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 (MRTTM), 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 MRTTM platform has been in development for over ten years, initially at Shire and at Translate since 2016. With the scientific founders of the MRTTM platform 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 are primarily focused on applying our MRT platform to pulmonary diseases caused by insufficient protein production or where production of proteins can modify disease. Our lead program is being developed as a treatment for cystic fibrosis (CF) and is in an ongoing Phase 1/2 clinical trial. We also believe our technology is applicable to a broad range of diseases, including diseases that affect the liver, eye and central nervous system. Additionally, the MRT platform may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as infectious disease and oncology; in the case of the latter, we have a collaboration with Sanofi Pasteur to develop a novel mRNA vaccine for SARS-CoV-2, the novel coronavirus responsible for the disease known as COVID-19.