Present and future focus

Our MRTTM platform is potentially applicable to a broad range of diseases caused by insufficient protein production or where production of proteins can modify disease. Our lead mRNA product candidate is in clinical development for the treatment of cystic fibrosis (CF). The primary focus of our research efforts is the evaluation of targets in pulmonary diseases utilizing our proprietary lung delivery platform with other discovery efforts in diseases that affect the lungs and liver. We are also pursuing the development of mRNA vaccines for infectious diseases under a collaboration with Sanofi Pasteur. Our MRTTM platform may also be applied to various classes of treatments, such as therapeutic antibodies.

TBio pipeline

About MRT5005

MRT5005 is the first clinical-stage mRNA product candidate designed to address the underlying cause of CF by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung epithelial cells through nebulization. MRT5005 is being developed to treat all patients with CF, regardless of the underlying genetic mutation, including those with limited or no CFTR protein. In 2015, the FDA granted orphan drug designation to MRT5005 for the treatment of CF. In 2018, we initiated a first-in-human, randomized, double-blind, placebo-controlled clinical trial designed to evaluate the safety and tolerability of single and multiple escalating doses of MRT5005. In July 2019, we announced interim results summarizing the single ascending dose (SAD) portion of this clinical trial. The multiple ascending dose (MAD) portion of the trial is ongoing. The trial is being conducted in collaboration with the Cystic Fibrosis Therapeutics Development Network.

Infectious Disease Vaccines

We are currently working with our partner Sanofi Pasteur to develop mRNA vaccines for infectious disease pathogens including SARS-CoV-2, the novel coronavirus responsible for the disease known as COVID-19. We have begun to produce multiple mRNA constructs and will use our mRNA platform to discover, design and manufacture SARS-CoV-2 vaccine candidates. We have established 100-gram single batch production with our clinical-stage mRNA therapeutics platform. We have dedicated manufacturing space through a contract manufacturing partner with the potential to accommodate 250-gram single batch mRNA production, subject to continued investment and timing of scale up activities. Depending on the final human dose, the mRNA platform of Translate Bio has excellent promise to meet the future demands for a pandemic response. We anticipate entering the clinic with our COVID-19 vaccine candidate in the first quarter of 2021.

Additional Pulmonary Programs

Beyond our CF clinical program, the primary focus of our research efforts is the evaluation of targets in additional pulmonary diseases utilizing our proprietary lung delivery platform. Preclinical research efforts include discovery-stage programs in CF, primary ciliary dyskinesia (PCD), pulmonary arterial hypertension (PAH) and idiopathic pulmonary fibrosis (IPF).

Expanded Access

Translate Bio is committed to developing a new class of medicines to treat diseases caused by protein or gene dysfunction. Our pipeline includes MRT5005, a phase 1/2 product candidate designed to deliver mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung. Prior to regulatory approval, Translate Bio believes the optimal way to gain access to an investigational medicine is to participate in an ongoing clinical trial. For information about our clinical trials, please consult www.clinicaltrials.gov. Expanded access is not available at this time. As clinical development progresses and more clinical data is available, Translate Bio will evaluate the possibility of expanded access.

Areas of Impact

Changing the course of disease