Our proprietary MRTTM platform has been designed with the potential to apply across a broad array of diseases and target tissues and through multiple routes of administration. The following chart summarizes key information about our two lead product candidates and programs:
We believe MRT5005 will be the first clinical-stage mRNA product candidate designed to deliver mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung. MRT5005 is being developed to treat all patients with CF, regardless of the underlying genetic mutation. In 2015, the FDA granted orphan drug designation to MRT5005 for the treatment of CF. We have initiated a double-blind, placebo-controlled Phase 1/2 clinical trial of MRT5005 in which we plan to enroll at least 32 patients with CF.
We are developing MRT5201 to treat patients with OTC deficiency. We have developed MRT5201 for intravenous administration and delivery of mRNA encoding fully functional OTC enzyme to the liver to enable the hepatocytes to produce the normal OTC enzyme. We anticipate initiating a Phase 1/2 clinical trial of MRT5201 in patients with OTC deficiency in the first half of 2019.
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 medicines 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.