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UK Approves IEcure's CTA Application to Start Ornithine Transcarbamylase Gene-Editing Trial

NEW YORK – IEcure on Wednesday said it has received permission from the UK Medicines and Healthcare Products Regulatory Agency to begin a clinical trial of ECUR-506, an investigational gene-editing therapy for ornithine transcarbamylase deficiency in infants.

Approval of the clinical trial authorization application in the UK enables the Philadelphia-based firm to expand plans for the OTC-HOPE study, which has already been approved in Australia.

The Phase I/II OTC-HOPE study is a first-in-human trial evaluating ECUR-506 in infant male patients with neonatal onset of genetically confirmed OTC deficiency, a rare and inherited disorder in which ammonia accumulates in the blood. The disorder is caused by mutations in the OTC gene and, in patients with the neonatal-onset form of the condition, is primarily seen in boys.

Investigators primarily will assess safety and tolerability of up to two dose levels of ECUR-506, as well as initial pharmacokinetics, efficacy, and exploratory endpoints including biomarkers, developmental milestones, and quality of life outcomes.

"We are in the process of ensuring sites are prepared to enroll patients and anticipate site initiations in the upcoming months," IEcure CEO Joe Truitt said in a statement. "Throughout 2024, we will continue to be in close contact with other regulatory bodies as we seek to expand the OTC-HOPE study into additional geographies."

ECUR-506, which is administered to patients intravenously, uses two adeno-associated virus capsids to deliver payloads in vivo. It comprises an Arcus nuclease vector that targets gene-editing in the PCSK9 gene, which serves as the insertion site for a functional copy of the OTC gene, which is itself delivered by a donor vector. IEcure has licensed the Arcus nuclease vector from Precision BioSciences, another gene-editing company.