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ProQR Therapeutics N.V. (PRQR)
Nasdaq · Manufacturing · Pharmaceutical Preparations
Core research established · 4/4 core data layers currently established
What ProQR Therapeutics N.V. does
A. History and Development of the Company We are a clinical-stage biotechnology company focused on advancing a pipeline of RNA editing therapeutics based on our proprietary Axiomer RNA-editing platform. Our lead program, AX-0810 for cholestatic diseases targeting NTCP, is currently being evaluated in a Phase 1 clinical trial in healthy volunteers. With funding from the Rett Syndrome Research Trust, we are also advancing AX-2402 targeting methyl CpG binding protein 2 mutations for Rett syndrome, a severe neurodevelopmental disorder. Additional pipeline programs include AX-2911 targeting PNPLA3 for metabolic dysfunction-associated steatohepatitis, AX-1412 targeting the B4GALT1 gene for CVDs, as well as a number of additional discovery-stage programs.
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