Submitted CTA for lead program AX-0810, targeting NTCP for cholestatic diseases Advancing AX-2402 program toward clinical candidate selection, targeting MECP2 (R270X) for Rett Syndrome Hosting fall Analyst and Investor Event (virtual), featuring detailed AX-0810 Phase 1 trial design and 2025 data expectations, plus updates across differentiated liver and CNS pipeline € 119.8 million cash and cash equivalents as of end Q2 –... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., July 28, 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 participate in the RNA Editing Summit taking place July 29-31, 2025, in Boston, MA. “We recently achieved a key milestone with the... Read More

First-in-human trial of AX-0810 will evaluate safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers with initial data expected in Q4 2025 Marks the first submitted Clinical Trial Application (CTA) advancing ProQR’s Axiomer™ ADAR-mediated RNA editing platform into clinical development LEIDEN, Netherlands & CAMBRIDGE, Mass., June 26, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR)... Read More

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 present at two upcoming scientific conferences: the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, taking place May... Read More

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... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., May 02, 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 Company management is scheduled to present at the Citizens (JMP) Life Sciences Conference in New York City on Thursday, May 8 at 11:30 AM EDT. A... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., May 01, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the Annual General Meeting (AGM) of Shareholders will take place on Tuesday, June 3, 2025 at 1530 CEST at the offices of Allen Overy Shearman... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 14, 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 the appointment of Dennis Hom as Chief Financial Officer (CFO) and Cristina Lopez Lopez, MD, PhD, as Chief Medical Officer (CMO). These key... Read More

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 Company expects up to four clinical data readouts in 2025 and 2026 across multiple programs, highlighted by first clinical data for AX-0810 anticipated in Q4 2025 € 149.4 million cash and cash equivalents as of end of 2024, providing runway... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 11, 2024 (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 an expansion of its collaboration with the Rett Syndrome Research Trust (RSRT). Building on the initial $1 million research grant announced in... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 10, 2024 (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 the appointment of Dr. Peter Beal, a professor of Chemistry at the University of California, Davis, as Chief ADAR Scientist. Dr. Beal, one of the top... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 05, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced that the Company will host a virtual Analyst and Investor Event on Wednesday, December 11, 2024 from 10:00 am until approximately 12:30 pm EST, including an Analyst Q&A session with members of the ProQR Management... Read More

AX-0810 targeting NTCP for cholestatic diseases to be first ProQR Axiomer™ RNA editing program to enter the clinic Company to host virtual Investor and Analyst Event in December to highlight translational data and clinical plans for AX-0810, as well as AX-1412, targeting B4GALT1 for cardiovascular diseases, and progress made across the Axiomer platform €89.4 million cash and cash equivalents as of end of Q3, plus $82.1... Read More

LEIDEN, the Netherlands and CAMBRIDGE, Mass., Oct. 25, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (“ProQR”), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the closing of its previously announced underwritten public offering of 18,000,000 ordinary shares (the “Offering”) at a public offering... Read More

LEIDEN, Netherlands and CAMBRIDGE, Mass., Oct. 22, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (“ProQR”), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer ™ RNA editing technology platform, today announced the pricing of its previously announced underwritten public offering of 18,000,000 ordinary shares (the “Offering”) at a public offering price... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Oct. 22, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (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 has commenced an underwritten public offering of its ordinary shares (the “Offering”). All of the shares are being offered by ProQR. In... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Oct. 07, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will present preclinical data for its proprietary Axiomer™ RNA editing technology platform and its AX-0810 pipeline program for cholestatic diseases targeting NTCP at the 20th Annual Meeting of the Oligonucleotide... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 10, 2024 (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 Company management is scheduled to present at the 2024 Cantor Global Healthcare Conference in New York City on Tuesday, September 17 at 9:10 am... Read More

AX-0810 program targeting NTCP for cholestatic diseases and AX-1412 program targeting B4GALT1 for cardiovascular diseases advancing to the clinic in late 2024/early 2025 with translational data and trial design details to be announced in H2 2024 $4.5 million in milestones achieved in H1 related to progress in the Lilly partnership €96.2 million cash position as of end of Q2 providing runway into mid-2026, plus additional... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., June 18, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will participate in the RNA Editing Summit, June 18-20, 2024, in Boston, Massachusetts. As part of the conference program, Gerard Platenburg, Chief Scientific Officer and Co-Founder, ProQR Therapeutics, will... Read More

Preclinical proof of concept data for the AX-0810 program targeting NTCP for cholestatic diseases presented at ASGCT 27 th Annual Meeting – Management to host webinar today at 8:00 am EDT to highlight data Martin Maier, PhD nominated to Board with Annual General Meeting scheduled for May 22, 2024 Continued strength of leading IP estate around ADAR-mediated RNA editing highlighted with successful defense of challenge to... Read More

ProQR scientists report for the first time in the ADAR RNA editing field in vivo proof of target engagement (RNA editing) leading to meaningful changes in biomarkers in NHPs using Axiomer™ RNA Editing Oligonucleotides Preclinical proof of concept for the Company’s AX-0810 program targeting NTCP for cholestatic diseases presented at ASGCT Annual Meeting Management webinar May 9, 2024 at 8:00 am EDT LEIDEN, Netherlands &... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 23, 2024 (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 the Annual General Meeting (AGM) of Shareholders will take place on Wednesday, May 22, 2024 at 1630 CET (10:30am EDT) at the offices of Allen &... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will present new preclinical data for its proprietary Axiomer™ RNA editing technology platform, including the first preclinical data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP at the... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 19, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it has again successfully defended against opposition filed against a key patent for its ADAR-mediated RNA editing platform Axiomer™. The opposition was filed in June 2021 with the European Patent Office (EPO) by... Read More

