Knocking Down Devastation.
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.
European Commission Grants Orphan Designation for iECURE’s Lead Product Candidate GTP-506 for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency/in news, press release/by Ellie Solorio
iECURE Appoints Gabriel M. Cohn, M.D., as Chief Medical Officer/in news, press release/by Ellie Solorio
iECURE Raises $65 Million to Advance In Vivo Gene Editing Programs for the Treatment of Rare Pediatric Liver Diseases/in news, press release/by Ellie Solorio