precision BioSciences & ARCUS®
Precision BioSciences, Inc. is a clinical stage gene editing company dedicated to improving life (NASDAQ:DTIL) with its novel and proprietary ARCUS® genome editing platform that is designed to differ from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, Precision's pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases such as chronic hepatitis B where no adequate treatments exist. For more information about Precision BioSciences, visit www.precisionbiosciences.com.
[1] iECURE has licensed the ARCUS® nuclease from Precision BioSciences for four gene insertion programs including OTC, CTLN1 and PKU.
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