ECUR-506 (iECURE)

ECUR-506 is an investigational in vivo gene editing therapy developed by iECURE in collaboration with Precision BioSciences for the treatment of neonatal-onset ornithine transcarbamylase (OTC) deficiency. The therapy utilizes a dual adeno-associated virus (AAVrh79) vector system to deliver two components: an ARCUS meganuclease (M2PCSK9) and a functional, codon-optimized OTC donor gene. The ARCUS nuclease is designed to create a targeted double-strand break at the *PCSK9* gene locus in hepatocytes, facilitating the stable insertion of the functional OTC gene. This approach aims to provide a durable, potentially curative treatment by restoring enzyme function in a manner that is resistant to dilution during hepatocyte proliferation in infants. ECUR-506 is currently being evaluated in the Phase 1/2/3 OTC-HOPE clinical trial.

Other names

adeno-associated virus serotype RH79 containing human OTC geneAAV-RH79-hOTCAAV-RH-79-hOTCAAV-RH 79-hOTCAAVrh79.hOTCAAVrh-79.hOTCAAVrh 79.hOTC