Translate Bio announced several updates relating to its two lead mRNA product candidates in development for the treatment of cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency. Cystic Fibrosis and MRT5005: targeted delivery to the lung A Phase 1/2 clinical trial of MRT5005, the Company’s lead mRNA product candidate, is currently ongoing. MRT5005 is designed to treat patients with CF by addressing the underlying cause of the disease regardless of the genetic mutation. The clinical trial consists of both a single-ascending dose (SAD) and multiple-ascending dose (MAD) part. The updates from this program are as follows: The Company received approval from the Protocol Review Safety Committee to begin dosing in the MAD portion of the trial. This approval followed a review of the initial safety and tolerability data from the first dose level of the SAD portion of the trial; The Company continues to dose patients in the SAD portion of the trial and anticipates first patient dosing in MAD to begin in early 2019; and The Company anticipates reporting interim data from this trial in the second half of 2019. OTC Deficiency and MRT5201: targeted delivery to the liverThe Company has advanced MRT5201, its second mRNA product candidate, towards the clinic. MRT5201 is designed to treat patients with OTC deficiency, the most common urea cycle disorder. The updates from this program are as follows: The Company submitted an Investigational New Drug (IND) Application to the U.S. Food and Drug Administration (FDA) to support the initiation of a Phase 1/2 clinical trial of MRT5201 in patients with OTC deficiency; and The Company anticipates initiating screening of patients with OTC deficiency for the Phase 1/2 clinical trial of MRT5201 in the first half of 2019.