QRX-411

QRX-411 is a first-in-class investigational RNA-based oligonucleotide designed for the treatment of Usher syndrome type II caused by the PE-40 (pseudo-exon 40; c.7595-2144A>G) mutation in the USH2A gene. The drug acts by binding to the mutated pre-mRNA, correcting aberrant splicing, and restoring wild-type USH2A mRNA, which leads to the production of functional USH2A protein necessary for normal vision. The mechanism directly targets the underlying genetic defect responsible for combined deafness and blindness in affected patients. QRX-411 has demonstrated efficacy in preclinical models, including patient-derived fibroblasts and optic cup organoids, restoring the production of wild-type RNA and protein. The program has received Orphan Drug Designation from both the FDA and EMA for retinitis pigmentosa, including Usher syndrome. Developed as part of ProQR's ophthalmology pipeline, QRX-411 is in preclinical/IND-enabling development and addresses a currently unmet medical need, as there are no approved therapies for this indication[1][3][4][5][10][15][17][21].

Other names

QRX-411QRX411QRX 411