Translate Bio is set to carry out a first-in-human clinical trial of MRT5005 to treat patients with cystic fibrosis (CF), following approval from the US Food and Drug Administration (FDA).
Prior to granting the approval, the FDA reviewed an Investigational New Drug (IND) application submitted by Translate Bio.
The randomised, placebo-controlled trial will enrol a minimum of 32 adult patients with CF who have at least one Class I or Class II mutation.
Its primary endpoint is the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulisation.
The trial will also measure forced expiratory volume in one second (FEV1) - a well-defined and accepted endpoint measuring lung function - at scheduled timepoints.
Translate Bio CEO Ronald Renaud said: “As our first IND submission and the first clinical trial to evaluate a product candidate derived from our mRNA technology (MRT) platform, this is a significant milestone for the company.
“It also represents a pivotal time in the mRNA field as it will be the first mRNA therapeutic to enter the clinic with targeted delivery to the lung.”
Cystic Fibrosis Foundation Therapeutics Development Network is also involved in the trial, which intends to begin dosing patients within this year.
CF is caused by genetic mutations that lead to dysfunctional or absent CFTR protein. It is estimated to be the most common fatal inherited disease in the US, affecting more than 30,000 patients in the country and 70,000 patients worldwide.
MRT5005 is a clinical-stage mRNA product candidate designed to address the primary cause of CF by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung epithelial cells through nebulisation.