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Find Clinical Drug Pipeline Developments & Deals by iECURE
ECUR-506, incorporates an ARCUS nuclease vector targeting gene editing in the well-characterized PCSK9 gene locus and a donor vector that inserts the desired functional OTC gene for the treatment of neonatal onset ornithine transcarbamylase deficiency.
Arcus is a platform designed to develop in vivo gene editing therapies, including gene insertion, excision, and elimination. It has received TGA approval for initiation of Phase 1/2 trial evaluating ECUR-506 for the treatment of Ornithine Transcarbamylase (OTC) deficiency.
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 that inserts the OTC gene.
GTP-506, a potential single-dose gene editing therapy designed to restore metabolic function in patients suffering with OTC deficiency, a rare urea cycle disorder.
The funds will be used to advance iECURE’s pipeline of up to 13 programs rely on the use of an endonuclease-based gene editing technology delivered via AAV.
Under the terms of the agreement, iECURE plans to file a clinical trial application to advance the PBGENE-PCSK9 clinical candidate through Phase 1 clinical studies for the treatment of familial hypercholesterolemia (FH).