![]() ![]() This press release contains forward-looking statements. Learn more about ProQR at Forward Looking Statements ![]() Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome and retinitis pigmentosa. 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. 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 a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene. To date, there are no pharmaceutical treatments approved or in clinical development that treat the vision loss associated with mutations in USH2A. Usher syndrome type 2a and non-syndromic retinitis pigmentosa can be caused by mutations in the USH2A gene. The vision loss can also occur without hearing loss in a disease called non-syndromic retinitis pigmentosa. People with Usher syndrome type 2a are usually born with hearing loss and start to have progressive vision loss during adulthood. Usher syndrome is the leading cause of combined deafness and blindness. The majority of the patients were followed for up to 48 weeks, with one patient followed up to 96 weeks.Ībout Usher Syndrome Type 2a and Non-Syndromic Retinitis Pigmentosa The population also varied in disease characteristics with both Usher syndrome (n=7) and nsRP (n=7) and genetic background with both homozygous (n= 9) and heterozygous (n=5) subjects for USH2A exon 13 mutations. Three different dose levels were studied. Six patients had advanced disease and eight patients had early-moderate disease. The 14 treatment patients enrolled varied in their disease stage and were classified as advanced patients (defined as patients with baseline visual acuity of <70 letters or equivalent to worse than 20/40 on a Snellen chart) or early-moderate patients. The study includes a total of 20 patients, of which 14 received a single dose of QR-421a and six received a single sham procedure for masking. The Stellar trial is a randomized, single ascending dose, global, multicenter, 24-month study. The archived webcast will be available for approximately 30 days following the presentation date. The live and archived webcast of this presentation will be accessible through the Events page of the “Investors and Media” section of the Company’s website, The dial-in details for the call are +1 63 (US), +31 (0) (NL), conference ID: 8596733. ![]() EDT the same day to discuss the results in detail. The data will be announced in a press release and management will host an investor conference call at 8:15 a.m. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today announced that Company management will present and discuss results from its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) on March 24, 2021. LEIDEN, Netherlands & CAMBRIDGE, Mass., Ma(GLOBE NEWSWIRE) - ProQR Therapeutics N.V. ![]()
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