PHILADELPHIA, PA — iECURE, Inc. will present new clinical data on its experimental gene therapy candidate, ECUR-506, at two international medical meetings in Kyoto, Japan, this September. The therapy is being studied in infants with neonatal onset ornithine transcarbamylase (OTC) deficiency, a rare and often life-threatening genetic disorder that disrupts the body’s ability to process nitrogen.
The findings come from the company’s ongoing OTC-HOPE trial, the first clinical study to test a liver-directed, adeno-associated virus (AAV) mediated gene insertion therapy for this condition. Earlier this year, iECURE reported that the first infant dosed in the trial showed a complete clinical response, with no hyperammonemic events — episodes of dangerously high ammonia levels — during the 24-week study period.
At the 6th International Symposium on Urea Cycle Disorders, scheduled for September 1–2, iECURE will share details of this initial case. George Diaz, M.D., Ph.D., vice president and therapeutic area lead for UCDs at iECURE, will present the results in a poster session.
The company will also present updated findings at the 15th International Congress of Inborn Errors of Metabolism (ICIEM), held September 3–6. A poster highlighting results from the first four patients enrolled in the trial has been selected for the Best Poster Award presentation session on September 4.
OTC deficiency, caused by mutations in the OTC gene, prevents the body from properly eliminating ammonia, which can build up quickly to toxic levels. In infants with severe forms, the condition can be fatal without rapid intervention. Current treatments are limited to dietary management, medications, or liver transplantation, leaving an urgent need for new options.
By inserting a healthy copy of the OTC gene directly into liver cells, ECUR-506 aims to correct the underlying defect. The encouraging early results suggest the therapy may provide a long-term solution for infants who otherwise face a difficult prognosis.
The presentations in Kyoto will mark the first time international audiences see expanded data from the OTC-HOPE trial, signaling an important step forward in the development of gene therapies for rare metabolic disorders.
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