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ECUR-506
- Development stage
- Unknown
- Lead developer
- iECURE
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
**ECUR-506**, formerly **GTP-506**, is iECURE's investigational, liver-directed in vivo gene-insertion therapy for neonatal-onset ornithine transcarbamylase deficiency. The treatment consists of two intravenously co-administered adeno-associated virus vectors: an ARCUS nuclease vector that creates a targeted genomic cut at the PCSK9 locus and a donor vector carrying a functional ornithine transcarbamylase gene cassette. Targeted integration is intended to establish durable hepatic expression of functional ornithine transcarbamylase, restore urea-cycle ammonia detoxification, and reduce hyperammonemic crises. ECUR-506 is being evaluated in the OTC-HOPE clinical study in male infants with genetically confirmed neonatal-onset ornithine transcarbamylase deficiency.
- Other names
- AAV-RH79-ARCUS-PCSK9AAV-RH-79-ARCUS-PCSK9AAV-RH 79-ARCUS-PCSK9adeno-associated virus serotype RH79 encoding a meganuclease targeting PCSK9
02
Targets
PCSK9 (Proprotein convertase subtilisin/kexin type 9)OCT (Organic cation transporter family)
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