Translate Bio - mRNA der Zukunft?

-- 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."