At our offices in Leiden we are advancing our proprietary Axiomer RNA-editing platform technology. Our ProQRians, representing over 30 nationalities, are passionate and driven to change the lives of patients and their loved ones.
ProQR's leadership is a strong team with proven track record. Find out who is on our management team, board of directors and scientific advisory board.
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
AX-0810 target engagement data in healthy volunteers on track for Q2 2026; biliary atresia selected as initial Phase 2 indicationPipeline expansion with advancement of AX-0811 (NTCP) and AX-0422 (IDUA) toward the clinic, supporting multiple upcoming clinical cataly
