LEIDEN, Netherlands and CAMBRIDGE, Mass., April 22, 2019 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced upcoming presentations at the Retinal Cell & Gene Therapy Innovation Summit, the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) and the annual meeting of the American Society for Gene and Cell Therapy (ASGCT).

Retinal Cell & Gene Therapy Innovation Summit

The conference is being held on Friday, April 26, 2019 in Vancouver, BC, Canada.

Abstract title:  Novel type of antisense oligonucleotide treatment (QR-421a) for retinitis pigmentosa (RP) due to exon 13 USH2A mutations
Presenter:  Robert K. Koenekoop, MD, PhD, principle investigator of the ProQR Phase 1/2 STELLAR clinical trial of QR-421a and Professor of Pediatric Surgery, Human Genetics and Ophthalmology at McGill University, Montreal
Presentation:  Friday, April 26 at 5:00pm PT
Session:  Non Viral Gene Editing
    
Abstract title:  Results of first-in-human study of an antisense oligonucleotide in subjects with LCA10 
Presenter:  Bart P. Leroy, MD, PhD, principle 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 
Presentation:  Friday, April 26 at 8:15am PT 
Session:   Gene Therapy: Clinical Applications and Outcomes

Annual meeting of the Association for Research in Vision and Ophthalmology (ARVO)

The conference is being held from April 28 – May 2, 2019 in Vancouver, BC, Canada.

Abstract title:  CEP290-associated LCA due to a photoreceptor cilium defect treated with an intravitreal antisense oligonucleotide results in improved vision
Presenter:  Artur V. Cideciyan, PhD, co-investigator of the PQ-110-001 clinical trial of sepofarsen and Research Professor of Ophthalmology at the Scheie Eye Institute of the University of Pennsylvania
Poster:   1834 - A0101
Session:   #240 - Visual Impairment - Measures of Visual Function
Date & time:   April 29, 2019 from 11:15 am to 1:00 pm PT
    
Abstract title:  QR-421a, an antisense oligonucleotide, for the treatment of retinitis pigmentosa due to USH2A exon 13 mutations 
Presenter  Hester van Diepen, Director Ophthalmology at ProQR 
Presentation:  3250   
Session:  #340 - Retina/RPE New drugs, Mechanisms of action, and Toxicity 
Date & Time:  April 30, 2019 from 1:00 pm to 1:15 pm PT
    
Abstract title:  Allele specific knock-down of human P23H rhodopsin mRNA and prevention of retinal degeneration in humanized P23H rhodopsin knock-in mouse, following treatment with an intravitreal GAPmer antisense oligonucleotide (QR-1123) 
Presenter:  Patricia Biasutto, VP, Project Lead for QR-1123 at ProQR 
Poster:   5719 - A0114 
Session:   #506 - Retina/RPE new drugs, mechanism of action, and toxicity 
Date & time:   May 2, 2019 from 8:00 am to 9:45 am PT

Annual meeting of the American Society for Gene and Cell Therapy (ASGCT)

The conference is being held from April 28 – May 2, 2019 in Washington, DC.

Presenter:  David Rodman, MD, EVP Research & Development of ProQR
Presentation:  Tuesday, April 30 at 9:00 – 9:30 am ET
Session:   RNA therapeutics education session

About sepofarsen

Sepofarsen is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s 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 is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to restore normal (wild-type) CEP290 mRNA leading to the production of normal CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. 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 from the FDA.

About QR-421a

QR-421a is a first-in-class investigational RNA-based oligonucleotide 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 United States and the European Union and received fast-track designation from the FDA.

About QR-1123

QR-1123 is a first-in-class investigational oligonucleotide (gapmer) that was developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology for the treatment of adRP due to the P23H mutation in the RHO gene. The therapy aims to inhibit the formation of the mutated toxic version of the rhodopsin protein by specifically binding the mutated RHO mRNA. Binding of QR-1123 causes allele specific knockdown of the mutated mRNA by a mechanism called RNase H mediated cleavage without affecting the normal RHO mRNA. QR-1123 is intended to be administered through intravitreal injections in the eye.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as Leber’s congenital amaurosis 10 and Usher syndrome type 2. 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 participation at the Retinal Cell & Gene Therapy Innovation Summit, the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) and the annual meeting of the American Society for Gene and Cell Therapy. 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.
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