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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.
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 N.V. (Nasdaq: PRQR) will hold its Annual General Meeting of Shareholders on May 19, 2021, at 16:00 CET via videoconference. Relevant documents, including the meeting agenda, will be available in the 'Investors & Media' section of their website. ProQR is focused on developing transformative RNA therapies for inherited retinal diseases, aiming to improve the lives of patients with conditions like Leber congenital amaurosis and retinitis pigmentosa.
ProQR Therapeutics (Nasdaq:PRQR) announced positive findings in the scientific journal Nature Medicine regarding its RNA therapy, sepofarsen, for treating Leber Congenital Amaurosis 10 (LCA10). The case report highlights significant visual improvements in a patient treated with sepofarsen, indicating potential for early-stage disease management. This therapy is designed to correct the underlying genetic mutation and has received various designations from regulatory authorities, reflecting its promise in treating this severe condition.
ProQR Therapeutics N.V. (Nasdaq: PRQR) successfully closed an underwritten public offering of 13,846,154 ordinary shares at a price of $6.50 per share, raising approximately $103.5 million in gross proceeds. The offering included the full exercise of the underwriters' option for an additional 2,076,923 shares. Citigroup and Evercore ISI led the offering, with other banks participating as joint managers. The shares are registered under an effective shelf registration statement filed with the SEC. ProQR focuses on developing transformative RNA therapies for inherited retinal diseases.
ProQR Therapeutics N.V. (Nasdaq:PRQR) will present a virtual paper on April 10, 2021, at the AAPOS annual meeting. The presentation focuses on the innovative genetic therapy using antisense oligonucleotide treatment for Leber Congenital Amaurosis 10 (LCA10), specifically targeting the prevalent p.Cys998X mutation in the CEP290 gene. This condition leads to significant vision loss in children, and there are currently no approved treatments. ProQR's investigational RNA therapy, Sepofarsen, aims to address this gap and has received multiple designations from regulatory authorities.
ProQR Therapeutics N.V. (Nasdaq: PRQR) has announced an underwritten public offering of 13,846,154 ordinary shares priced at $6.50 each, aiming for approximately $90 million in gross proceeds. The underwriters have a 30-day option to purchase an additional 2,076,923 shares. The offering is managed by Citigroup and Evercore ISI among others and is set to close on April 2, 2021. This follows a previous shelf registration with the SEC. ProQR focuses on transformative RNA therapies for inherited retinal diseases.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced its intention to conduct an underwritten public offering of ordinary shares. The company plans to use the net proceeds for advancing clinical development of product candidates, progressing RNA-editing technology platforms, and for general corporate purposes. In addition, the underwriters may purchase an additional 15% of the offering within 30 days at the offering price. The offering is subject to market conditions and there are no guarantees on completion or terms.
ProQR Therapeutics announced promising results from the Phase 1/2 Stellar trial of QR-421a, aimed at treating Usher syndrome and non-syndromic retinitis pigmentosa due to USH2A mutations. The study showed significant improvements in vision metrics, such as best corrected visual acuity (BCVA) and retinal imaging. QR-421a was well tolerated with no serious adverse events. Based on these results, ProQR plans to initiate two pivotal Phase 2/3 trials by the end of 2021. This development marks a critical step towards addressing the unmet need for effective therapies in these debilitating retinal diseases.
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.