Core Insights - ProQR Therapeutics reported strong financial and operational results for Q1 2025, highlighting a solid balance sheet and a focus on executing its RNA editing programs [2][5][8] Financial Performance - As of March 31, 2025, ProQR held cash and cash equivalents of €132.4 million, down from €149.4 million at the end of 2024 [8] - The net loss for Q1 2025 was €10.1 million, or €0.10 per diluted share, compared to a net loss of €7.7 million, or €0.09 per diluted share, for the same period last year [10][19] - Research and development costs increased to €12.3 million in Q1 2025 from €9.3 million in Q1 2024, while general and administrative costs decreased slightly to €3.2 million from €3.5 million [9] Business Updates - ProQR is on track to submit a Clinical Trial Application (CTA) for its lead RNA editing program, AX-0810, targeting NTCP for cholestatic diseases in Q2 2025, with initial clinical data expected in Q4 2025 [5][6] - The company has strengthened its leadership team with the appointments of Dennis Hom as Chief Financial Officer and Dr. Cristina Lopez Lopez as Chief Medical Officer [6][5] - ProQR achieved a milestone in its collaboration with Eli Lilly, earning $1.0 million (€918,000) during the first quarter [8] Upcoming Milestones - Key upcoming events include the CTA submission for AX-0810 in Q2 2025 and the first clinical data readout in Q4 2025 [5][6] - Other programs in the pipeline include AX-2402 for Rett Syndrome and AX-2911 for MASH, with clinical candidate selections expected in 2025 [6] Research and Development Focus - ProQR's Axiomer RNA editing technology is advancing across liver and CNS programs, with a focus on addressing cholestatic liver diseases and other conditions [5][11] - The company presented at the RNA Editing Gordon Research Conference and plans to showcase multiple abstracts at the upcoming ASGCT Annual Meeting [6]

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]