eluforsen

Eluforsen (formerly QR-010) is an experimental antisense oligonucleotide developed by ProQR Therapeutics for the treatment of cystic fibrosis, specifically targeting patients with the F508del mutation in the CFTR gene. It is a single-stranded, 33-nucleotide RNA-based molecule designed to bind to the mRNA region around the F508del mutation and restore normal function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in airway epithelial cells. By binding to this specific region, eluforsen aims to correct defective CFTR protein production and improve chloride ion transport across cell membranes, thereby addressing one of the root causes of cystic fibrosis in affected individuals. Eluforsen is administered via inhalation and has shown safety and some clinical benefit in early-phase clinical trials[1][3][4][6].

Other names

eluforsenQR-010QR010QR 010