ProQR Announces Expert Perspectives Call on LCA10 and Sepofarsen

Call on July 20 will include a snapshot from the ongoing InSight open-label extension study of sepofarsen, including preliminary second eye treatment data

LEIDEN, Netherlands & CAMBRIDGE, Mass., July 13, 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 that the Company will host an Expert Perspectives call on July 20, 2020 at 5:00 pm EDT, which will focus on Leber congenital amaurosis 10 (LCA10) and the investigational RNA therapy for LCA10, sepofarsen experience to date.

The call will include a snapshot from the ongoing InSight Phase 1/2 extension study of sepofarsen, including new preliminary data on second eye treatment. Participation in this extension study was offered to patients who completed their participation in the Phase 1/2 study of sepofarsen.

The call will feature a presentation by Aniz Girach, MD, Chief Medical Officer of ProQR Therapeutics. Dr. Girach will then discuss the interpretation of the data with Ian M. MacDonald MSc, MD, CM, Professor Emeritus in the Department of Ophthalmology and Visual Sciences, University of Alberta. They will review topics related to the extension trial data, as well as key takeaways in relation to the overall sepofarsen experience to date.

Event Details

Date/Time: July 20, 2020, 5:00-6:00 pm EDT

To register please follow this link.

Following the presentation and discussion, a portion of the call will be dedicated to Q&A. The archived presentation will be available on the Company’s website for approximately 30 days following the presentation date.

Ian M. MacDonald MSc, MD CM is one of the top experts in the Inherited Retinal Diseases field, and is currently Professor Emeritus in the Department of Ophthalmology and Visual Sciences, University of Alberta and past Chair of the Department for 20 years. Prior to becoming Chair in Edmonton, in 1992, he was a Career Scientist of the Ontario Ministry of Health at the University of Ottawa. From 2007-2008, he was Branch Chief of Ophthalmic Genetics and Visual Function at the National Eye Institute of the NIH. Dr. MacDonald completed residency training in Family Medicine at McGill University, Montreal, Ophthalmology residency training at the University of Ottawa and Clinical Genetics fellowship training at the University of Ottawa, Queen's University, Kingston and the Hospital for Sick Children, Toronto. He is an Editor Emeritus of the Canadian Journal of Ophthalmology and currently serves on the Editorial Board of the American Journal of Ophthalmology for Genetics.

About sepofarsen

Sepofarsen (QR-110) 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 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 bind to the mutated location in the pre-mRNA and enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein.

In a Phase 1/2 clinical trial sepofarsen reported rapid, significant and durable improvements in vision with a favorable benefit/risk profile. Sepofarsen is currently being investigated in a pivotal Phase 2/3 clinical trial.

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 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*


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 sepofarsen. 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
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LifeSci Advisors
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