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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 (Nasdaq: PRQR) reported its Q3 2022 results, focusing on its Axiomer RNA base editing technology for treating genetic disorders. The company continues its partnership with Eli Lilly and plans to unveil initial pipeline targets in early 2023. Financially, ProQR's cash decreased to €100.4 million, with a net loss of €24.4 million (or €0.34 per share). Operating expenses rose, with R&D costs at €15.4 million. Positive developments include the appointment of key management members and a cash runway extending into 2026.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced its participation in the Stifel 2022 Healthcare Conference. The Company's fireside chat is scheduled for November 16, 2022, from 10:20 to 10:50 am EST, with a replay available for 30 days. ProQR specializes in RNA therapies and is pioneering Axiomer®, a technology for making precise RNA edits to treat genetic diseases. The Company emphasizes its commitment to developing transformative therapies aimed at improving patients' lives.
ProQR Therapeutics N.V. (Nasdaq: PRQR) has announced a strategic shift focusing on its Axiomer® RNA editing technology after receiving guidance from the European Medicines Agency (EMA) regarding sepofarsen. An additional pivotal trial for sepofarsen is now required prior to a Marketing Authorisation Application. The company will seek a strategic partner for its ophthalmology assets while winding down current trials, which will lead to workforce reductions but extend its cash runway into 2026.
ProQR Therapeutics has accelerated its Axiomer® RNA base-editing technology and plans to update on initial targets in H2 2022. The company is in discussions with the EMA and FDA regarding data from the Illuminate trial and the regulatory path for ultevursen. Ongoing enrollment in the Sirius Phase 2/3 trial for USH2A-mediated Usher syndrome continues. Financially, ProQR reported €156.4 million in cash as of June 30, 2022, with a net loss of €14.7 million for the quarter. R&D expenses increased to €11.4 million compared to last year.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced its participation in the Life Sci 2nd Annual Genetic Medicines Symposium on June 29, 2022, focusing on the RNA Editing track. The presentation will be webcast live from 10:00 to 10:30 am EDT and available for replay for 30 days. ProQR is pioneering RNA therapies using their Axiomer® technology, aiming to create transformative medicines for genetic diseases. More details can be found on ProQR’s official website.
ProQR Therapeutics (Nasdaq: PRQR) announced key leadership changes and the Annual General Meeting (AGM) on June 30, 2022. René Beukema joins as Chief Corporate Development Officer and General Counsel, while Gerard Platenburg takes on the Chief Scientific Officer role overseeing the Axiomer® RNA-editing platform. John Maraganore extends his role as a strategic advisor to the Supervisory Board. Smital Shah will leave the company at the end of 2022, with a search for a new CFO underway. The company focuses on advancing its RNA therapies and Axiomer technology.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced its participation in the TIDES USA conference from May 9-12, 2022, in Boston, MA. Gerard Platenburg, Co-Founder and Chief Innovation Officer, will present an oral presentation titled Development of RNA Base Editing Technologies for Precision Medicines on May 11, 2022, at 11:15 AM EDT. ProQR is advancing RNA therapies with its proprietary Axiomer® technology, which utilizes the body's own editing system, ADAR, to create targeted RNA modifications, potentially leading to novel treatments for genetic disorders.
ProQR Therapeutics reported Q1 2022 results, revealing a net loss of €14.5 million, or €0.20 per diluted share. The company's cash reserves stood at €167.6 million, down from €187.5 million in Q4 2021. Key highlights include ongoing trials for sepofarsen and ultevursen, with encouraging post-hoc efficacy data from the Illuminate trial. ProQR plans to meet with the EMA and FDA in Q3 to discuss findings and intends to expand its Axiomer RNA editing platform beyond ophthalmology.
Financially, R&D costs increased to €13.4 million from €8.9 million year-over-year.
ProQR Therapeutics (Nasdaq: PRQR) announced presentations at the 7th Annual Retinal Cell and Gene Therapy Innovation Summit on April 29, 2022, and the ARVO Annual Meeting from May 1-4, 2022, in Denver, CO. Dr. Artur Cideciyan presented on AON treatment for CEP290-LCA, while Dr. Bart Leroy discussed the efficacy of sepofarsen in treating LCA10. Notable presentations include long-term safety data and genetic therapies aimed at restoring vision in rare inherited diseases. The company focuses on delivering transformative RNA therapies to combat severe genetic conditions.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced its participation in the Kempen Life Sciences Conference scheduled for April 20, 2022. The company specializes in transformative RNA therapies, utilizing a unique technology called Axiomer® to edit RNA and potentially treat genetic diseases. ProQR aims to develop innovative medicines using its proprietary RNA repair platform, prioritizing patient needs. For further details, visit www.proqr.com.
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