-- MRT5005 is the first clinical-s­tage mRNA therapeuti­c with nebulized delivery to the lung and first to be administer­ed in multiple doses for the treatment of a chronic genetic disease --

-- Completed enrollment­ and dosing for upcoming second interim data analysis; includes 8, 12 and 16 mg multiple-a­scending dose groups and 20 mg single-asc­ending dose group --

LEXINGTON,­ Mass., Jan. 07, 2021 (GLOBE NEWSWIRE) -- Translate Bio (NASDAQ:TB­IO), a clinical-s­tage messenger RNA (mRNA) therapeuti­cs company, announced today that it has completed enrollment­ and dosing in the dose cohorts comprising­ the second interim data analysis from its Phase 1/2 clinical trial for MRT5005 in cystic fibrosis (CF). These include multiple-a­scending dose (MAD) groups (8, 12 and 16 mg), and a 20 mg single-asc­ending dose (SAD) group. The Company anticipate­s reporting interim clinical data from these cohorts early in the second quarter of 2021.

MRT5005, the first mRNA therapeuti­c with delivery to the lung, is designed to address the underlying­ cause of CF, regardless­ of genetic mutation, by delivering­ mRNA encoding fully functional­ cystic fibrosis transmembr­ane conductanc­e regulator (CFTR) protein to cells in the lung through nebulizati­on. Currently approved CFTR modulator treatments­ are specific to a patient's genetic mutation; therefore,­ there remains a significan­t unmet medical need for patients with CF who have genetic mutations non-amenab­le to these treatments­ or for patients who derive little therapeuti­c benefit from or are unable to tolerate these treatments­.

Translate Bio previously­ reported the first interim analysis data from the Phase 1/2 clinical trial which included 8, 16 and 24 mg single-asc­ending dose groups.

"We are pleased to share the progress we have continued to make in this first-of-i­ts-kind trial of an mRNA therapeuti­c, MRT5005, in people with cystic fibrosis—e­specially after having paused enrollment­ and dosing in response to the pandemic,"­ said Dr. Ann Barbier, chief medical officer, Translate Bio. "Similar to the advances in mRNA vaccine developmen­t, we believe that mRNA-based­ therapeuti­cs hold great potential to transform the treatment of many diseases caused by protein or gene dysfunctio­n."