Aerosolized Therapy Restores CFTR in Patient-derived Cells

ReCode Therapeutics’ investigational aerosolized RNA-based therapies for cystic fibrosis (CF) restored CFTR function, the defective protein in CF, in patient-derived lung cells. Delivered by the company’s selective organ targeting (SORT) lipid nanoparticle (LNP) platform, the therapy showed no signs of toxicity in epithelial cells that line the airways.

RNA-based Therapies Show Promise in Early Studies, ReCode Reports

RCT223 and RTX0001, ReCode Therapeutics’ experimental RNA-based therapies for cystic fibrosis (CF), safely restored function to CFTR, the faulty protein in CF, in patient-derived lung cells. Delivered through the company’s non-viral platform — called the selective organ targeting (SORT) lipid nanoparticle (LNP) platform — the therapies were also…