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 MRT5005, a clinical-stage mRNA product candidate for the treatment of cystic fibrosis (CF), a next-generation CF discovery program, 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.
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. RESTORE-CF is a first-in-human, randomized, double-blind, placebo-controlled clinical trial designed to evaluate the safety and tolerability of single and multiple ascending doses of MRT5005. We have announced results from two interim analyses summarizing the single ascending dose (SAD) and multiple ascending dose (MAD) portions of this clinical trial. Clinical results reported to date suggest that MRT5005 delivered in multiple doses to the lungs of CF patients is generally safe and well tolerated. The trial is being conducted in collaboration with the Cystic Fibrosis Therapeutics Development Network and the Emily’s Entourage Patient Registry. The FDA granted orphan drug and fast track designation to MRT5005 for the treatment of CF.
Our next-generation CF 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-generation CF 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 COVID-19. We have produced multiple mRNA constructs and are using our mRNA platform to discover, design and manufacture SARS-CoV-2 vaccine candidates. In March 2021, we and our partner Sanofi Pasteur announced the start of a Phase 1/2 clinical trial for MRT5500, an mRNA COVID-19 vaccine candidate. We expect interim results from this trial in the third 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) and pulmonary arterial hypertension (PAH).
Translate Bio is committed to developing a new class of medicines to treat diseases caused by protein or gene dysfunction. 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 trial, please consult www.clinicaltrials.gov. Expanded access is not available at this time.