iECURE, Inc., a gene editing company focused on the development of mutation-agnostic in vivo gene insertion therapies for the treatment of liver disorders with significant unmet need, today announced that initial data from the first participant to complete the ongoing OTC-HOPE clinical trial evaluating in vivo gene editing candidate ECUR-506 in neonatal onset ornithine transcarbamylase (OTC) deficiency will be presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place May 12 – 17, 2025 in New Orleans and the European Society of Human Genetics (ESHG) Conference taking place May 24 – 27, 2025 in Milan, Italy. In addition to the clinical data, iECURE will also participate in a symposium session highlighting the company’s clinical advances in genome editing at ASGCT.

The poster, oral and symposium presentations will build upon data the company 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.

ASGCT Details:

Poster 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 (852)

• Date/Time: Tuesday, May 13, 6:00 p.m. - 7:30 p.m. ET
• Location: Poster Hall, Hall I2
• Presenter: Gabriel Cohn, M.D., MBA, Chief Medical Officer, iECURE

Symposium Title: Advances In Genome Editing: Novel Large DNA Insertion Technologies and Their Potential Towards Curative Therapies

• Date/Time: Wednesday, May 14, 8:00 a.m. – 9:40 a.m. ET
• Location: New Orleans Theater B
• iECURE Title: OTC-HOPE: The first in-vivo, liver directed, AAV-mediated, gene insertion clinical trial in infants
• iECURE Presenter: Gabriel Cohn, M.D., MBA, Chief Medical Officer, iECURE

ESHG Details:

Oral Presentation Title: Initial clinical results from the first in vivo, liver directed, AAV-mediated gene insertion study with ECUR-506 in neonatal OTC deficiency: complete clinical response in first male infant to complete 24-week Phase 1/2 study (C27.05)

• Date/Time: Monday, May 26, 11:30 a.m. – 11:45 a.m. CEST
• Location: Brown 3
• Presenter: Julien Baruteau, M.D., Ph.D., University College London Great Ormond Street Institute of Child Health

Exhibition Booth

• Dates: Saturday, May 24 – Tuesday, May 27
• Location: Hall 4, Booth #232

About the OTC-HOPE Study

The OTC-HOPE study is a Phase 1/2 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 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 gene editing company focused on developing therapies that utilize mutation-agnostic in vivo 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 mutation type, 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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