On August 27, 2026, iECURE announced updated clinical data from the ongoing clinical trial evaluating its AAV-based gene therapy, ECUR-506, for neonatal-onset ornithine transcarbamylase (OTC) deficiency. The data were presented in an oral presentation at the Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM).

ECUR-506 is an investigational, in vivo targeted gene insertion therapy designed to restore OTC enzyme activity by inserting a functional copy of the OTC gene into the well-characterized PCSK9 gene locus in liver cells. The therapy utilizes a novel dual-AAV delivery strategy using the same capsid, each carrying a distinct payload. One vector contains an ARCUS® nuclease designed to create an insertion site within the PCSK9 locus, while the second vector delivers a functional OTC gene for targeted insertion.

According to the updated data, ECUR-506 was generally well tolerated across all three dose cohorts, with no unexpected treatment-related safety events reported. In the low-dose cohort, administration of ECUR-506 resulted in a 57% reduction in the annualized rate of hyperammonemic events (HAEs) and a 74% reduction in the annualized rate of hyperammonemic crises (HACs), the more severe subset of HAEs. According to one of the study investigators, these findings support the continued evaluation of higher ECUR-506 dose levels.

Given that the study includes some of the youngest and most severely affected patients with OTC deficiency, these clinical findings highlight the potential of in vivo gene editing and underscore the importance of safe and effective delivery technologies. In addition, higher doses of AAV vectors place greater demands on AAV purity and manufacturing quality.

As part of the broader gene therapy ecosystem, Genevoyager provides end-to-end CRO/CDMO services, from drug discovery to large-scale manufacturing, leveraging both HEK293 and our proprietary Bac/Sf9 AAV production platforms to produce high-quality AAV vectors with minimal impurities and no detectable rcAAV. With 30+ drug development projects and multiple IIT/IND submissions supported, Genevoyager has helped advance AAV-based gene therapies and recombinant protein vaccines into clinical development, including programs targeting Parkinson’s disease, refractory epilepsy, hepatitis B, and cancer, with selected programs having received FDA IND clearance and entered Phase I trials. Backed by extensive experience, we are committed to delivering safe, high-quality solutions to academic, pharmaceutical, and biotech partners worldwide.

Source:

iecure.com

Image source:

iecure.com