ProQR Therap

ProQR Therapeutics N.V., a clinical-stage biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company develops an RNA editing platform through its Axiomer RNA editing technology for genetic disorders with unmet needs. Its lead product candidate, AX-0810, targets na-taurocholate cotransporting polypeptide (NTCP) for the treatment of cholestatic diseases. The company also develops AX-2402, which targets methyl CpG binding protein 2 mutations for Rett syndrome; AX-2911, which targets PNPLA3 for metabolic dysfunction-associated steatohepatitis; and AX-1412, which targets the B4GALT1 gene for cardiovascular diseases.