iECURE

We are focused on using mutation-agnostic in vivo gene insertion. We are focusing our efforts initially on gene editing for liver disorders, especially in children. We are currently developing gene editing approaches to treat ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder in humans, citrullinemia type 1 (CTLN1), a second urea cycle disorder, and phenylketonuria (PKU), a metabolic disorder caused by a loss of function in the enzyme that metabolizes the amino acid phenylalanine. 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. In contrast, some approaches focus on altering the specific bases in a gene that drive disease, creating a need for a resource-intensive, near-custom approach to diseases where dozens if not hundreds of mutations can cause a disease. This enzyme cuts the genome at the well-characterized PCSK9 site, which serves as the insertion site for the healthy copy of the gene. The GTP is comprised of more than 300 experts in vector engineering, discovery, early-stage development, gene therapy and gene editing manufacturing. The GTP’s expertise forms the backbone of early R&D for iECURE, with the GTP’s team responsible for executing all preclinical development work.