The FDA has granted fast track designation to QR-1123, an investigational antisense oligonucleotide, ProQR Therapeutics announced in a press release.
QR-1123, designed to treat the underlying causes of vision loss associated with autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin gene, inhibits the formation of the mutated toxic version of the rhodopsin protein by binding the RHO mRNA.
“We are very pleased to receive fast track designation by the FDA for QR-1123. There are no current treatment options available for patients to improve vision or prevent vision loss due to [autosomal dominant retinitis pigmentosa]. Further, this designation emphasizes the high unmet need in this disease,” Daniel de Boer, CEO of ProQR, said in the release. “We look forward to beginning enrollment in the phase 1/2 (Aurora) clinical trial for QR-1123 in the coming months.”