Gene editing company iECURE has said data from its Phase I/II ornithine transcarbamylase (OTC) deficiency trial could suggest partial restoration of functional OTC.
New results from the company’s OCT-Hope trial (NCT06255782) saw mean blood urea nitrogen (BUN) levels after treatment with the company’s pipeline candidate ECUR-506 increase to near what is considered a normal level, implying potentially increased functional activity.
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Earlier this year, the company reported that the first patient dosed in the OTC-Hope Trial was a 6.5-month-old male baby receiving a single low dose of ECUR-506. That child later went on to see a complete clinical response along with no longer needing ammonia scavenger medication after 12 weeks.
iECURE’s chief medical officer Dr Gabriel Cohn said: “Long-term restoration of OTC activity has the potential to allow infants afflicted with neonatal onset OTC deficiency to live healthier lives unburdened by hyperammonemic crises and ongoing medical management.”
OTC deficiency is a rare genetic disorder that can cause newborn infants to build up potentially toxic levels of ammonia in the blood. It is one of the conditions newborns are typically screened for when born and is much more common in infant boys than girls. OTC is an enzyme that is needed for the conversion of ammonia to urea in the liver, becoming urine. In the case of the first infant dosed, plasma ammonia levels were found to be maintained after treatment.
The study’s principal investigator Dr Julien Baruteau said: “For families who have struggled to manage their babies’ health while waiting for a liver transplant, currently the only long-term solution for those afflicted with the most severe forms of OTC deficiency, a solution that could restore OTC activity would be an incredibly exciting option.”
The results were announced as part of the 2025 American College of Medical Genetics and Genomics taking place in Los Angeles, with additional results finding the treatment to be generally safe and well tolerated. So far the OTC-Hope trial has recruited approximately 17 patients at seven sites across the US, UK, Australia and Spain.
Research by GlobalData’s Pharmaceutical Intelligence Center estimates that by the end of 2030, ECUR-506 is likely to bring in just $2m for the company. Within that same time frame, GlobalData estimates that the global OTC deficiency market will bring in $708m.
GlobalData is the parent company of Clinical Trials Arena.
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