It uses its in vivo gene insertion technology to deliver a healthy copy of the defective gene, independent of the underlying mutations.
US-based iECURE is developing gene-editing therapies to treat rare genetic liver diseases in children. It uses its in vivo gene insertion technology to deliver a healthy copy of the defective gene, independent of the underlying mutations. Its pipeline includes treatments for conditions like ornithine transcarbamylase deficiency, citrullinemia type 1, and phenylketonuria.