The US Food and Drug Administration (FDA) has granted clearance for iECURE's Investigational New Drug (IND) application, paving the way for a Phase I/II clinical trial of ECUR-506 to treat Ornithine Transcarbamylase (OTC) deficiency.

Known as the OTC-HOPE study, the first-in-human trial will enrol newborn boys aged up to seven months with genetically confirmed neonatal onset OTC deficiency.

The primary goal of the trial is to evaluate the safety and tolerability of a single intravenous dose of ECUR-506.

Secondary objectives include analysing the pharmacokinetics and efficacy of the single-dose treatment.

In addition to these primary and secondary endpoints, the trial will explore disease-specific biological markers, developmental milestones, and quality-of-life measures.

ECUR-506 utilises a two-vector system: an ARCUS nuclease vector for targeted gene editing at the PCSK9 gene locus, as well as a donor vector to insert the functional OTC gene.

The innovative approach of ECUR-506, which involves the ARCUS nuclease creating an insertion point for a functional gene, is claimed to be the first of its kind in the clinical setting.

Prior to the FDA's clearance, the OTC-HOPE study received approval in the UK from the Medicines and Healthcare Products Regulatory Agency (MHRA) and in Australia by the Therapeutic Goods Administration (TGA).

iECURE CEO Joe Truitt said: “With this IND clearance, we are now activating sites in three countries in distinct geographical areas – US, UK and Australia – which will facilitate families’ ability to access this landmark clinical trial.

“We are working diligently to open sites so we can start enrolling patients for dosing. The trial will be accepting eligible baby boys with neonatal onset OTC deficiency from all over the world, and we hope to see positive safety data and indications of efficacy with this trial.”