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 clinic Accelerated development of Axiomer™ for CNS applications via new research partnership with Rett Syndrome Research Trust Completed divestment of sepofarsen and ultevursen ophthalmic programs to... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 15, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it has successfully defended against an opposition filed in Japan against a patent directed to its ADAR-mediated RNA editing platform Axiomer™. The opposition was filed in September 2023 with the Japanese Patent... Read More

Platform demonstrates robust in vivo editing capabilities reporting up to 70% editing of ACTB in the liver of non-human primates (NHPs) and mice Functional effect demonstrated in mice in vivo via modulation of ANGPLT3 protein properties leading to favorable increase in LPL enzymatic activity and meaningful impact of biomarkers Additional data to be presented throughout 2024, including in vitro and in vivo data for pipeline... Read More

Partnership focused on utilizing Axiomer ™ to develop editing oligonucleotides targeting an underlying genetic variant that causes Rett syndrome Collaboration expands ProQR’s commitment to advance the development of therapies for central nervous system diseases LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan. 05, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through... Read More

Divestment of sepofarsen and ultevursen completed – Théa to continue development of sepofarsen and ultevursen for patients with LCA10 and Usher syndrome Agreement provides ProQR with initial payment of €8M and up to €165M in earn-out payments, as well as potential double-digit royalties based on commercial sales in the US and EU Transaction supports ProQR’s strategic focus on its proprietary Axiomer ® RNA editing technology... Read More

Continued advancement of platform and initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1 Further strengthened leading intellectual property (IP) position with issuance of new patent in the United States and successful defense against opposition to its IP in Japan €120.6 M cash and cash equivalents as of September 30, 2023 providing runway... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Nov. 06, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it has further strengthened its leading intellectual property (IP) estate with the issuance of a new patent in the United States and that it has successfully defended against an opposition to its IP in Japan. “The... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 28, 2023 (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 Daniel A. de Boer, Founder and CEO, will participate in the ADAR Editing Panel at the Chardan 7 th Annual Genetic Medicines Conference in New... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 27, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies today provided an update that its previously announced agreement to divest the Company’s late-stage ophthalmic assets to Laboratoires Théa S.A.S. (Théa) has been terminated. The completion of the transaction was conditional on... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 07, 2023 (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 Company management will participate in the HC Wainwright 25 th Annual Global Investment Conference. A webcast of the Company’s presentation... Read More

Divestment of sepofarsen and ultevursen ophthalmic programs supports ProQR’s strategic focus on Axiomer® RNA editing platform technology and continued advancement of pipeline €129 M cash and cash equivalents as of June 30, 2023 providing runway into mid-2026 LEIDEN, Netherlands & CAMBRIDGE, Mass., Aug. 03, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through... Read More

Agreement provides ProQR with initial payment of €12.5M and up to €135M in further payments, as well as potential additional earn outs based on commercial sales in the US and EU Divestment of sepofarsen and ultevursen supports ProQR’s strategic focus on the Axiomer® RNA editing technology platform and continued advancement of pipeline programs, AX-0810 and AX-1412, focused on genetic diseases originating in the liver LEIDEN,... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., July 06, 2023 (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 a presentation at the RNA Editing Summit, July 11-13, 2023 in Boston, MA, USA. The 2023 edition of the RNA Editing Summit will focus on optimizing... Read More

Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1 Development of Axiomer ® RNA base editing technology platform continues across multiple therapeutic 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... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., May 09, 2023 (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 a presentation at the Oligonucleotide and Peptide Therapeutics conference (TIDES USA), May 7-10, 2023 in San Diego, CA, USA. ProQR presentation... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 20, 2023 (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 Company management will participate in the following upcoming investor conferences and events. Van Lanschot Kempen Life Sciences Conference –... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 18, 2023 (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 the Annual General Meeting (AGM) of Shareholders will take place on Wednesday, May 17, 2023 at 16:00 CET (10:00am EDT) at the offices of Allen &... Read More

Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1; initiation of clinical trials anticipated in late 2024/early 2025 Axiomer activity demonstrated across multiple preclinical in vitro , organoid, and in vivo models; up to 50% editing observed in rodent and non-human primates Beyond correction, broad applicability of Axiomer demonstrated... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., March 14, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it has successfully defended against two oppositions that were filed against a key patent for its ADAR-mediated RNA editing platform Axiomer®. The oppositions were filed in February 2021 with the European Patent... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., March 07, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced several presentations highlighting the Company’s proprietary Axiomer ® RNA editing technology platform at upcoming scientific and industry conferences, including the 8 th annual Oligonucleotide and Precision... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 28, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced that the Company will host a virtual R&D event on Wednesday, March 29, 2023 from 10:00 am until 12:30 pm EDT, including an Analyst Q&A session with members of the ProQR Management Team. During the event, the... Read More

LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 22, 2022 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced that ProQR management will host an investor conference call and webcast to discuss the Company’s Axiomer RNA editing technology platform following the recently announced partnership expansion with Lilly. ProQR will... Read More

INDIANAPOLIS; LEIDEN, Netherlands; and CAMBRIDGE, Mass., Dec. 22, 2022 (GLOBE NEWSWIRE) -- Eli Lilly and Company (NYSE: LLY) and ProQR Therapeutics N.V . (Nasdaq: PRQR), today announced the expansion of their licensing and collaboration agreement focused on the discovery, development and commercialization of new genetic medicines. The collaboration, originally announced in September 2021, applied ProQR’s proprietary Axiomer®... Read More