ProQR Therapeutics is a NASDAQ-listed biotechnology company headquartered in Leiden, the Netherlands, with an office in Cambridge, Massachusetts, USA, developing innovative RNA-editing medicines through its proprietary Axiomer™ platform.
ProQR Therapeutics is a NASDAQ-listed biotechnology company headquartered in Leiden, the Netherlands with an office in and Cambridge, Massachusetts, USA.
ProQR Therapeutics is a NASDAQ-listed biotechnology company headquartered in Leiden, the Netherlands with an office in and Cambridge, Massachusetts, USA.
ProQR Therapeutics is a NASDAQ-listed biotechnology company headquartered in Leiden, the Netherlands with an office in and Cambridge, Massachusetts, USA.
First clinical validation of Axiomer™ RNA editing established through NTCP modulation with positive AX-0810 clinical target engagement data; full Phase 1 dataset to be presented at a medical or scientific conference later this yearMultiple clinical milestones, including
Data from ongoing Phase 1 study of AX-0810 in healthy volunteers demonstrated dose-dependent target engagement on all key biomarkers in the evaluable 3 mg/kg and 6 mg/kg cohortsAX-0810 demonstrated up to 8-fold change (6 mg/kg) in total bile acids in serum, exceeding the