QR-504a

QR-504a is a first-in-class investigational antisense oligonucleotide RNA therapy developed by ProQR Therapeutics for the treatment of Fuchs endothelial corneal dystrophy (FECD), specifically targeting cases caused by trinucleotide repeat (TNR) expansion mutations in the TCF4 gene. The drug is designed to modulate RNA splicing and utilize RNA interference mechanisms to reduce aggregation and formation of toxic nuclear RNA foci in corneal endothelial cells, aiming to prevent or slow down vision loss associated with FECD. It is administered via intravitreal injection. Development has reached Phase 1 clinical trials, but further development was suspended as of April 2022[1][4][5][6].