We are a leading mRNA therapeutics company developing a new class of potentially transformative medicines to treat, or prevent, debilitating or life-threatening diseases. 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 diseases caused by protein or gene dysfunction, or prevent disease by generating protective immunity.
Our MRTTM platform has been in development for more than a decade, initially at Shire and since 2016 at Translate Bio. 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 transformative mRNA medicines to patients.
We are primarily focused on applying our MRTTM platform to treat pulmonary diseases caused by insufficient protein production or where the production of proteins can modify disease. Our clinical-stage pulmonary product candidate, MRT5005, is being evaluated as an inhaled treatment for cystic fibrosis (CF) in an ongoing Phase 1/2 clinical trial. We also have a next-generation CF discovery program that we are advancing towards investigational new drug (IND)-enabling studies. Additional pulmonary diseases are being evaluated in discovery-stage research programs that utilize our proprietary lung delivery platform.
We believe that mRNA can be delivered to target tissues via multiple routes of administration and that our technology may apply to a broad range of diseases beyond pulmonary diseases. We are pursuing an innovative alternative to conventional vaccines under a collaboration with Sanofi Pasteur — the development of mRNA vaccines for multiple infectious diseases. In addition, we are pursuing discovery efforts in diseases that affect the liver, and our platform may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as oncology.