Drugs / ECUR-506
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT06255782 | Apr 2024 → Dec 2027 expected | ornithine carbamoyltransferase deficiency | iECURE, Inc. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 23
| Date | Issuer | Release |
|---|---|---|
| 2026-08-27 | iECURE, Inc. | Results Decreased rate of hyperammonemiccrises in infants with neonatal-onset OTC deficiency (OTCD) post ECUR-506 administration iecure.com ↗
Decreased rate of hyperammonemic crises in infants with neonatal-onset OTC deficiency (OTCD) post ECUR-506 administration |
| 2026-08-26 | iECURE, Inc. | Results iECURE Presents Updated Clinical Data from OTC-HOPE Trial of ECUR-506 in Neonatal-Onset Ornithine Transcarbamylase Deficiency at SSIEM 2026 iecure.com ↗
Updated data from the low-dose cohort demonstrated a 57% reduction in the annualized rate of hyperammonemic events (HAEs; p=0.02) and a 74% reduction in the annualized rate of hyperammonemic crises (HACs; p=0.01) following ECUR-506 administration. |
| 2026-06-10 | iECURE, Inc. | Results Decreased rate of hyperammonemic crises in infants with neonatal-onset OTC deficiency post ECUR-506 administration: Preliminary update from the OTC-HOPE study iecure.com ↗
ECUR-506 was generally well tolerated across all dose cohorts. |
| 2026-05-15 | iECURE, Inc. | Results Sustained Clinical Response in First Patient Dosed With ECUR-506 One Year Post Discontinuation of Standard of Care iecure.com ↗ |
| 2026-05-15 | iECURE, Inc. | Results iECURE Reports Sustained Clinical Response in the First Patient Dosed with ECUR-506 & Reductions in the Annualized Rates of Hyperammonemic Events and Crises in the First Completed Cohort of ECUR-506 in Ongoing OTC-HOPE Trial iecure.com ↗
The first infant treated with ECUR-506 demonstrated a durable clinical response, with sustained discontinuation of standard-of-care therapies and no hyperammonemic events (HAEs) through 18 months post dose, as of February 11, 2026. |
| 2026-05-12 | iECURE, Inc. | Results iECURE Presents Preliminary Clinical Data from Ongoing Clinical Trial of ECUR-506 in Ornithine Transcarbamylase Deficiency at the ASGCT Annual Meeting iecure.com ↗
Preliminary data from participants enrolled in three dose cohorts (n=7) demonstrate that 71% of participants experienced no hyperammonemic crises (HACs) following ECUR-506 treatment, as of the April 20, 2026 data cutoff date. |
| 2026-05-12 | iECURE, Inc. | Results Decreased rate of hyperammonemic crises in infants with neonatal-onset OTC deficiency post ECUR-506 administration: preliminary update from the OTC-HOPE study iecure.com ↗
Decreased rate of hyperammonemic crises in infants with neonatal-onset OTC deficiency post ECUR-506 administration: preliminary update from the OTC-HOPE study |
| 2026-05-01 | iECURE, Inc. | Results Eighteen-Month Clinical Update from the First Patient Dosed in OTC-HOPE iecure.com ↗
Eighteen-Month Clinical Update from the First Patient Dosed in OTC-HOPE |
| 2026-04-29 | iECURE, Inc. | Results iECURE to Present Preliminary Clinical Data from Ongoing OTC-HOPE Trial of ECUR-506 in Neonatal-Onset Ornithine Transcarbamylase Deficiency at the ASGCT and SIMD Annual Meetings iecure.com ↗
The oral presentation at ASGCT will include preliminary data from study participants in the first three dose cohorts (n=7) of the OTC-HOPE study, including a decreased rate of hyperammonemic crises (HAC) following ECUR-506 administration. |
| 2026-03-17 | iECURE, Inc. | Regulatory iECURE Selected for U.S. FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot Program to Support Manufacturing Readiness of ECUR-506 Ahead of Planned BLA Submission iecure.com ↗ |
| 2026-01-07 | iECURE, Inc. | Regulatory iECURE Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ECUR-506 for Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency iecure.com ↗
the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ECUR-506 |
| 2025-10-16 | iECURE, Inc. | Results OTC-HOPE: The first in vivo, liver directed, AAV-mediated, gene insertion clinical trial in infants with Ornithine Transcarbamylase Deficiency iecure.com ↗ |
| 2025-03-26 | Precision BioSciences, Inc. | Results Precision BioSciences Reports Fourth Quarter and Fiscal Year 2024 Financial Results and Provides Business Update precisionbiosciences.com ↗
