ProQR Therapeutics NV, a renowned Dutch biopharmaceutical company dedicated on finding treatments for rare genetic diseases, has reportedly announced the dosage of first patient in Phase 1/2 Aurora study of QR-1123.

For the record, QR-1123 is a first of its kind investigational RNA-based oligonucleotide developed for the treatment of autosomal dominant retinitis pigmentosa (adRP). It was first discovered and developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology.

According to reports, the Aurora trial would initially have up to 35 adults diagnosed with adRP due to P23H in the RHO (rhodopsin) gene. The study would include multiple-dose escalation groups (double-masked) and single-dose escalation groups (open-label) in which one eye would receive intravitreal injections of the QR-1123 or sham processes.

The goals of the study includes tolerability and safety evaluation. Efficacy as calculated by enhancement of retinal structure and visual function would be assessed by ophthalmic endpoints like optical coherence tomography, visual acuity and visual field. This trial would be conducted in expert sites in North America.    

David G. Birch, Ph.D., Scientific Director of the Retina Foundation of the Southwest in Dallas, Texas and Principal Investigator of Aurora, said that, from the previous clinical study, the company has seen that RNA treatments can be a good approach for patients diagnosed with inherited retinal diseases. There is a high demand for new approaches to diseases that presently don’t have any treatments.

Birch added that QR-1123 has showcased encouraging outcomes in preclinical models and the company hopes to see that replicated to the clinic during this first-in-human study in patients suffering from adRP.

As per ProQR’s Executive Vice President of Research & Development, David Rodman, QR-1123 aims to stop the expression of toxic mutated rhodopsin protein present inside the retina. This would further target the underlying cause of vision loss which is associated with adRP owing to P23H mutation. The company is thrilled to have initiated a clinical study for third RNA treatment in its inherited retinal disease pipeline.    
 

Source Credit: https://ir.proqr.com/news-releases/news-release-details/proqr-announces-first-patient-dosed-phase-12-aurora-trial-qr