At Translate Bio, we are focused on developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction. Using our novel mRNA therapeutic platform (MRT), we create mRNA that encodes functional proteins. When our mRNA is delivered to the target cell, the cell's own machinery recognizes it and translates it, restoring or augmenting protein function to treat or prevent disease. We believe that our MRT platform is applicable to a broad range of diseases caused by insufficient protein production or where production of proteins can modify disease. Our two lead programs are being developed as treatments for cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency.