Inhaled Messenger RNA Treatment Fast-Tracked for Cystic Fibrosis

The Food and Drug Administration has granted Fast Track designation to MRT5005 (Translate Bio), an inhaled messenger RNA (mRNA) therapeutic being investigated for the treatment of cystic fibrosis.

MRT5005 is designed to address the underlying cause of cystic fibrosis, regardless of genetic mutation, by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung epithelial cells through nebulization. A phase 1/2 trial investigating the safety and tolerability of single and multiple escalating doses of MRT5005 is currently ongoing.

Interim results from 12 patients showed that the treatment was generally well tolerated with cough and headache observed as the most common adverse events. In addition, marked increases in percent predicted forced expiratory volume in one second (ppFEV1) were noted after a single dose of MRT5005.

“MRT5005 has the potential to treat all people with CF, including those with mutations that result in limited to no CFTR protein production,” said Dr Ann Barbier, chief medical officer, Translate Bio. “The Fast Track designation will help Translate Bio to expedite the clinical development of this potentially transformative therapeutic.”