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ProQR Announces CTA Submission for Phase 1 Study of AX-0810 Targeting NTCP
Globenewswire· 2025-06-26 20:30
Core Viewpoint - ProQR Therapeutics has submitted a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) for its lead program AX-0810, which targets NTCP to address cholestatic liver diseases, marking a significant milestone in advancing its Axiomer RNA editing platform into clinical development [1][2][8] Company Overview - ProQR Therapeutics is focused on developing transformative RNA therapies using its proprietary Axiomer™ RNA editing technology platform, which utilizes ADAR-mediated RNA editing to create specific nucleotide changes in RNA [4][5] - The company aims to address unmet medical needs in both rare and prevalent diseases through its innovative RNA repair platform [5] Product Details - AX-0810 is an investigational RNA editing oligonucleotide designed to treat cholestatic liver disease by targeting NTCP, which is involved in bile acid transport [3] - The mechanism of AX-0810 is supported by human genetics data indicating that certain NTCP variants can reduce bile acid reuptake, suggesting potential benefits in improving liver health [3] Clinical Trial Information - The proposed Phase 1 study for AX-0810 will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy adult volunteers, with initial data expected in Q4 2025 [2][8] - The study will also explore biomarkers to assess early signals of target engagement, and is set to commence at a single site in the Netherlands pending regulatory clearance [2]
ProQR Announces Annual General Meeting of Shareholders to be Held June 3, 2025
GlobeNewswire News Room· 2025-05-01 12:00
Company Overview - ProQR Therapeutics N.V. 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 will hold its Annual General Meeting (AGM) on June 3, 2025, at 15:30 CEST in Amsterdam, Netherlands [1] - Relevant documents for the AGM will be available on ProQR's website and the SEC's website [2]
ProQR Strengthens Leadership with Appointments of CFO and CMO to Support Next Phase of Growth
Newsfilter· 2025-04-14 11:00
Core Insights - ProQR Therapeutics has appointed Dennis Hom as Chief Financial Officer and Dr. Cristina Lopez Lopez as Chief Medical Officer to advance its Axiomer platform and RNA editing programs into clinical stages [1][2][3] Leadership Appointments - Dennis Hom brings over 25 years of financial leadership experience, having raised more than $4.5 billion in capital and executed transactions totaling over $57 billion in disclosed value [2] - Dr. Cristina Lopez Lopez has over 20 years of experience in translational R&D, previously serving as Global Head of Neurodegeneration at Johnson & Johnson and holding senior roles at Roche and Novartis [3] Transition of Current Executives - Current CFO Jurriaan Dekkers and Chief Corporate Development Officer René Beukema will step down but remain during a transitional period to ensure continuity [4][5] Axiomer Technology Overview - ProQR is pioneering Axiomer™, a next-generation RNA base editing technology that allows specific single nucleotide edits in RNA, potentially leading to new medicines for various diseases [5][7] - Axiomer™ Editing Oligonucleotides (EONs) utilize the human cell's ADAR machinery to correct disease-causing mutations in RNA, modulating protein expression or altering protein functions [5]