ProQR Therapeutics has dosed the first patient in a phase 1/2 trial of QR-1123, its treatment candidate for autosomal dominant retinitis pigmentosa, according to a press release.
The first-in-human AURORA trial, consisting of single-dose and multiple-dose escalation groups, will include up to 35 patients with autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin gene.
Patients will receive intravitreal injections of QR-1123, an investigational RNA-based oligonucleotide, or sham, with the trial evaluating safety and tolerability, as well as efficacy.
“QR-1123 aims to block expression of the toxic mutated rhodopsin protein in the retina, thereby targeting the underlying cause of the vision loss associated with [autosomal dominant retinitis pigmentosa] due to the P23H mutation,” David Rodman, MD, ProQR executive vice president of research and development, said in the release. “We are excited to have started a clinical trial for the third RNA therapy in our inherited retinal disease pipeline.”
Initial data from the trial are expected in 2021, the release said.