MRT5005

MRT5005 is a first-in-class, inhaled mRNA-based therapy developed for the treatment of cystic fibrosis (CF). It consists of a codon-optimized messenger RNA (mRNA) encoding the human cystic fibrosis transmembrane conductance regulator (CFTR) protein, delivered to lung epithelial cells via aerosolized lipid nanoparticles or branched polyethyleneimine complexes. The therapeutic approach is genotype-agnostic, aiming to restore CFTR function regardless of the specific underlying CFTR gene mutation. Upon inhalation, MRT5005 delivers functional CFTR mRNA directly to bronchial epithelial cells in the lungs, enabling these cells to produce fully functional CFTR protein and thereby address the root cause of CF by restoring salt-water transport across lung surfaces. This prevents buildup of thick mucus characteristic of cystic fibrosis[1][2][4][5][6]. Clinical trials showed that MRT5005 was generally safe and well tolerated but did not demonstrate consistent improvements in lung function; development for this indication has been discontinued[1][3].

Other names

CO-hCFTR mRNAcodon-optimized human CFTR mRNACFTR mRNA therapeutic