Pipeline

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 MRTTM platform may also be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as infectious disease and oncology. Our lead mRNA product candidate is in development for the treatment of cystic fibrosis (CF). Beyond CF, the primary focus of our research efforts is the evaluation of targets in additional pulmonary diseases utilizing our proprietary lung delivery platform with other discovery efforts in diseases that affect the liver, eye and central nervous system.

Translate Bio Pipeline Chart

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 with data expected in 2020. The trial is being conducted in collaboration with the Cystic Fibrosis Therapeutics Development Network.

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 primary ciliary dyskinesia (PCD), pulmonary arterial hypertension (PAH) and idiopathic pulmonary fibrosis (IPF).

Discovery-Stage Programs

We have several discovery-stage programs to identify additional mRNA product candidates to treat diseases in the liver, eye, and CNS. In addition, we are working to develop mRNA vaccines for up to six infectious disease pathogens through our collaboration with Sanofi Pasteur; the sixth indication, for SARS-CoV-2, was added in 2020.

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