LEIDEN, Netherlands & CAMBRIDGE, Mass., May 12, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that it will p
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Strengthened leadership with appointments of Chief Financial Officer and Chief
First-in-human trial of AX-0810 will evaluate safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers with initial data expected in Q4 2025Marks the first submitted Clinical Trial Application (CTA) advancing ProQR’s Axiomer™ ADAR-mediated RNA
Preclinical and translational pipeline program data for AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease targeting B4GALT1 anticipated in 2024, building further momentum toward the clinicAccelerated development of Axiomer™ for CNS ap
… in rodent and non-human primates Beyond correction, broad applicability of Axiomer demonstrated in preclinical … with Eli Lilly that is exclusively focused on RNA editing, leading IP position, and cash runway into mid-2026, ProQR is … 50% editing reported in the nervous system of NHP in vivo Additionally, ProQR’s Axiomer technology achieves increased …
… Results Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and … areas Strength of intellectual property estate and leading IP position supported with successful defense of opposition against a key Axiomer patent protecting ADAR-mediated RNA editing Well-capitalized with €139 M, in …
… specific RNA for A-to-I editing by recruiting endogenous Adenosine Deaminase Acting on RNA (ADAR). This presentation will highlight the therapeutic … limited to disease causing mutations and can potentially address high unmet medical needs by editing wild-type RNA to …
Preclinical proof of concept data for the AX-0810 program targeting NTCP for cholestatic diseases presented at ASGCT 27th Annual Meeting – Management to host webinar today at 8:00 am EDT to highlight dataMartin Maier, PhD nominated to Board with Annual General
