Translate Bio was formed in 2017 when a group of researchers joined forces with RaNA Therapeutics, and its initial focus is on developing treatments for cystic fibrosis and ornithine transcarbamylase deficiency.

The company took a step forward in April 2018 when the US Food and Drug Administration cleared it to begin a first-in-human clinical trial of MRT5005 in patients with cystic fibrosis, the agency having previously requested more detailed information for the drug related to chemistry, manufacturing and controls (CMC), delaying the green light for a few months. Translate Bio now says it intends to start dosing patients in a Phase I/II clinical trial in mid-2018.