Pipeline

Present and future focus

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:

About MRT5005

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.

About MRT5201

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.

Discovery-Stage Programs

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.

Areas of Impact

Changing the course of disease