For more information on the ongoing clinical trial in OTC deficiency please visit

Knocking Down Devastation.
Inserting Hope.

Patients with devastating genetic disorders require long-lasting, curative medicines. At iECURE, we believe in vivo gene editing is the answer to meeting this immense patient need, and we believe reversing the course of these conditions is now within reach.

A disruptive technology paired with an unrivaled partnership.

Our mutation-agnostic in vivo insertion methods knock in healthy copies of disease-causing genes, offering long-term, stable expression of those genes in patients. To accomplish this, we work hand-in-hand with the University of Pennsylvania’s Gene Therapy Program: a world-class translational engine that forms the backbone of our early R&D initiatives.

Realizing the promise of gene editing in the liver.

There are hundreds of rare, monogenic liver diseases affecting tens of thousands of patients across the world. As our team strives for cures, our programs are initially focused on “loss of function” liver disorders where an unhealthy gene may have many disease-driving mutations.

A team made up of genetic medicine experts.

Our management team has deep biotechnology experience in everything from rare disease to gene therapy. With our proven track record and our exclusive liver disease partnership with the University of Pennsylvania’s renowned Gene Therapy Program, we can now realize a new era of genetic medicine.

Read the latest updates from iECURE.

iECURE Announces FDA Clearance of Investigational New Drug Application for ECUR-506 to Initiate OTC-HOPE Trial for Treatment of Neonatal Onset Ornithine Transcarbamylase Deficiency in the U.S.