ECUR-506D

ECUR-506D is the donor DNA template component of **ECUR-506**, an investigational *in vivo* gene insertion therapy developed by **iECURE** for the treatment of neonatal-onset ornithine transcarbamylase (OTC) deficiency. ECUR-506 utilizes a dual-vector approach: **ECUR-506N**, which carries an ARCUS meganuclease (licensed from Precision BioSciences) to create a site-specific double-strand break in the well-characterized PCSK9 locus in the liver, and **ECUR-506D**, which provides the functional OTC gene template for insertion via homology-directed repair or non-homologous end joining. By inserting the functional gene into the genome of hepatocytes, the therapy aims to provide a permanent, stable source of the OTC enzyme, which is critical for the urea cycle. This approach is specifically designed for infants, where traditional AAV-based gene replacement might be diluted as the liver grows. The program originated from research at the University of Pennsylvania's Gene Therapy Program.

Other names

OTC donor vectorAAV8-OTC donorAAV-8-OTC donorAAV 8-OTC donor