Announced complete clinical response in first infant dosed by partner iECURE in ongoing Phase 1/2 clinical trial in Ornithine Transcarbamylase (OTC) Deficiency |
| 2025-03-21 | iECURE, Inc. | Results iECURE Announces Presentation of Full Data for the First Infant Dosed with ECUR-506 in OTC-HOPE Phase 1/2 Clinical Trial at the 2025 ACMG Annual Clinical Genetics Meeting iecure.com ↗
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 data presented at the 2025 ACMG Annual Clinical Genetics Meeting from the ongoing OTC-HOPE Phase 1/2 study of ECUR-506 suggest partial restoration of functional ornithine transcarbamylase (OTC) enzyme activity in the first patient dosed. |
| 2025-01-09 | Precision BioSciences, Inc. | Results Precision BioSciences Announces Complete Clinical Response in First Infant Dosed by Partner iECURE in Ongoing Phase 1/2 Clinical Trial in Ornithine Transcarbamylase (OTC) Deficiency precisionbiosciences.com ↗
Treatment with ECUR-506 resulted in a complete clinical response from three months post exposure to the end of study (six months post exposure) |
| 2025-01-08 | iECURE, Inc. | Results iECURE Reports Complete Clinical Response in First Infant Dosed with its In Vivo Gene Editing Candidate ECUR-506 in an Ongoing Phase 1/2 Clinical Trial in Ornithine Transcarbamylase (OTC) Deficiency iecure.com ↗
Twelve weeks after a single dose of ECUR-506, ammonia scavenger medication was discontinued and mean daily protein intake was increased to age-appropriate levels. |
| 2024-05-07 | Precision BioSciences, Inc. | Regulatory Precision BioSciences Announces Receipt of FDA Fast Track Designation for ECUR-506, iECURE’s Program for the Treatment of Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency precisionbiosciences.com ↗ |
| 2024-05-07 | iECURE, Inc. | Regulatory iECURE Receives FDA Fast Track Designation for ECUR-506 for the Treatment of Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency iecure.com ↗ |
| 2024-04-04 | iECURE, Inc. | Regulatory iECURE Announces FDA Clearance of Investigational New Drug Application for ECUR-506 to Initiate OTC-HOPE Trial for Treatment of Neonatal Onset Ornithine Transcarbamylase Deficiency in the U.S. iecure.com ↗ |
| 2024-03-06 | Precision BioSciences, Inc. | Regulatory Precision BioSciences Announces MHRA Approval for Partner iECURE to Expand Phase 1/2 Clinical Trial of ARCUS Gene Editing Program in OTC Deficiency precisionbiosciences.com ↗
today announced that its partner iECURE has received approval from the U.K. Medicines & Healthcare products Regulatory Agency (MHRA) for the company’s Clinical Trial Authorization (CTA) application to expand the Phase 1/2 OTC-HOPE study evaluating ECUR-506 into the U.K. |
| 2024-03-05 | iECURE, Inc. | Regulatory iECURE Secures Approval from U.K. Medicines & Healthcare Products Regulatory Agency to Expand the OTC-HOPE Study of ECUR-506 iecure.com ↗ |
| 2023-12-13 | Precision BioSciences, Inc. | Regulatory Precision BioSciences Announces Approval of First Clinical Trial Application of ARCUS Gene Insertion Program by Partner iECURE precisionbiosciences.com ↗
The acceptance of iECURE’s Clinical Trial Application marks an important milestone for patients with OTC deficiency and for both the iECURE and Precision BioSciences teams. |
| 2023-12-12 | iECURE, Inc. | Regulatory iECURE Secures Clearance from Australian Therapeutic Goods Administration for its Clinical Trial Application for the OTC-HOPE Phase 1/2 Study of ECUR-506 iecure.com ↗
The CTN approval represents the first of such regulatory approvals to begin clinical trials, with additional approvals expected in 2024. |
All press releases naming this drug 28 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ECUR-506 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06255782 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “ECUR-506 uses a delivery system based on adeno-associated virus (AAV), a commonly used viral vector” NCT06255782 ↗ |
| Route | Intravenous | “The study drug is administered as a single IV infusion.” NCT06255782 ↗ |
| Target | OTC | “ECUR-506 contains a functional copy of the OTC gene, along with a gene to encode an editing enzyme that enables insertion of the OTC gene into the genome.” NCT06255782 ↗ |