Investigational RNA therapy QR-1123 for RHO mediated Retinitis Pigmentosa

Close up female eyes

QR-1123 is an investigational RNA therapy that aims to stop vision loss or restore vision in people with retinitis pigmentosa, or RP, due to the P23H mutation, also known as c.68C>A, in the rhodopsin (RHO) gene. This mutation causes an autosomal dominant form of RP.

How does QR-1123 work?

QR-1123 aims to prevent vision loss or restore vision in people with retinitis pigmentosa due to the P23H mutation in the RHO gene. This mutation causes a mistake in the RHO RNA and the production of mutated rhodopsin protein. Over time, the mutated protein blocks the function of the normal rhodopsin protein and eventually becomes toxic to the retina.

QR-1123 works by binding to the mutated RHO RNA so that it is removed by the cell through a mechanism called RNase H mediated cleavage. Once the mutated version of the RNA is removed, the normal version of the protein can perform its function in the retina again. 

Genetic testing

To know what mutation is causing RP, genetic testing is necessary. Genetic testing is the only way to receive an accurate diagnosis and will help find out if there are treatments or clinical trials available.

Internal link Learn about genetic testing
Female scientist in the lab

QR-1123 clinical trials

We are conducting a Phase 1/2 clinical trial of QR-1123, to test whether it is safe and leads to better vision.

Learn more about clinical trials and why you should consider participating.

Aurora study - Phase 1/2 clinical trial

Ongoing, not recruiting

Aurora clinical trial logo

Aurora is a clinical trial of QR-1123 in the US. Eleven adults that have autosomal dominant retinitis pigmentosa (adRP) due to the P23H mutation participated in the study. They received QR-1123 through an intravitreal (into the eye) injection.

Clinical trial results

Results from Aurora suggested the single injection with QR-1123 was well tolerated with a manageable safety profile. The Aurora study also showed signs of target engagement.

Based on these positive findings we plan to proceed with a Phase 2 repeated dose clinical trial.

More about Aurora

Designations granted to QR-1123 by authorities

  • FDA orphan drug designation
  • FDA fast track designation

Latest QR-1123 press releases

Internal link More press releases
Female scientist working in ProQR's laboratories

You might like this

  • Meet Maartje - living with vision loss

    At a glance, it’s hard to tell Maartje de Kok – 41, married and mother of four – has Usher syndrome. Though she manages her life very well, it took her a long time to get to that point. “I have learned to be optimistic, but the somberness is always there.”

    Close-up of Maartje, living with Usher syndrome

  • All about retinitis pigmentosa

    RP describes a group of inherited retinal diseases (IRDs) affecting the photoreceptor (light-sensing) cells which line the back of the eye, called the retina. People living with RP experience a gradual decline in their vision because the two photoreceptor cell types – rods and cones – degenerate.

    An ophthalmologist performing an eye exam.

  • Scientific presentations and publications

    Find all relevant presentations and publications for each of our programs. From review papers to preclinical posters and clinical data presentations at medical conferences.

    Two ProQR scientists working in the lab