First patient dosed in ProQR retinitis pigmentosa trial

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.

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.