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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 N.V. (Nasdaq: PRQR) announced virtual presentations at key ophthalmology forums, focusing on advancements in treatments for inherited retinal diseases. Highlights include:
- Ophthalmology Futures Retina Forum: Presentation on managing inherited retinal diseases by CMO Aniz Girach on September 30, 2020.
- Euretina Congress: Results from Phase 1b/2 trial of sepofarsen in Leber congenital amaurosis (LCA10) presented by Stephen R. Russell, available from October 2, 2020.
- AAOpt Meeting: Discussion on early genetic diagnosis and patient referrals in retinal diseases on October 7, 2020.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced participation in several upcoming investor conferences aimed at discussing its focus on RNA medicines for inherited retinal diseases. Key events include the Citi's 15th Annual BioPharma Virtual Conference on September 10, the H.C. Wainwright 22nd Annual Conference on September 15, the Cantor Virtual Global Healthcare Conference on September 16, and the Chardan Virtual 4th Annual Genetic Medicines Conference on October 6. Details of the presentations can be found on the company's website, with archived webcasts available post-presentation.
ProQR Therapeutics (Nasdaq: PRQR) reported positive preliminary data from the InSight extension study of sepofarsen for LCA10, reinforcing confidence in the Phase 2/3 Illuminate trial. A strategic convertible debt financing extends the company’s runway into 2023. ProQR is actively enrolling patients across three clinical-stage RNA therapies, with a fourth program set for clinical testing. Financially, cash and cash equivalents stood at €87.1 million, while net cash used in operating activities was €10.5 million. The net loss for Q2 2020 was €4.1 million, down from €11.7 million in the prior year.
ProQR Therapeutics (Nasdaq: PRQR) has enhanced its Scientific Advisory Board (SAB) with the addition of leading experts in inherited retinal disease and RNA therapies. This strategic move aims to support the advancement of ProQR's RNA therapeutics pipeline. CEO Daniel A. de Boer expressed optimism about the diverse perspectives these experts will contribute. The SAB includes notable figures like James Shannon, Mike Cheetham, and Thaddeus Drija, among others, who collectively bring significant experience in the fields of ophthalmology and RNA drug development.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced a $30 million convertible debt financing agreement with Pontifax Medison Debt Financing to enhance financial flexibility and extend its cash runway into 2023. The deal allows Pontifax to convert the debt into equity at $7.88 per share, a 50% premium to the average share price prior to the agreement. This funding is expected to accelerate the company's RNA therapy pipeline for severe genetic diseases. The financing will occur in three tranches, maturing over 54 months with an initial interest-only period of 24 months.
ProQR Therapeutics (Nasdaq: PRQR) will host an Expert Perspectives call on July 20, 2020, at 5:00 PM EDT, focusing on Leber congenital amaurosis 10 (LCA10) and investigational RNA therapy, sepofarsen. The call will showcase new preliminary data from the ongoing InSight Phase 1/2 extension study, particularly concerning second-eye treatment. Aniz Girach, MD, and Ian MacDonald, MD, will present and interpret the data. Sepofarsen, designed to address the p.Cys998X mutation in the CEP290 gene, has shown promising results with significant vision improvements in trials.
ProQR Therapeutics (Nasdaq: PRQR) announced the launch of its Expert Perspectives call series aimed at discussing topics related to its pipeline and platform. The inaugural call is scheduled for June 22, 2020, at 12 PM EDT, focusing on Usher syndrome and retinitis pigmentosa. It will feature discussions between ProQR's CMO, Aniz Girach, and Dr. Robert Koenekoop from McGill University, covering the investigational therapy QR-421a. Future calls will explore other related topics in genetic rare diseases, emphasizing the company's commitment to advancing RNA therapies.
ProQR Therapeutics (Nasdaq: PRQR) announced a virtual presentation of data from its Phase 1/2 trial of sepofarsen, an RNA therapy for Leber congenital amaurosis 10. The presentation will be made available via ARVO on June 15, 2020, following the cancellation of the annual meeting due to COVID-19. Sepofarsen, designated as an orphan drug, aims to address the genetic mutation causing this rare form of blindness. The trial data, presented by Dr. Stephen R. Russell, will highlight the results of a novel treatment that restores normal function of the CEP290 gene.
ProQR Therapeutics N.V. (NASDAQ: PRQR) announced its Annual General Meeting of Shareholders will be held on June 23, 2020, at 15:00 CET via videoconference. Shareholders can access all relevant documents, including the notice and agenda, on ProQR’s website and the SEC’s website. ProQR is focused on developing transformative RNA therapies for severe genetic rare diseases, leveraging its proprietary RNA repair platform technologies since 2012.
ProQR Therapeutics reported promising interim results from the Phase 1/2 Stellar trial of QR-421a for Usher syndrome and non-syndromic retinitis pigmentosa, with plans for dose expansion and escalation. The company expects to provide updated sepofarsen data in H2 2020. As of March 31, 2020, ProQR held €98.1 million in cash, with a net loss of €16.1 million for Q1 2020. Research and development expenditures increased to €12.8 million, reflecting ongoing clinical trials amidst COVID-19 disruptions. ProQR anticipates sufficient funding to support operations into H2 2022.