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 two lead mRNA product candidates are in development for the treatment of cystic fibrosis (CF) and ornithine transcarbamylase, and discovery efforts are ongoing in diseases that affect the lung, liver, eye, central nervous system and lymphatic system.
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.
MRT5201 is designed to treat patients with OTC deficiency by intravenous delivery of mRNA encoding fully functional OTC enzyme to the liver to enable the hepatocytes, the predominant type of liver cell, to produce the normal OTC enzyme. MRT5201 has been granted orphan drug designation for the treatment of OTC deficiency in the U.S. and EU.
In addition to the programs above, we have several discovery-stage programs and are currently conducting lead identification activities to identify additional mRNA product candidates to treat diseases in the lung, liver, eye, CNS and lymphatic system.
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 it not available at this time. As clinical development progresses and more clinical data is available, Translate Bio will evaluate the possibility of expanded access.