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ProQR Announces Virtual Presentations at American Academy of Ophthalmology (AAO)
LEIDEN, Netherlands & CAMBRIDGE, Mass., Nov. 02, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through
About this update from Proqr Therapeutics N.v.
[{"type":"text","content":"LEIDEN, Netherlands & CAMBRIDGE, Mass., Nov. 02, 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 virtual poster presentations at the Annual Meeting of the American Academy of Ophthalmology (AAO) held November 13-15, 2020. \n ProQR’s presentations Presentation title: Phase 1b/2 trial results of intravitreal sepofarsen RNA therapy in Leber congenital amaurosis 10 (LCA10) Presenter: Stephen R. Russell, MD, Professor and Director of Vitreoretinal Diseases and Surgery Service, Department of Ophthalmology and Visual Sciences, University of Iowa Date: The poster presentation will be available on the AAO meeting portal starting November 11, 2020 Presentation title: Full-field stimulus testing (FST) to assess sepofarsen patient response in Leber congenital amaurosis type 10 (LCA10) Presenter: Allen Ho, MD, Professor of Ophthalmology, Wills Eye Hospital, Thomas Jefferson University Date: The poster presentation will be available on the AAO meeting portal starting November 11, 2020 About SepofarsenSepofarsen (QR-110) is being evaluated in the pivotal Phase 2/3 Illuminate trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of Leber congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene. The p.Cys998X mutation leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein. Sepofarsen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA. About ProQRProQR 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 autosomal dominant retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are g...