iECURE to Present Initial Data from Phase 1/2 OTC-HOPE Clinical Trial in Ornithine Transcarbamylase Deficiency at ACMG Annual Clinical Genetics Meeting
iECURE announced it will present initial data from its Phase 1/2 OTC-HOPE clinical trial for ECUR-506, an in vivo gene editing treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency, at the 2025 ACMG Annual Clinical Genetics Meeting in Los Angeles.
The presentation will expand on previously reported data from January 2025, which showed that the first infant treated in the trial achieved a complete clinical response according to study protocol. Dr. Julien Baruteau from Great Ormond Street Hospital for Children, London, will present the findings on March 21, 2025.
iECURE ha annunciato che presenterà i dati iniziali del suo trial clinico di Fase 1/2 OTC-HOPE per ECUR-506, un trattamento di editing genetico in vivo per la carenza di ornitina transcarbamilasi (OTC) con esordio neonatale, durante l'Annual Clinical Genetics Meeting 2025 a Los Angeles.
La presentazione approfondirà i dati precedentemente riportati a gennaio 2025, che hanno mostrato che il primo neonato trattato nel trial ha raggiunto una risposta clinica completa secondo il protocollo dello studio. Il Dott. Julien Baruteau dell'Ospedale Great Ormond Street per Bambini, Londra, presenterà i risultati il 21 marzo 2025.
iECURE anunció que presentará datos iniciales de su ensayo clínico de Fase 1/2 OTC-HOPE para ECUR-506, un tratamiento de edición genética in vivo para la deficiencia de ornitina transcarbamilasa (OTC) de inicio neonatal, en la Reunión Anual de Genética Clínica ACMG 2025 en Los Ángeles.
La presentación ampliará los datos previamente reportados en enero de 2025, que mostraron que el primer bebé tratado en el ensayo alcanzó una respuesta clínica completa de acuerdo con el protocolo del estudio. El Dr. Julien Baruteau del Hospital Great Ormond Street para Niños, Londres, presentará los hallazgos el 21 de marzo de 2025.
iECURE는 LA에서 열리는 2025 ACMG 연례 임상 유전학 회의에서 ECUR-506에 대한 1/2상 OTC-HOPE 임상 시험의 초기 데이터를 발표할 것이라고 발표했습니다. ECUR-506은 신생아 발병 오르니틴 트랜스카바밀라제 (OTC) 결핍을 위한 생체 내 유전자 편집 치료입니다.
이번 발표는 2025년 1월에 보고된 데이터에 대한 추가 설명을 제공하며, 해당 데이터는 시험에서 첫 번째로 치료받은 아기가 연구 프로토콜에 따라 완전한 임상 반응을 달성했음을 보여주었습니다. 런던의 그레이트 오르몬드 스트리트 아동병원 소속의 Julien Baruteau 박사가 2025년 3월 21일 결과를 발표할 예정입니다.
iECURE a annoncé qu'il présentera des données initiales de son essai clinique de Phase 1/2 OTC-HOPE pour ECUR-506, un traitement d'édition génétique in vivo pour la déficience en ornithine transcarbamylase (OTC) à début néonatal, lors de la Réunion Annuelle de Génétique Clinique ACMG 2025 à Los Angeles.
La présentation approfondira les données précédemment rapportées en janvier 2025, qui ont montré que le premier nourrisson traité dans l'essai a atteint une réponse clinique complète selon le protocole de l'étude. Le Dr. Julien Baruteau de l'Hôpital Great Ormond Street pour Enfants, Londres, présentera les résultats le 21 mars 2025.
iECURE gab bekannt, dass es erste Daten aus seiner Phase 1/2 OTC-HOPE-Studie für ECUR-506, eine in vivo Genbearbeitungstherapie für die neonatale Ornithintranscarbamylasemangel (OTC), auf der ACMG Jahrestagung für Klinische Genetik 2025 in Los Angeles präsentieren wird.
Die Präsentation wird auf zuvor berichtete Daten aus Januar 2025 eingehen, die zeigten, dass das erste im Versuch behandelte Baby eine vollständige klinische Antwort gemäß dem Studienprotokoll erreicht hat. Dr. Julien Baruteau vom Great Ormond Street Hospital for Children in London wird die Ergebnisse am 21. März 2025 vorstellen.
- First infant in Phase 1/2 trial achieved complete clinical response
- Successful initial proof of concept for gene editing therapy ECUR-506
- data from only one patient so far
The poster presentation will provide further insights and analysis to 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.
Presentation Details:
- Title: A First In Human, Single Arm, Open Label Phase 1/2 Study Evaluating ECUR-506 in Neonatal OTC Deficiency: Initial Observations (Poster #P008)
- Date/Time: Friday, March 21 at 10:30 a.m. – 11:30 a.m. PT
- Location: West Exhibit Hall A
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Presenter: Julien Baruteau, MD, Great Ormond Street Hospital for Children,
London, U.K.
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, 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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20250312255770/en/
Investors:
David Garrett
dgarrett@iecure.com
Media:
Janine Bogris
Inizio Evoke Comms
janine.bogris@inizioevoke.com
Source: iECURE, Inc.
FAQ
What were the initial results of iECURE's ECUR-506 Phase 1/2 trial for OTC deficiency?
When will iECURE present the detailed OTC-HOPE trial data?
What is ECUR-506 designed to treat?
Who will present iECURE's OTC-HOPE trial results at ACMG 2025?
