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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

via Business Wire

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 European Society of Gene & Cell Therapy (ESGCT) Annual Congress, taking place October 7-10, 2025 in Sevilla, Spain and the American Society of Human Genetics (ASHG) Annual Meeting, taking place October 14-18 in Boston.

The upcoming poster and oral presentations will expand on the previously reported January 2025 results, which demonstrated that the first infant treated in the ongoing OTC-HOPE study experienced a complete clinical response in line with the study protocol.

European Society of Gene & Cell Therapy Annual Congress:

Presentation: OTC-HOPE: The first in vivo, liver directed, AAV-mediated, gene insertion clinical trial in infants

  • Session: Metabolic Diseases I (Session 2a)
  • Date/Time: Tuesday, October 7, 5:00 p.m. – 7:30 p.m. CEST
  • Location: Room Parallel A
  • Presenter: George Diaz, M.D., Ph.D., VP, Therapeutic Area Lead for Urea Cycle Disorders

American Society of Human Genetics Annual Meeting:

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

  • Session: Lightning Talk – Scaling Genomic Medicine: From Patient-Centered Tools to Population and Therapeutic Insights (Session 82)
  • Date/Time: Friday, October 17, 1:30 p.m. – 2:30 pm ET
  • Location: Room 206AB/Level 2, Thomas Michael Menino Convention & Exhibition Center
  • Presenter: Gabriel Cohn, M.D., MBA, Chief Medical Officer, iECURE

Poster: OTC-HOPE: The first in vivo, liver directed, AAV-mediated, gene insertion clinical trial in infants with Ornithine Transcarbamylase Deficiency (Board 6001T)

  • Session: Genetic Therapies and Precision Medicine Poster Thursday Session
  • Date/Time: Thursday, October 16, 2:30 p.m. - 4:30 p.m. ET
  • Location: Exhibit & Poster Hall, Lower Level, Thomas Michael Menino Convention & Exhibition Center
  • Presenter: Gabriel Cohn, M.D., MBA, Chief Medical Officer, iECURE

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, tolerability and efficacy of intravenous administration of a single dose of ECUR-506. It will also assess the pharmacokinetics and additional 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 ECUR-506

iECURE’s approach to targeted gene insertion for its initial programs, including OTC deficiency, relies on the delivery of two adeno-associated virus (AAV) vectors comprised of the same capsid, but each carrying different payloads. ECUR-506 comprises two vectors, an ARCUS® nuclease vector targeting gene editing in the well-characterized PCSK9 gene locus and a donor vector that inserts the desired functional OTC gene. iECURE has licensed the ARCUS® nuclease for ECUR-506 from Precision BioSciences (Nasdaq: DTIL).1

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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