Sirius and Celeste are the names of our two Phase 2/3 studies of ultevursen (previously named QR-421a) an experimental RNA therapy in people with USH2A associated retinitis pigmentosa (RP) and Usher syndrome. We have advanced to the final phase of clinical development following the positive readout of our Phase 1/2 Stellar in March 2021.
Usher syndrome is the leading cause of combined deafness and blindness. People with USH2A mediated Usher syndrome are usually born with hearing loss and start to have progressive vision loss during adulthood. The vision loss can also occur without hearing loss in a disease called retinitis pigmentosa, or RP.