Welcome to our dedicated page for ProQR Therapeutics N.V. Ordinary Shares news (Ticker: PRQR), a resource for investors and traders seeking the latest updates and insights on ProQR Therapeutics N.V. Ordinary Shares stock.
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 reported its Q2 2021 financial results, highlighting significant progress in its clinical trials and partnerships. The Illuminate trial of sepofarsen for LCA10 is set to release top-line data in H1 2022. The QR-421a trials for Usher syndrome and retinitis pigmentosa aim to start by year-end 2021, while initial data from the QR-1123 trial is expected in Q4 2021. Financially, ProQR had €139.4 million in cash as of June 30, 2021, with a net loss of €15.8 million for the quarter. New partnerships and a public offering bolstered its financial position.
ProQR Therapeutics (Nasdaq: PRQR) announced its management will present during a fireside chat at the JMP Securities Life Sciences Conference on June 17, 2021, from 3:00 to 3:25 pm EDT. The presentation will be available via a live webcast on ProQR's website under the Investors & Media section, with an archived version accessible for 30 days post-event. ProQR focuses on transformative RNA therapies for genetic eye diseases such as Leber congenital amaurosis 10 and Usher syndrome, utilizing proprietary RNA repair technologies.
ProQR Therapeutics reported significant progress in Q1 2021, completing enrollment for the pivotal Illuminate trial of sepofarsen for LCA10. Positive Phase 1/2 data for QR-421a was shared, with plans for two pivotal Phase 2/3 trials by year-end. The company secured $103.5 million from a public offering, bolstering its balance sheet for further clinical developments. Q1 2021 net loss was €12.6 million, improving from €16.1 million year-over-year. Upcoming milestones include top-line results from Illuminate anticipated in H1 2022.
Yarrow Biotechnology, formed by RTW Investments, has licensed ProQR Therapeutics' antisense oligonucleotide (ASO) technology for non-ophthalmology applications. ProQR stands to gain up to $115 million in upfront and milestone payments, alongside royalties on future sales. ProQR will also receive an undisclosed equity stake in Yarrow. The partnership aims to develop ASO-based therapies targeting disorders with significant unmet needs. Roderick Wong from RTW believes in the transformative potential of RNA-based therapies in genetically-defined diseases.
ProQR Therapeutics N.V. (Nasdaq:PRQR) announced a virtual presentation at ARVO on May 6, 2021, detailing data from its Phase 1/2 InSight trial of sepofarsen, aimed at treating Leber Congenital Amaurosis type 10 (LCA10). This presentation follows earlier discussions from July 2020, with new data expected in H2 2021. Currently, there are no approved treatments for LCA10, affecting around 2,000 individuals in the Western world. Sepofarsen is a pioneering RNA therapy addressing the underlying genetic cause, with significant designations from regulatory bodies.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced its participation in the Kempen Life Sciences Conference 2021 on April 28, 2021, hosting 1x1 meetings. The company focuses on developing transformative RNA therapies for inherited retinal diseases, such as Leber congenital amaurosis 10, Usher syndrome, and retinitis pigmentosa. ProQR utilizes proprietary RNA repair platform technologies to advance its pipeline, emphasizing patient-centered approaches.
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.
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