LEIDEN,
Presentation at USH2019
A presentation will be delivered by Dr.
| 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.
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 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, 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.
*Since 2012*
FORWARD-LOOKING STATEMENTS
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Source: ProQR Therapeutics N.V.