Recent Updates — PRQR

ProQR Therapeutics announced it will present clinical and preclinical updates for its RNA editing programs at three major events. The company will showcase ADAR-mediated RNA editing advances at TIDES Europe in November, followed by Phase 1 data for the NTCP-targeting oligonucleotide AX-0810 at AASLD The Liver Meeting 2026. Additionally, Cohort 3 results and new preclinical proof-of-concept data for the next-generation candidate AX-0811 will be presented in December. Initial clinical data for AX-0811 is expected in early January 2027. ProQR Therapeutics operates as a clinical-stage biotechnology company developing transformative RNA therapies using its proprietary Axiomer platform.

ProQR Therapeutics N.V. announced the appointment of Chris Hart as Chief Data and AI Officer and Thomas Wolf to its Board of Directors as a Strategic AI Advisor on September 23, 2026. Dr. Hart joins from Eli Lilly, where he served as Vice President of Data Science and AI/ML, bringing over two decades of experience in computational biology and oligonucleotide drug development. Dr. Wolf is the co-founder and Chief Science Officer of Hugging Face, an open-source AI platform recently acquired by Nvidia for $12.9 billion. These appointments support ProQR's strategy to scale artificial intelligence across its Axiomer RNA editing platform. The company highlighted AX-0811, the first program identified through this AI-enabled discovery process, which is expected to provide target engagement data in early January 2027. ProQR anticipates up to five clinical data readouts over the next 12 months across four development programs. ProQR Therapeutics N.V. operates in the biotechnology industry, developing transformative RNA therapies using its proprietary Axiomer RNA editing technology.

ProQR Therapeutics N.V. reported second quarter 2026 financial results and provided a business update. The company announced positive Phase 1 target engagement data for AX-0810, establishing the first clinical validation of its Axiomer RNA editing platform through NTCP modulation. In June, ProQR closed an underwritten registered direct offering and concurrent private placement with Eli Lilly and Company, raising approximately $59.2 million in gross proceeds. This financing strengthened the balance sheet, bringing cash and cash equivalents to €117.1 million as of June 30, 2026, supporting operations through mid-2028. Net loss for the quarter was €8.7 million, or €0.08 per diluted share, compared to a net loss of €12.2 million in the prior year period. The company anticipates initial Phase 1 data for its next-generation candidate AX-0811 by year-end 2026 and plans to initiate an investigator-initiated trial for biliary atresia in China with data expected in the first half of 2027. ProQR Therapeutics is a clinical-stage biotechnology company developing transformative RNA therapies based on its proprietary Axiomer RNA editing technology platform.

ProQR Therapeutics N.V. reported second quarter 2026 financial results and provided a business update. The company closed an underwritten registered direct offering of $50.0 million alongside a concurrent private placement with Eli Lilly for $9.2 million, totaling approximately $59.2 million in gross proceeds. This financing strengthened the balance sheet, ending Q2 2026 with €117.1 million in cash and cash equivalents, supporting operations through mid-2028. Net loss for the quarter was €8.7 million, or €0.08 per diluted share, compared to a net loss of €12.2 million, or €0.12 per diluted share, in the same period last year. R&D costs were €12.7 million and general and administrative costs were €4.9 million. Clinically, ProQR announced positive AX-0810 Phase 1 target engagement data establishing first clinical validation of its Axiomer RNA editing platform for NTCP modulation. The company also submitted a Clinical Trial Application to the EMA for AX-0811, with initial data expected by year-end 2026. ProQR Therapeutics is a clinical-stage biotechnology company developing transformative RNA therapies using its proprietary Axiomer RNA editing technology.