We are a leading mRNA therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction. Using our proprietary mRNA therapeutic platform (MRT), we create mRNA that encodes functional proteins. Our mRNA is delivered to the target cell where the cell’s own machinery recognizes it and translates it, restoring or augmenting protein function to treat or prevent disease.
Our MRT platform has been in development for the past ten years, initially at Shire and internally at Translate since 2016. With the scientific founders of MRT now part of our leadership team, we have built on Shire’s initial pioneering work and investment to advance our goal of bringing our transformative mRNA approach to patients.
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, including diseases that affect the lung, liver, eye, central nervous system, lymphatic system and circulatory system. Our two lead programs are being developed as treatments for cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency.