We’re here to change patients’ lives.
Also, here to change genetic medicine as we know it today.
At iECURE, we believe permanently correcting devastating, “loss of function” diseases is now possible. This is why we are pursuing mutation-agnostic in vivo gene insertion approaches to knock healthy genes into patients’ chromosomes, allowing the expression of a missing gene which may eliminate symptoms and extend life.
We were founded to advance the life’s work of James M. Wilson, M.D., Ph.D., Rose H. Weiss professor and director, Orphan Disease Center; professor in Departments of Medicine and Pediatrics, Perelman School of Medicine; and director of the Gene Therapy Program (GTP) at the University of Pennsylvania.
More than 35 years ago, Dr. Wilson was studying ornithine transcarbamylase (OTC) deficiency: a urea cycle disorder and inborn error of metabolism that often presents in newborns, causes toxic ammonia buildup and leads to death in early childhood. Dr. Wilson hypothesized that if science had a way to permanently supply a healthy copy of the gene that codes for OTC, the disorder could be cured, yet technical challenges historically stymied this vision.
New gene editing technologies hold the promise of first making a cut in genomic DNA, and then inserting a healthy copy of the disease-causing gene into the chromosome. While this approach is applicable in a broad range of tissues, our team is starting with an initial focus on liver disorders.
iECURE has established a foundational collaboration with the GTP and their 300+ experts in vector engineering, discovery, early-stage development gene therapy and gene editing manufacturing. This expertise is the backbone of our early R&D, and we are poised to assume responsibility from the GTP and advance this vision of mutation-agnostic in vivo gene insertion into the clinic and beyond.
Our team is comprised of seasoned experts in biotechnology, gene therapy and liver disorders who have extensive experience in building successful companies.