Our platform based on intravitreal delivery uses chemically modified single-stranded RNA oligonucleotides to repair defective RNA. Given the high unmet need across more than 300 inherited retinal diseases, our RNA therapies have broad potential to change lives by stopping or reversing vision loss. We are evaluating sepofarsen in a pivotal Phase 2/3 trial for LCA10, QR-421a in a Phase 1/2 trial for RP and Usher syndrome, and QR-1123 for RP, along with advancing earlier stage assets.
Investors & Media Value through vision
Located in Leiden, Netherlands and Cambridge, Mass., ProQR Therapeutics (Nasdaq:PRQR) is a clinical-stage biotechnology company developing RNA therapies for the treatment of severe genetic rare diseases with a focus on inherited blindness.
Presentations and publications
The latest scientific publications for our development programs
ProQR Share Performance
Press Releases
-
ProQR Announces First Quarter 2021 Operating and Financial Results
-
ProQR Therapeutics and Yarrow Biotechnology, an RTW Investments, LP Incubated Company, Announce Exclusive Worldwide License and Discovery Collaboration for Undisclosed Target
-
ProQR Announces Virtual Presentation of Phase 1/2 Sepofarsen Extension Trial Data at the Association for Research in Vision and Ophthalmology (ARVO) 2021
