Benzinga | Dec 16, 2021 07:04AM EST

ProQR Announces First Patients Dosed In Phase 2/3 Pivotal Trials Of QR-421a For USH2A Mediated Retinitis Pigmentosa

ProQR Therapeutics N.V. (NASDAQ:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced the first patients dosed in Phase 2/3 Sirius and Celeste clinical trials of investigational RNA therapy QR-421a for people with USH2A mediated retinitis pigmentosa (RP) and Usher syndrome.

"Retinitis pigmentosa (RP) and Usher syndrome associated with USH2A are inherited retinal diseases that can lead to blindness because there is no treatment available to stop the progressive vision loss", said David Birch, Ph.D., investigator in the Sirius and Celeste studies at the Retina Foundation of the Southwest in Dallas, Texas, U.S. "These are the first, potentially pivotal trials for these conditions, that we see as a promising milestone that brings hope to the RP and Usher syndrome communities."

"We are pleased to bring our second RNA therapy into potentially the final stage of clinical testing," said Aniz Girach, M.D., Chief Medical Officer of ProQR. "In a previous clinical study, QR-421a appeared to be well tolerated and demonstrated concordant benefit in multiple measures of vision in treated eyes compared to untreated. Our goal is that the Sirius and Celeste studies further validate QR-421a's ability to stabilize vision loss in people with USH2A mediated retinitis pigmentosa and Usher syndrome."