Benzinga | Jan 31, 2022 07:39AM EST

The Usher Syndrome Coalition Announces Partnership With ProQr To Support Clinical Trial Enrollment For A Potential Therapy For USH2A Mediated Retinitis Pigmentosa

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: www.proqr.com/community-stories-and-news/positive-results-achieved-in-our-ongoing-usher-syndrome-and-retinitis-pigmentosa-research.

ProQR and the Usher Syndrome Coalition will host a webinar on February 7th at 12:30 pm EST for those who are in the USH Trust registry. Individuals with Usher syndrome can easily join the USH Trust at https://www.usher-registry.org to make sure you are notified of the latest developments.

The Usher Syndrome Coalition's goal is to connect the global Usher community: with each other, with information and resources, and with companies & researchers working towards treatments. The Coalition is the only organization in the world working to find and support everyone living with Usher syndrome, of all ages and all types of USH.

The Coalition currently maintains contact with the Usher community throughout 71 countries and across all 50 states in the US through services like our USH Trust registry, our USH Ambassadors program, our USH Blue Book private email group, our annual USH Connections Conferences, and more.

Usher syndrome is the most common genetic cause of combined deafness and blindness. By partnering together, ProQR and the Coalition will aim to utilize the USH Trust, the largest international Usher syndrome registry.

ProQR Therapeutics seeks to recruit 200+ participants for Sirius and Celeste, two final-stage clinical trials of an investigational RNA therapy for USH2A mediated Usher syndrome and retinitis pigmentosa. ProQR is committed to patient-focused drug development and recognizes the challenge of finding individuals with USH2A mediated retinitis pigmentosa. The Usher Syndrome Coalition has identified and is engaged with hundreds of potential candidates located near the proposed trial sites. Through the USH Trust registry, the Coalition is uniquely positioned to collaborate with the clinical trial sites to identify and recruit participants for the Sirius and Celeste trials at an accelerated pace, as well as meet the varied accessibility needs of the Usher syndrome community.