iECURE: Clinical Trial for OTC
Who are iECURE?
iECURE is a biotechnology company based in the United States developing genome editing therapies for rare, life-threatening diseases in children. The company focuses on conditions caused by missing or non-functioning metabolic enzymes, including ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder.
iECURE’s clinical development program is supported by a multidisciplinary team with experience in inherited metabolic diseases, pediatric care, and gene-based medicines. This includes Dr. George Diaz, Vice President and Therapeutic Area Lead at iECURE, who has spent many years caring for children and adults with inherited metabolic disorders, including OTC deficiency.
What work is iECURE doing?
iECURE is developing ECUR-506, an investigational targeted gene insertion therapy for neonatal onset OTC deficiency, a severe form of the disease that typically presents in the first weeks of life and can lead to dangerous elevations of ammonia in the blood.
ECUR-506 is designed to insert a functional copy of the OTC gene into liver cells, enabling those cells to produce the OTC enzyme needed to help remove ammonia from the body. By restoring enzyme activity in the liver, this approach aims to improve metabolic function.
Targeted gene insertion is designed to allow the introduced gene to remain active as liver cells grow and divide, which may support durable enzyme production over time.
ECUR-506 is investigational and has not been approved for use outside of clinical studies.
Clinical Study Information
ECUR-506 is currently being evaluated in OTC-HOPE, an open-label, first-in-human clinical study designed to evaluate the safety and potential effects of ECUR-506 in male infants with genetically confirmed neonatal onset OTC deficiency.
The study is enrolling participants at clinical sites in the United States, United Kingdom, Spain, and Australia. For inquiries about the UK study sites, please contact Great Ormond Street Hospital (GOSH) in London (Christopher Jackson, Christopher.jackson@gosh.nhs.uk) or Great North Children’s Hospital in Newcastle (Dr. Mark Anderson, m.anderson10@nhs.net).
