Welcome to our dedicated page for Proqr Therapeuti news (Ticker: PRQR), a resource for investors and traders seeking the latest updates and insights on Proqr Therapeuti stock.
ProQR Therapeutics (PRQR) is a clinical-stage biopharmaceutical company pioneering RNA-based therapies for severe genetic disorders. This page provides investors and industry observers with timely updates on clinical developments, regulatory milestones, and strategic partnerships related to its Axiomer RNA editing platform.
Access verified press releases and objective news coverage spanning key areas including clinical trial progress, research collaborations, and intellectual property advancements. Our curated collection ensures you stay informed about PRQR's work in addressing unmet medical needs through precise RNA modification technologies.
Discover updates on therapeutic programs targeting conditions like cystic fibrosis and Rett syndrome, financial disclosures, and partnership announcements with industry leaders. All content is sourced from official channels and reputable financial publications to maintain accuracy and compliance.
Bookmark this page for streamlined access to PRQR's latest scientific advancements and corporate updates. Check regularly for new developments in RNA editing innovation and the company's progress toward delivering transformative genetic medicines.
ProQR Therapeutics reported Q1 2022 results, revealing a net loss of €14.5 million, or €0.20 per diluted share. The company's cash reserves stood at €167.6 million, down from €187.5 million in Q4 2021. Key highlights include ongoing trials for sepofarsen and ultevursen, with encouraging post-hoc efficacy data from the Illuminate trial. ProQR plans to meet with the EMA and FDA in Q3 to discuss findings and intends to expand its Axiomer RNA editing platform beyond ophthalmology.
Financially, R&D costs increased to €13.4 million from €8.9 million year-over-year.
ProQR Therapeutics (Nasdaq: PRQR) announced presentations at the 7th Annual Retinal Cell and Gene Therapy Innovation Summit on April 29, 2022, and the ARVO Annual Meeting from May 1-4, 2022, in Denver, CO. Dr. Artur Cideciyan presented on AON treatment for CEP290-LCA, while Dr. Bart Leroy discussed the efficacy of sepofarsen in treating LCA10. Notable presentations include long-term safety data and genetic therapies aimed at restoring vision in rare inherited diseases. The company focuses on delivering transformative RNA therapies to combat severe genetic conditions.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced its participation in the Kempen Life Sciences Conference scheduled for April 20, 2022. The company specializes in transformative RNA therapies, utilizing a unique technology called Axiomer® to edit RNA and potentially treat genetic diseases. ProQR aims to develop innovative medicines using its proprietary RNA repair platform, prioritizing patient needs. For further details, visit www.proqr.com.
ProQR Therapeutics (PRQR) announced encouraging post-hoc analyses from the Illuminate trial of sepofarsen, indicating efficacy when comparing treated and untreated eyes. The company plans to engage with regulators in Q3 regarding these findings. ProQR's strategy now focuses on accelerating its Axiomer® RNA base-editing platform and selected inherited retinal disease programs, along with a 30% workforce reduction to extend its cash runway into 2025. Despite setbacks in achieving the primary endpoint of the Illuminate trial, the results support ongoing development and further regulatory discussions.
On April 4, 2022, ProQR Therapeutics (Nasdaq: PRQR) announced its participation in the 3rd RNA Editing Summit in Boston, MA. Co-founder Gerard Platenburg will present Axiomer®, a proprietary RNA base editing platform, on April 6 at 11:00am ET. This innovative technology utilizes the body's ADAR editing machinery to reverse disease-causing RNA mutations. Axiomer® has broad potential, targeting genetic diseases like Leber congenital amaurosis and Usher syndrome, with a strong patent portfolio reinforcing its promise in treating previously untreatable conditions.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced participation in the Cantor Virtual Rare Orphan Disease Summit on March 29, 2022, from 2:45-3:45 PM EDT. The Company focuses on developing transformative RNA therapies for genetic eye diseases, including Leber congenital amaurosis 10 and Usher syndrome. ProQR uses proprietary RNA repair technologies to grow its pipeline, prioritizing patients' needs. For more information, visit www.proqr.com.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced the appointment of Dr. John Maraganore as a strategic advisor to its Supervisory Board. Maraganore, a biopharma leader and founding CEO of Alnylam Pharmaceuticals, will aid in defining ProQR's future direction as the company analyzes data from its Illuminate trial of sepofarsen. Despite recent setbacks, Maraganore highlighted ProQR's solid fundamentals, including deep RNA expertise and a strong cash position. This partnership is expected to bring valuable insights to advance ProQR's RNA therapies for genetic eye diseases.
ProQR Therapeutics N.V. (Nasdaq: PRQR) reported its Q4 and full-year 2021 results, revealing a net loss of €61.7 million or €0.96 per diluted share. The Phase 2/3 Illuminate trial of sepofarsen for LCA10 failed to meet primary endpoints, prompting ongoing analyses. The company plans to advance several clinical trials including ultevursen for Usher syndrome and retinitis pigmentosa. Despite recent setbacks, ProQR maintains a strong cash position with €187.5 million, providing a runway into mid-to-late 2024. Updates on key programs are expected throughout 2022.
ProQR Therapeutics announced that its pivotal Phase 2/3 Illuminate trial of sepofarsen for Leber congenital amaurosis 10 (LCA10) failed to meet its primary endpoint of Best Corrected Visual Acuity (BCVA) at Month 12. The trial showed no significant benefits in either treatment group compared to the sham. While sepofarsen was generally well-tolerated, and the company maintains a strong cash position into mid-2024, the disappointing trial results pose a setback for the targeted RNA therapy aimed at a high unmet medical need. Further analyses are planned for future presentations.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced its participation in the virtual SVB Leerink 11th Annual Global Healthcare Conference on February 17, 2022. The presentation will be available via live webcast from 1:40 – 2:05 PM EST. ProQR focuses on transformative RNA therapies targeting genetic eye diseases like Leber congenital amaurosis 10 and Usher syndrome. Details for the webcast can be accessed through the company’s website, with archived content available for 30 days post-event.
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