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Proqr Therapeuti - PRQR STOCK NEWS

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.

Overview of ProQR Therapeutics

ProQR Therapeutics is a Netherlands-based biopharmaceutical company dedicated to the discovery and development of transformative RNA therapies. Utilizing its proprietary Axiomer RNA editing platform, ProQR focuses on addressing severe genetic disorders by correcting disease-causing mutations at the RNA level. With a foundation built on breakthrough science and by industry experts, the company is committed to enhancing patient outcomes for unreached medical needs.

Innovative RNA Editing Technology

The cornerstone of ProQR's approach is its Axiomer platform. This technology harnesses the naturally occurring enzyme ADAR (Adenosine Deaminase Acting on RNA) to perform precise, single nucleotide modifications within target RNA molecules. By converting an adenine (A) to an inosine (I) — ultimately interpreted as guanine (G) during protein synthesis — the platform offers a way to restore normal function or adjust protein expression. The use of chemically modified editing oligonucleotides within the Axiomer process enhances stability and efficiency, ensuring robust and targeted RNA editing.

Pipeline and Therapeutic Focus

ProQR Therapeutics has built a diverse pipeline that targets a range of rare and genetic conditions. The company is actively developing treatment strategies for diseases that currently have limited therapeutic options, such as rare forms of cystic fibrosis, Leber's congenital amaurosis, dyastrophic epidermolysis bullosa, and Rett syndrome. Each program is underpinned by a deep scientific rationale, supported by human genetic data that inform the design of editing oligonucleotides. This tailored approach allows ProQR to focus on the most critical disease drivers, offering hope for significant clinical benefits.

Strategic Collaborations and Intellectual Property

Integral to its business model, ProQR has forged strategic partnerships with established industry players. Collaborations with companies like Eli Lilly serve as a cornerstone for both technological advancement and funding support. Through these alliances, ProQR attains valuable insights as well as financial resources to drive its research and early-stage clinical studies. Additionally, its expansive intellectual property portfolio, built over several years of innovation, protects its cutting-edge RNA editing technology and provides a competitive advantage in the evolving landscape of RNA therapeutics.

Operational Excellence and Research Commitment

The company employs a rigorous research and development approach that is characterized by robust preclinical proof of concept studies and translational research. Its operational excellence is further demonstrated by the strategic organization of its research programs and active pursuit of regulatory milestones. ProQR’s detailed scientific evaluation and continuous platform enhancements underscore its commitment to using precise biotechnology to pave new avenues in drug development.

Position in the Biopharmaceutical Landscape

Within a competitive biotech environment, ProQR Therapeutics distinguishes itself through its unique scientific methodology and a comprehensive focus on RNA-based interventions. The company’s clear emphasis on RNA editing — a field at the intersection of genetics and molecular medicine — positions it as a forward-thinking participant in biopharmaceutical innovation. By systematically addressing the genetic underpinnings of rare disorders with unmet needs, ProQR has carved a niche that not only reflects deep industry expertise but also underscores its dedication to changing the therapeutic paradigm.

In summary, ProQR Therapeutics embodies a blend of advanced science, strategic collaboration, and cutting-edge technology. Its approach to harnessing RNA editing for therapeutic purposes reflects an informed and measured strategy aimed at delivering novel treatment options for patients with severe genetic disorders. The company's structured research, robust IP estate, and broad industry partnerships collectively affirm its relevance in today’s innovation-driven biopharmaceutical market.

Rhea-AI Summary

ProQR Therapeutics (NASDAQ: PRQR) has reported its financial and operating results for 2024, highlighting significant progress in its RNA editing pipeline. The company ended 2024 with €149.4 million in cash, providing runway into mid-2027.

Key developments include anticipated clinical data readouts for four programs in 2025-2026, led by AX-0810 targeting NTCP for Cholestatic diseases. The company successfully completed an $82.1 million public offering in October 2024 and expanded its collaboration with the Rett Syndrome Research Trust for up to $9.2 million.

Financial highlights show research and development costs increased to €36.4 million in 2024 (vs €25.1 million in 2023), while general and administrative costs decreased to €13.7 million (vs €16.2 million in 2023). Net loss for 2024 was €27.8 million (€0.32 per share), compared to €27.7 million (€0.35 per share) in 2023.

