ProQR Announces Virtual Presentation of Phase 1/2 Sepofarsen Data Through the Association for Research in Vision and Ophthalmology (ARVO)
ProQR Therapeutics (Nasdaq: PRQR) announced a virtual presentation of data from its Phase 1/2 trial of sepofarsen, an RNA therapy for Leber congenital amaurosis 10. The presentation will be made available via ARVO on June 15, 2020, following the cancellation of the annual meeting due to COVID-19. Sepofarsen, designated as an orphan drug, aims to address the genetic mutation causing this rare form of blindness. The trial data, presented by Dr. Stephen R. Russell, will highlight the results of a novel treatment that restores normal function of the CEP290 gene.
- Presentation of Phase 1/2 trial data for sepofarsen, a promising RNA therapy.
- Sepofarsen received orphan drug designation and fast-track status from the FDA.
- Dr. Stephen R. Russell, a respected expert, is presenting the findings.
- The presentation is virtual due to the cancellation of the ARVO annual meeting.
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LEIDEN, Netherlands & CAMBRIDGE, Mass., June 08, 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 a virtual presentation of data from the Company’s Phase 1/2 trial of sepofarsen. The data will be shared via a video presentation through the Association for Research in Vision and Ophthalmology (ARVO). It is the first time this data, previously announced in a press release and conference call in October 2019, will be presented in association with an ophthalmology medical congress.
Due to COVID-19, the ARVO 2020 Annual Meeting was cancelled and instead video-recorded presentations will be available on ARVOLearn, ARVO’s online learning platform.
Details of ProQR’s presentation are as follows:
Presenter: Stephen R. Russell, MD, Professor and Director of Vitreoretinal Diseases and Surgery Service, Department of Ophthalmology and Visual Sciences, University of Iowa
Presentation Title: Results of a phase 1b/2 trial of intravitreal (IVT) sepofarsen (QR-110) antisense oligonucleotide in Leber congenital amaurosis 10 (LCA10) due to p.Cys998X mutation in the CEP290 gene
Date: The video presentation will be available on ARVOLearn starting June 15, 2020.
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 of data through ARVO. 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
skiely@proqr.com
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LifeSci Advisors
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Media Contact:
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LifeSci Public Relations
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