iECURE: Clinical Trial for OTC

Who are iECURE?

iECURE, Inc. (iECURE) is a biotechnology company researching gene editing therapies for the treatment of rare and life-threatening disorders in children. Specifically, we are focused on developing gene editing therapies that address metabolic disorders in the liver such as urea cycle disorders, including Ornithine Transcarbamylase (OTC) deficiency. The technology and science behind our gene editing programs comes from a collaboration with the University of Pennsylvania’s Gene Therapy Program (GTP). Our most advanced program is focused on the development of a new gene editing therapy for neonatal onset OTC deficiency.   

iECURE has built a strong team of experts to help us achieve our goals. Our clinical development team for the OTC deficiency program is led by Dr. George Diaz. George joined iECURE after serving as the leader of the Program for Inherited Metabolic Diseases at the Icahn School of Medicine at Mount Sinai in New York City. George has been treating newborns and adults with inherited metabolic disorders for many years and has established a reputation as a go-to expert in urea cycle disorders like OTC deficiency. George has worked extensively on the clinical development and research of many different potential therapies for treating urea cycle disorders and was drawn to iECURE based on the potential of the gene editing technology.

What work is iECURE doing?

Gene editing is an investigational technology that targets the underlying cause of a disease at the cellular level. iECURE’s approach is designed to introduce a full, healthy copy of a gene responsible for the disorder of interest in a very precise spot in the DNA of targeted cells in order to restore the ability of these cells to produce the missing enzyme and regain normal function.

The hope is that the insertion of a healthy OTC gene will allow newborns with neonatal onset OTC deficiency to make healthy copies of the OTC enzyme, resolving the broken metabolic pathway that causes OTC deficiency.

iECURE uses a “knock-in” gene editing approach to introduce a healthy copy of the target gene into a person’s DNA. This is different from other gene editing approaches such as gene “knock-out” and “base editing” that seek to modify the existing genes in a person’s DNA. Gene therapy differs from gene editing in that with gene therapy a new functional gene is introduced into a person’s cells but the person’s DNA remains the same.


Trial locations

iECURE has received approval from regulatory authorities in the UK and Australia to begin a first-in-human clinical trial called OTC-HOPE for ECUR-506, an investigational gene editing therapy being developed by iECURE for the treatment of OTC deficiency in pediatric (or neonatal) patients.

OTC-HOPE is a Phase 1/2 first-in-human clinical research study to evaluate the safety and potential effect of ECUR-506 in baby boys with genetically confirmed OTC deficiency.

First study site in the United Kingdom (UK)
The first site is now open for recruitment at Great Ormond Street Hospital (GOSH).
If you’re interested in the study, please contact Christopher Jackson via

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