Penn gene therapy spinout iEcure, co-founded by Jim Wilson, MD, PhD, the Rose H. Weiss Orphan Disease Center Director’s Professor, has gained FDA clearance to conduct the first-ever test of a gene editing therapy aiming to treat ornithine transcarbamylase deficiency, a severe metabolic condition. iECURE’s approach involves directly editing genes of newborns and infants, with the aim of targeting the root cause of inherited diseases before severe complications arise. Utilizing ARCUS, a gene editing tool developed by Precision BioSciences, the therapy aims to provide a lifelong fix for this disease, potentially benefiting thousands of individuals in the US alone. Read more here: