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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.
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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 (Nasdaq: PRQR) announced the upcoming presentation of results from its Phase 1/2 Stellar trial of QR-421a for treating adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) on March 24, 2021. The randomized trial involved 20 patients, with 14 receiving QR-421a and six undergoing a sham procedure. QR-421a, designed to restore vision by targeting mutations in the USH2A gene, holds significant potential as no treatments currently exist for these conditions. The investor call will provide further insights into the findings.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced its management will present at the HC Wainwright Global Life Sciences Conference on March 9, 2021. A webcast of the presentation will be available on the company's website. ProQR focuses on developing transformative RNA therapies for inherited retinal diseases. The company emphasizes its commitment to creating impactful treatments for genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome, and retinitis pigmentosa.
ProQR Therapeutics has completed enrollment in the pivotal Phase 2/3 Illuminate trial for sepofarsen, targeting LCA10, with top-line results expected in H1 2022. The company is progressing its pipeline, with the Brighten pediatric trial planned in 2021 and further data from the InSight extension study anticipated in H2 2021. Key financials reveal a decrease in cash equivalents to €75.8 million from €112.0 million in 2019, with a net loss of €46.6 million for 2020. The company strengthened its advisory board and secured a strategic financing deal, providing operational flexibility.
ProQR Therapeutics (Nasdaq: PRQR) announces an Expert Perspectives call on February 22, 2021, at 12:00 PM EST. Led by CMO Aniz Girach and Dr. Paul Yang, the session will discuss key vision measures in Usher Syndrome and non-syndromic Retinitis Pigmentosa, focusing on the Phase 1/2 Stellar trial of QR-421a. QR-421a is a first-in-class RNA therapy targeting vision loss caused by mutations in the USH2A gene. The call will include a Q&A session and an archived presentation will be available for 30 days post-event.
ProQR Therapeutics (Nasdaq: PRQR) announced the completion of patient enrollment in its Phase 2/3 Illuminate trial for sepofarsen, an investigational RNA therapy targeting Leber Congenital Amaurosis 10 (LCA10) caused by a mutation in the CEP290 gene. This marks a significant milestone, with top-line results expected in H1 2022. The study aims to seek marketing approval, as there are currently no approved treatments for LCA10. Sepofarsen has received orphan drug and fast-track designations, highlighting its potential to address a critical unmet medical need.
ProQR Therapeutics (Nasdaq: PRQR) announces participation in the HC Wainwright Virtual BIOCONNECT 2021 Conference, scheduled for January 11, 2021. The company, focused on developing RNA therapies for inherited retinal diseases, aims to transform lives through its proprietary RNA repair platform. A webcast of the presentation will be available on the ProQR website under the 'Investors & Media' section. ProQR emphasizes its commitment to addressing severe genetic rare diseases such as Leber congenital amaurosis 10 and Usher syndrome.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced that its management will participate in a fireside chat at the 3rd Annual Evercore ISI Virtual HealthCONx Conference. The event is scheduled for December 2, 2020, at 3:55 PM EST and will be webcast live. Following the presentation, an archived version will be accessible for 30 days on ProQR's website. ProQR focuses on developing RNA therapies for inherited retinal diseases utilizing its proprietary RNA repair platform.
ProQR Therapeutics reported Q3 2020 results, emphasizing clinical trial advancements and financial stability. The Illuminate Phase 2/3 trial of sepofarsen is projected to complete enrollment in Q1 2021. Data from the QR-421a and QR-1123 trials is expected in 2021, with the QR-504a study on Fuchs to begin in H1 2021. Financially, cash reserves stand at €88.8 million, with a net loss of €13.2 million for the quarter. The company’s runway extends into 2023 due to strategic financing agreements.
ProQR Therapeutics (Nasdaq: PRQR) announced virtual presentations at the American Academy of Ophthalmology Annual Meeting from November 13-15, 2020. Findings will include Phase 1b/2 trial results of sepofarsen for Leber congenital amaurosis 10 (LCA10), presented by Stephen R. Russell, MD. Another presentation by Allen Ho, MD, will focus on patient responses assessed via full-field stimulus testing. Sepofarsen is a pivotal RNA therapy targeting the p.Cys998X mutation in the CEP290 gene, designed to restore normal protein function and has received several designations from health authorities.
ProQR Therapeutics has appointed Naveed Shams, MD, PhD as its new Chief Scientific Officer, succeeding David Rodman, MD. Dr. Shams, with over 20 years of experience in ophthalmology, previously held prominent roles at Santen and Genentech, where he contributed to key drug developments like Lucentis. His expertise will focus on enhancing ProQR's research and development of innovative RNA therapies for inherited retinal diseases. David Rodman will assist during the transition in an advisory capacity.