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 Medical Officer€ 132.4 million cash and cash equivalents as of end Q1 providing runway into mid-2027, plus additional potential milestones from Lilly partnership LEIDEN, Netherlands & CAMBRIDGE, Mass., May 08, 2025 (GLOBE NEWSWIRE) -- ProQR Thera ...

Company Overview - ProQR Therapeutics NV is focused on developing transformative RNA therapies using its proprietary Axiomer RNA editing technology platform [1][4] - The company aims to create a new class of medicines targeting both rare and prevalent diseases with unmet medical needs [4] Axiomer Technology - Axiomer is a next-generation RNA base editing technology that allows for specific single nucleotide changes in RNA [3][4] - The technology utilizes the human cell's own ADAR (Adenosine Deaminase Acting on RNA) machinery to convert Adenosine (A) to Inosine (I), which is then translated as Guanosine (G), effectively correcting disease-causing mutations [3] Upcoming Events - ProQR management is scheduled to present at the Citizens (JMP) Life Sciences Conference in New York City on May 8, 2025, at 11:30 AM EDT [1] - A live webcast of the presentation will be available on ProQR's website, with a replay accessible for approximately 30 days [2]