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iECURE, a gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need, announced that data presented at the International Conference on Ureagenesis Defects and Allied Conditions 2022 by its research collaborators at the University of Pennsylvania’s Gene Therapy Program (GTP), showed stable insertion of the therapeutic gene one year post-dosing in newborn and infant macaques and potential signs of efficacy in mouse Ornithine Transcarbamylase (OTC) Deficiency knock out models.

iECURE’s approach to gene editing for its initial programs, including OTC deficiency, relies on the delivery of twin adeno-associated virus (AAV)-based vectors carrying different payloads. GTP-506 comprises two vectors, an ARCUS® nuclease vector (GTP-506A) targeting gene editing in the well-characterized PCSK9 gene locus and a therapeutic donor vector (GTP-506D) that inserts the OTC gene to provide the desired genetic correction.1 The cut in the PCSK9 site serves as the insertion site for the therapeutic gene, providing a potential path to permanent expression of a healthy gene.

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