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.

QR-1123

Disease
RHO mediated retinitis pigmentosa
Discovered by Ionis

Program QR-1123 for RHO mediated retinitis pigmentosa is now in the phase 1/2, start phase

QR-1123 is an RNA therapy that aims to prevent vision loss or restore vision in retinitis pigmentosa (RP) due to the P23H mutation in the RHO gene.

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. 

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, recruiting

Aurora clinical trial logo

Aurora is a clinical trial of QR-1123 in the US. Adults that have autosomal dominant retinitis pigmentosa (adRP) due to the P23H mutation, also known as c.68C>A, in the RHO gene can participate. They will receive up to four intravitreal (into the eye) injections of QR-1123 or undergo a sham-procedure (simulated intravitreal procedure but no study medicine administered).

More about Aurora and participation

Designations granted to QR-1123 by authorities

  • FDA orphan drug designation
  • FDA fast track designation