Tackling Cystic Fibrosis from Multiple Angles
CF is a progressive, fatal genetic disease which, if left untreated, can cause persistent lung infections and respiratory failure.
CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and impacts approximately 130,000 people worldwide.
Despite advancements in CFTR modulator treatments, no available therapies exist to restore function for Class I mutations in the CFTR gene since those mutations result in no CFTR protein being produced that can be modulated.
“Our inhaled CFTR mRNA therapy is optimized to reach the correct cells in the lungs and instruct them to make a functional version of the CFTR protein. This would represent an exciting step in developing a successful therapy for all CF patients.”
John G. Matthews, MBBS, MRCP, Ph.D.
Senior Vice President, Clinical DevelopmentChanging the landscape of treatment options for CF
Using our SORT LNP platform, ReCode is targeting the underlying cause of the disease so that more people with CF may benefit, including the 10-13 percent of people with CF who have Class 1 mutations.
ReCode’s CFTR mRNA (RCT2100) is a clinical-stage candidate in development to treat the 10-13 percent of CF patients who have Class I mutations in the CFTR gene and do not respond to currently approved CFTR modulators.
Formulated using ReCode’s proprietary SORT LNP delivery platform, RCT2100 is an inhaled therapy designed to deliver CFTR mRNA to target cells and instruct them to make a functional version of the CFTR protein.