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iECURE to Present Clinical Data from Ongoing OTC-HOPE Trial of ECUR-506 in Neonatal-Onset Ornithine Transcarbamylase Deficiency at SSIEM Annual Symposium

Business Wire

PHILADELPHIA, August 20, 2026--iECURE, Inc., a clinical-stage genome editing company developing variant-agnostic, in vivo targeted gene insertion therapies for severe inherited neurometabolic disorders, today announced that data from the ongoing OTC-HOPE clinical trial evaluating ECUR-506 for neonatal-onset ornithine transcarbamylase (OTC) deficiency will be featured in an oral presentation at the Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM), taking place August 25-28, 2026, in Helsinki, Finland.

The presentation will include updated safety observations from the ongoing OTC-HOPE study, and genome editing data on measures of metabolic control from the low-dose cohort. Additional analyses will explore the relationship between baseline disease severity and clinical response.

Annual Symposium of the Society for the Study of Inborn Errors of Metabolism

Oral Presentation: Decreased rate of hyperammonemic crises in infants with neonatal-onset OTC deficiency post ECUR-506 administration (O-124)

  • Session: Parallel abstract sessions E; E2: Urea cycle disorders

  • Date/Time: Thursday, August 27, 3:30 – 5:00 p.m. EEST

  • Location: Hall 5B

  • Presenter: Julien Baruteau, M.D., Ph.D., University College London Great Ormond Street Institute of Child Health

iECURE will also be exhibiting at SSIEM at Booth B16, August 25-28, at the Messukeskus Helsinki Expo and Convention Centre, where members of the medical team will be available to discuss the OTC-HOPE study and the company's genome editing platform.

About OTC Deficiency

Ornithine transcarbamylase (OTC) deficiency is a rare, serious genetic disorder caused by a defect in a liver enzyme responsible for removing ammonia from the bloodstream. As a result, ammonia, a waste product generated when the body breaks down protein, accumulates in the blood (hyperammonemia) to levels that are toxic to the brain.

The disease is characterized by recurrent and often unpredictable hyperammonemic crises (HACs), which can lead to hospitalization, irreversible neurological injury, and death. Newborns with neonatal-onset OTC deficiency typically present shortly after birth with symptoms such as lethargy, poor feeding, and vomiting, which can rapidly progress to seizures, coma, and life-threatening complications if not promptly treated.

Current management includes a strict protein-restricted diet and ammonia scavenger medications, which must be taken multiple times daily and often throughout a patient's life. While these approaches may help manage ammonia levels, they do not eliminate the risk of metabolic crises or ongoing disease burden, and patients remain vulnerable to acute decompensation and long-term complications.

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