Welcome to our dedicated page for Proqr Therapeuti news (Ticker: PRQR), a resource for investors and traders seeking the latest updates and insights on Proqr Therapeuti stock.
ProQR Therapeutics N.V. (Nasdaq: PRQR) generates a steady flow of news as a clinical-stage biopharmaceutical company advancing RNA editing therapies. Its updates center on the Axiomer RNA base editing platform and a pipeline of editing oligonucleotides designed to correct or modulate disease-related targets at the RNA level. News coverage on this page reflects company announcements, clinical milestones, financial updates, and scientific presentations related to its programs in liver, CNS, and other genetically defined diseases.
Investors and followers of PRQR can expect news about the progress of AX-0810, ProQR’s lead investigational RNA editing oligonucleotide targeting NTCP for cholestatic diseases. Recent releases describe the submission and authorization of a Clinical Trial Application, the initiation of a Phase 1 first-in-human study in healthy volunteers in the Netherlands, and early safety and pharmacokinetic observations from the first cohort. Updates also outline plans for target engagement readouts and the future inclusion of a patient cohort in the same trial.
News items also highlight earlier-stage pipeline programs such as AX-2402 for Rett syndrome (MECP2 R270X), AX-2911 for metabolic liver disease including MASH (PNPLA3 I148M), and AX-1412 for cardiovascular disease (B4GALT1). ProQR reports non-clinical proof-of-concept data, development candidate selections, and functional results in disease-relevant models. Financial press releases provide quarterly operating and financial results, cash and cash equivalents, collaboration revenue from Eli Lilly, and comments on cash runway.
Additional PRQR news covers participation in scientific and investor conferences, including ASGCT, the RNA Editing Summit, OTS, TIDES USA, and healthcare investor events. These communications feature presentations on ADAR-mediated RNA editing of NTCP, PNPLA3, and MECP2, as well as platform-focused talks on optimizing editing oligonucleotides for therapeutic use. Bookmark this page to follow ProQR’s disclosed clinical data, pipeline milestones, collaboration developments, and financial reporting as they are released through press announcements and SEC-furnished materials.
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.
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.
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