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ProQR Therapeutics N.V., trading under the symbol PRQR, is a pioneering biotech company headquartered in Leiden, Netherlands, and Cambridge, Massachusetts. The company is at the forefront of developing RNA therapeutics for patients suffering from severe genetic rare diseases. ProQR's core focus is on leveraging a proprietary RNA editing technology platform known as Axiomer™ to create transformative RNA therapies.
The Axiomer™ technology utilizes a natural cellular mechanism called ADAR (Adenosine Deaminase Acting on RNA) to make precise edits at the RNA level, correcting disease-causing mutations, modulating protein expression, or adding new protein functions to treat diseases. This innovative approach has the potential to yield treatments for various genetic disorders such as cystic fibrosis, Leber's congenital amaurosis type 10, dystrophic epidermolysis bullosa, and beyond.
Recently, ProQR announced a notable collaboration with the Rett Syndrome Research Trust (RSRT) to develop editing oligonucleotides (EONs) using Axiomer™ technology to correct mutations in the transcription factor Methyl CpG binding protein 2 (MECP2), which is associated with Rett syndrome. This collaboration could significantly advance therapeutic options for this rare neurodevelopmental disorder.
Furthermore, the company continues to impress with its cutting-edge research and intellectual property milestones. ProQR has robustly defended its patents globally, ensuring a strong IP estate around its Axiomer technology. Notably, the company successfully defended its patents in Japan and Europe, showcasing the innovative and proprietary nature of its RNA editing platform.
ProQR's pipeline is rapidly advancing, with key programs targeting cholestatic diseases and cardiovascular diseases. The AX-0810 program, targeting the NTCP gene, has shown promising preclinical proof of concept, indicating its potential to become a disease-modifying treatment for conditions like primary sclerosing cholangitis (PSC) and biliary atresia (BA). The AX-1412 program aims to tackle cardiovascular diseases by introducing protective gene variants.
The company also reported positive financial results for the year ended December 31, 2023, with a solid cash position of €118.9 million, up from €94.8 million in 2022. ProQR continues to invest in advancing its RNA editing platform and pipeline, with a strategic focus on entering clinical trials in late 2024 or early 2025.
Under the leadership of CEO Daniel A. de Boer, ProQR is well-positioned to continue its mission of changing lives through transformative RNA therapies, backed by strong scientific evidence, a robust IP portfolio, and strategic collaborations, including a fruitful partnership with Eli Lilly. Keep up with the latest updates and developments by visiting their website at www.proqr.com.
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.
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.
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