ProQR Announces Virtual Presentations at Scientific Conferences

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 Forum

Presentation title: Advancements in Inherited Retinal Diseases Management

Presenter: Aniz Girach, MD, Chief Medical Officer of ProQR

Date: September 30, 2020

ProQR’s presentations at Euretina

Presentation 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 Iowa

Date: The poster presentation will be available on the starting October 2, 2020.

Presentation title: RNA antisense oligonucleotide sepofarsen in Leber Congenital Amaurosis type 10 (LCA10): disease severity and patient response based on genotype

Presenter: 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 University

Date: The poster presentation will be available on the starting October 2, 2020.

ProQR’s presentation at AAOpt

Presentation 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 diseases

Presenter: Ramiro S. Maldonado, MD, Assistant Professor of Ophthalmology at Shriners UK Advances Eye Care at the University of Kentucky

Date: The poster presentation will be available on the 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 Sepofarsen

Sepofarsen (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 ProQR

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 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 STATEMENTS

This 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.

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