| Gene symbol | OTC | Synonyms | OCTD, OTC1, OTCD, OTCase | Type of gene | protein-coding |
| Chromosome | X | Map location | Xp11.4 | dbXrefs | |
| Description | ornithine transcarbamylase | ||||
| GTO ID | GTC3758 |
| Trial ID | NCT06255782 |
| Disease | Ornithine Transcarbamylase Deficiency Disease | Inborn Urea Cycle Disorder |
| Altered gene | OTC |
| Therapeutic/Target gene | Target gene |
| Therapy | Gene editing |
| Treatment | ECUR-506 |
| Phase | Phase1|Phase2 |
| Recruitment status | Recruiting |
| Title | A Phase I/II First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of a Single Intravenous (IV) Administration of ECUR-506 in Males Less Than 9 Months of Age With Genetically Confirmed Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency |
| Year | 2024 |
| Country | United States |
| Company sponsor | iECURE, Inc. |
| Other ID(s) | ECUR-506-OTC-101|OTC HOPE |
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| Cohort 1 | |||||||||
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