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ProQR scientist in the lab

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.

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.


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