Investigational RNA therapy QR-1123 for RHO mediated Retinitis Pigmentosa

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

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.

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

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 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