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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) hosted a virtual Analyst Event on November 18, 2021, highlighting its clinical-stage RNA therapies for genetic eye diseases. Key updates include the Phase 2/3 Illuminate trial for sepofarsen, with top-line results expected in Q1/Q2 2022, showing potential visual acuity improvements. Additionally, data from the QR-421a and QR-1123 trials indicated benefits in vision for Usher syndrome and retinitis pigmentosa patients. ProQR is advancing its pipeline with strategic clinical trials, aiming for impactful RNA therapies.
ProQR Therapeutics N.V. (Nasdaq: PRQR) will host a virtual Analyst Event on November 18, 2021, from 12-2 PM ET. The event will showcase advancements in its clinical pipeline and the Axiomer® RNA editing platform. Key highlights include data from the Phase 1/2 InSight extension study of sepofarsen and an overview of the Phase 2/3 Illuminate trial. Attendees can pre-register for the live webcast or access the archived session on ProQR’s website. ProQR focuses on transformative RNA therapies for genetic eye diseases such as Leber congenital amaurosis and retinitis pigmentosa.
ProQR Therapeutics N.V. (Nasdaq: PRQR) has announced its participation in two upcoming investor conferences. The conferences include the Stifel 2021 Virtual Healthcare Conference on November 15 and the Evercore ISI 4th Annual HealthCONx Conference on December 2. Each session will feature a fireside chat with company management, offering insights into ProQR's innovative RNA therapies aimed at treating genetic eye diseases. Details will be available on ProQR's website, with archived webcasts accessible for 30 days post-event.
ProQR Therapeutics reported Q3 2021 results, showing a net loss of €15.1 million or €0.22 per share, an increase from a €13.2 million loss year-over-year. The company holds €156.1 million in cash and equivalents. Key developments include the anticipated top-line data from the Phase 2/3 Illuminate trial for sepofarsen in late Q1 2022, and a collaboration with Lilly involving a $50 million upfront payment and potential milestones of $1.25 billion. Upcoming analyst event on November 18, 2021, will outline clinical advancements and the Axiomer RNA editing platform.
ProQR Therapeutics (Nasdaq: PRQR) has appointed Theresa Heggie as Chief Commercial Officer, effective immediately. Ms. Heggie brings extensive experience in global rare disease commercialization, previously holding senior roles at Alnylam and Shire. Her appointment coincides with the upcoming top-line data from the Phase 2/3 pivotal Illuminate trial of sepofarsen for CEP290-mediated LCA10, expected in H1 2022. This leadership change is aimed at strengthening the company’s commercial strategy and preparations for the trial results.
ProQR Therapeutics (Nasdaq: PRQR) announced an investor conference call on September 9, 2021, at 8:15 am EDT to discuss the Axiomer RNA editing technology platform after its partnership with Eli Lilly. Axiomer aims to create transformative RNA therapies for genetic eye diseases by inducing specific nucleotide changes using the cell's machinery. The live and archived webcast will be available on their website. This technology could address over 20,000 known G to A mutations causing human diseases, providing significant potential for medical advancements.
ProQR Therapeutics has entered a global licensing and research collaboration with Eli Lilly, focusing on developing RNA editing therapies for genetic disorders affecting the liver and nervous system. ProQR will receive an upfront payment of $20 million and a $30 million equity investment, totaling $50 million. The company is also eligible for up to $1.25 billion in milestone payments and royalties from product sales. The collaboration will utilize ProQR's Axiomer® platform technology, allowing precise RNA edits to potentially reverse over 20,000 disease-causing mutations.
ProQR Therapeutics (Nasdaq: PRQR) announced its participation in the EURETINA virtual congress from September 9-12, 2021, where it will present data from the Phase 1/2 Stellar trial of its investigational RNA therapy QR-421a. This therapy targets vision loss due to mutations in the USH2A gene, the leading cause of combined deafness and blindness. The presentation, led by Dr. David G. Birch, will mark the first public data presentation from the Stellar trial.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced participation in various upcoming investor conferences focused on RNA therapies for genetic eye diseases. Key events include:
- Citi’s 16th Annual BioPharma Virtual Conference on September 9, 4:10-4:55 pm EDT.
- HC Wainwright 23rd Annual Global Investment Conference available on demand from September 13, 7am EDT.
- Cantor Virtual Global Healthcare Conference on September 28, 3:20-3:50 pm EDT.
- Chardan’s 5th Annual Genetic Medicines Conference on October 5, 4:00-4:30 pm EDT.
Details are available on the company’s website.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced that its management will present at the HC Wainwright Ophthalmology Virtual Conference. The presentation will be available on demand starting August 17, 2021, at 7:00 am EDT. An archived version will be accessible for 30 days on the company’s website. ProQR is committed to developing transformative RNA therapies for genetic eye diseases, leveraging its proprietary RNA repair technologies.
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