LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 24, 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 presentations at the Ophthalmology Futures Retina Forum, European Society of Retina Specialists (Euretina) congress and the Annual Meeting of the American Academy of Optometry (AAOpt).ProQR’s presentation at Ophthalmology Futures Retina ForumPresentation title: Advancements in Inherited Retinal Diseases ManagementPresenter: Aniz Girach, MD, Chief Medical Officer of ProQRDate: September 30, 2020ProQR’s presentations at EuretinaPresentation title: Phase 1b/2 trial results of intravitreal sepofarsen RNA therapy in Leber congenital amaurosis 10 (LCA10)Presenter: Stephen R. Russell, MD, principal investigator of the ProQR Phase 1/2 clinical trial of sepofarsen Professor and Director of Vitreoretinal Diseases and Surgery Service, Department of Ophthalmology and Visual Sciences, University of IowaDate: The poster presentation will be available on the Euretina Virtual Congress portal starting October 2, 2020.Presentation title: RNA antisense oligonucleotide sepofarsen in Leber Congenital Amaurosis type 10 (LCA10): disease severity and patient response based on genotypePresenter: Bart P. Leroy, MD, PhD, principal investigator of the ProQR Phase 1/2 clinical trial of sepofarsen and Chairman and Head of the Department of Ophthalmology at the Center for Medical Genetics at the Ghent UniversityDate: The poster presentation will be available on the Euretina Virtual Congress portal starting October 2, 2020.ProQR’s presentation at AAOptPresentation title: Survey of Early Genetic Diagnosis of Inherited Retinal Diseases (IRDs) and Patient Referrals: The important role of Optometrists in the early management of blinding genetic diseasesPresenter: Ramiro S. Maldonado, MD, Assistant Professor of Ophthalmology at Shriners UK Advances Eye Care at the University of KentuckyDate: The poster presentation will be available on the AAOpt Virtual Platform starting October 7, 2020. A live questions and answers session will be held on Friday October 9, 2020, 7:30-9:30 PM EDT.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 growing our pipeline with patients and loved ones in mind.
*Since 2012*FORWARD-LOOKING STATEMENTSThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Such statements include those relating to our presentation at Retina Forum, Euretina and AAopt. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.ProQR Therapeutics N.V.Investor Contact:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
[email protected]
or
Hans Vitzthum
LifeSci Advisors
T: +1 617 535 7743
[email protected]  Media Contact:
Sara Zelkovic
LifeSci Public Relations
T: +1 646 876 4933
[email protected]


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