RCT2100

RCT2100 is an investigational inhaled mRNA-based therapy developed for the treatment of cystic fibrosis (CF), particularly targeting the 10–13% of patients with Class I or nonsense mutations in the CFTR gene who do not respond to currently approved CFTR modulator therapies. Unlike existing treatments that attempt to correct or enhance faulty CFTR proteins, RCT2100 delivers healthy CFTR mRNA directly to lung cells using a proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform. This approach instructs lung cells to produce new, fully functional CFTR protein, aiming to address the underlying cause of cystic fibrosis by restoring proper protein function rather than just managing symptoms. The drug is being developed by ReCode Therapeutics and has received Orphan Drug Designation from the U.S. FDA. It is currently in Phase 1 clinical trials for cystic fibrosis[1][2][3][5][6][7].