ProQR Announces Presentation on QR-421a Program in Ophthalmology at Usher Syndrome Coalition Conference in July
LEIDEN, Netherlands and CAMBRIDGE, Mass., July 02, 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 an upcoming presentation at the 11th Annual Usher Connections Conference.
Presentation at USH2019
A presentation will be delivered by Dr. Aniz Girach, MD, chief medical officer of ProQR, on QR-421a for Usher Syndrome during the USH2019 Annual Usher Connections Conference to be held on July 13, 2019 in Philadelphia, PA.
|Abstract title:||QR-421a, An Antisense Oligonucleotide for the Treatment of Retinitis Pigmentosa Due To USH2A Exon 13 Mutations|
|Presenter:||Aniz Girach, MD, CMO of ProQR|
|Presentation Time:||Saturday, July 13 at 11-11:30 a.m. ET|
About Usher Syndrome Type 2
Usher syndrome is the leading cause of combined deafness and blindness. Patients with this syndrome generally progress to a stage in which they have very limited central vision and moderate to severe deafness. Usher syndrome type 2 is one of the most common forms of Usher syndrome and can be caused by mutations in the USH2A gene. To date, there are no treatments approved or products in clinical development that treat the vision loss associated with Usher syndrome type 2.
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.
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, Usher syndrome type 2 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.
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 11th Annual USH Connections Conference. 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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