iECURE

iECURE is a clinical-stage biotechnology company developing genome editing therapies for rare, life-threatening genetic diseases in children. The company's variant-agnostic approach uses in vivo targeted gene insertion to introduce a functional copy of a gene directly into a patient's genome, aiming to address the underlying cause of disease rather than manage symptoms. Founded on pioneering academic research at the University of Pennsylvania's Gene Therapy Program, iECURE's initial focus is on neurometabolic disorders such as ornithine transcarbamylase (OTC) deficiency, where genetic loss-of-function leads to toxic ammonia buildup and life-threatening consequences early in life.