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 pulmonary diseases portfolio includes a lead mRNA product candidate for the treatment of cystic fibrosis (CF), and ongoing research utilizing our proprietary lung delivery platform to address additional pulmonary disease targets. 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 oncology.

Translate Bio 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. We are conducting 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. 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 with data expected in the 2nd quarter of 2021. The trial is being conducted in collaboration with the Cystic Fibrosis Therapeutics Development Network. The FDA granted orphan drug and fast track designation to MRT5005 for the treatment of CF.

Next-Generation CFTR

Our next-generation CFTR mRNA preclinical candidate applies advances in mRNA science, lipid nanoparticle (LNP) chemistry and cystic fibrosis transmembrane conductance regulator (CFTR) biology. Our goal is to design a second CF product candidate with novel mRNA sequences encoding wild-type and rationally engineered CFTR protein. Preclinical studies are ongoing to select the optimal mRNA sequence and LNP for a next-geneneration CFTR candidate.

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 produced multiple mRNA constructs and are using our mRNA platform to discover, design and manufacture SARS-CoV-2 vaccine candidates. We have established 100 gram single-batch production with its clinical-stage mRNA therapeutics platform. Build-out is underway of dedicated manufacturing space through a contract manufacturing partner to accommodate at least two 250 gram batches per month. 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