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ProQR seeking Clinical Trial Participants

The Usher Syndrome Coalition announces partnership with ProQR to support clinical trial enrollment for a potential therapy for USH2A mediated retinitis pigmentosa.

WESTFORD, Mass., Jan. 31, 2022 /PRNewswire/ -- For more than ten years, the Usher Syndrome Coalition has been working to build its international "USH Trust" registry in anticipation of the day when researchers would launch a final-stage clinical trial for the Usher syndrome community. That time has come. The Coalition is pleased to partner with ProQR to help recruit 200+ participants worldwide for this exciting study.

Clinical trials Sirius and Celeste aim to determine whether the investigational RNA therapy QR-421a is effective at stopping vision loss and is safe for people with retinitis pigmentosa and Usher syndrome due to mutations in exon 13 of the USH2A gene. These Phase 2/3 clinical trials have been planned after positive findings in a Phase 1/2 clinical trial named Stellar. More information on Stellar can be found here:


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