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 year Multiple clinical milestones, including initial AX-0811 Phase 1 data expected by year-end 2026, followed in H1 2027 by:...
LEIDEN, Netherlands and CAMBRIDGE, Mass., June 25, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (“ProQR”), a clinical-stage biotechnology company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer ™ RNA editing technology platform, today announced the pricing of an underwritten registered...
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 cohorts AX-0810 demonstrated up to 8-fold change (6 mg/kg) in total bile acids in serum, exceeding the 2-fold threshold identified by the Company as a meaningful indicator of NTCP...