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ProQR to Present Findings from Phase 1/2 Interim Analysis of QR-421a for Usher Syndrome
LEIDEN, Netherlands & CAMBRIDGE, Mass., March 25, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives
About this update from Proqr Therapeutics N.v.
[{"type":"text","content":"LEIDEN, Netherlands & CAMBRIDGE, Mass., March 25, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced that Company management will present and discuss findings from a planned three-month interim analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa on March 31, 2020. The findings will be announced in a press release and management will host an investor conference call at 8:15 a.m. ET the same day to discuss the results in detail. \n The live and archived webcast of this presentation can be accessed through the Events and Presentations page on the Investors section of the Company’s website, www.ProQR.com. The dial-in details for the call are +1 631-510-7495 (US), +31 (0)20 714 3545 (NL), conference ID: 5986384. The archived webcasts will be available for approximately 30 days following the presentation date. About the Stellar Phase 1/2 Trial Stellar, or PQ-421a-001, is a first-in-human study of QR-421a in adults who have vision loss due to mutations in exon 13 of the USH2A gene and is conducted at expert sites in North America and Europe. It is a double-masked, randomized, 24-month study exploring the safety and efficacy of a single intravitreal injection of several dose levels of QR-421a and a control sham procedure into one eye. About QR-421a QR-421a is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional Usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the US and the European Union and received fast-track and rare pediatric disease designations from the FDA. About Usher Syndrome Type 2 and Non-Syndromic Retinitis Pigmentosa Usher syndrome is the leading cause of combined deafness and blindness. People with Usher syndrome type 2 are usually born with hearing loss and start to have progressive vision loss during adulthood. The...