Copernicus CFTR DNA nanoparticles

Copernicus Therapeutics is developing a non-viral gene therapy for the treatment of cystic fibrosis. The candidate utilizes the company's proprietary nucleic acid nanoparticle (NAP) platform, which involves the compaction of plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator (CFTR) gene. These nanoparticles are formed using polyethylene glycol (PEG)-conjugated poly-L-lysine (specifically CK30, a 30-mer lysine peptide), resulting in stable, unimolecular DNA structures. This non-viral approach is designed to overcome the limitations of viral vectors, such as immunogenicity and cargo size constraints, potentially allowing for repeated aerosolized administration. The therapy aims to deliver functional CFTR genes to the airway epithelium to restore chloride ion transport and improve pulmonary function in patients regardless of their specific CFTR mutation.

Other names

Copernicus cystic fibrosis gene therapyCFTR gene therapy (Copernicus)