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ProQR Therapeutics (NASDAQ: PRQR) announced an expanded collaboration with the Rett Syndrome Research Trust (RSRT), securing an additional $8.1 million in funding, bringing the total to $9.1 million. The funding will support the advancement of AX-2402, a therapeutic candidate based on ProQR's Axiomer RNA editing platform, into clinical trials.

The treatment targets individuals with Rett syndrome who have the R270X mutation in MECP2 gene. Rett Syndrome, affecting approximately 350,000 people worldwide, predominantly girls, is a rare neurodevelopmental disorder with no current cure. The expanded collaboration focuses on optimizing therapeutic candidates targeting the MECP2 protein and advancing them toward clinical development.

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ProQR Therapeutics (Nasdaq: PRQR) has appointed Dr. Peter Beal as Chief ADAR Scientist. Dr. Beal, a professor of Chemistry at the University of California, Davis, and a former Scientific Advisory Board member, will oversee platform optimization and scientific developments in RNA editing oligonucleotides design.

As Chief ADAR Scientist, Dr. Beal will leverage his extensive expertise in ADAR biology and RNA chemistry to advance ProQR's Axiomer™ RNA editing technology platform. His research has focused on ADAR-mediated RNA-editing mechanisms, including applications for treating conditions like Rett syndrome, Alzheimer's disease, and Parkinson's disease.

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ProQR Therapeutics (Nasdaq: PRQR) announced a virtual Analyst and Investor Event scheduled for December 11, 2024, from 10:00 am to 12:30 pm EST. The event will showcase the company's Axiomer™ ADAR-mediated RNA editing platform and provide updates on development candidates, including NTCP and B4GALT1 programs, AX-0810, and AX-1412.

Key presenters include Daniel A. de Boer (Founder and CEO), Gerard Platenburg (Chief Scientific Officer), and René Beukema (Chief Corporate Development Officer). Guest speakers include Dr. Peter Beal, Professor at UC Davis and expert in ADAR enzymes, and Prof. Gideon Hirschfield, specialist in autoimmune liver diseases from the Toronto Centre for Liver Disease.

The event will be accessible via webcast on ProQR's website, with a phone option available through pre-registration. A replay will be available for approximately 30 days following the event.

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ProQR Therapeutics reported Q3 2024 financial results, highlighting €89.4 million in cash and cash equivalents, plus $82.1 million from October financing extending runway into mid-2027. The company's first Axiomer™ RNA editing program, AX-0810, targets NTCP for cholestatic diseases and is advancing toward clinical trials. Q3 net loss was €8.1 million (€0.10 per share), compared to €5.4 million (€0.07 per share) in Q3 2023. R&D costs increased to €9.4 million from €5.4 million year-over-year, while G&A costs remained stable at €3.3 million. The company achieved $5.5 million in milestones from its Eli Lilly collaboration during the first three quarters of 2024.

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ProQR Therapeutics announced the closing of its underwritten public offering of 18,000,000 ordinary shares at $3.50 per share, raising gross proceeds of $63.0 million. Additionally, the company completed a concurrent private placement with Eli Lilly and Company for 3,523,538 ordinary shares at the same price, generating approximately $12.3 million in gross proceeds. The combined offerings total $75.3 million in gross proceeds. Evercore ISI, Cantor, Raymond James and Oppenheimer & Co. served as joint lead bookrunning managers for the public offering.

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ProQR Therapeutics (Nasdaq: PRQR) has priced an underwritten public offering of 18 million ordinary shares at $3.50 per share, raising gross proceeds of $63.0 million. The company also granted underwriters a 30-day option to purchase up to 2.7 million additional shares. Concurrently, Eli Lilly agreed to purchase 3.52 million shares in a private placement at the same price to maintain its 16.4% ownership stake, contributing additional gross proceeds of $12.3 million. The combined proceeds will fund research, development, and clinical programs, along with working capital and general corporate purposes. The offering is expected to close on October 24, 2024.

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ProQR Therapeutics N.V. (Nasdaq: PRQR), a company focused on RNA therapies using its Axiomer™ RNA editing technology platform, has announced a proposed underwritten public offering of its ordinary shares. The company intends to grant underwriters a 30-day option to purchase up to an additional 15% of the offering amount. Proceeds from the offering will be used to fund research and development, clinical development, and general corporate purposes.

Joint lead bookrunning managers for the offering include Evercore ISI, Cantor, Raymond James, and Oppenheimer & Co. A shelf registration statement on Form F-3 has been filed with the SEC and declared effective. The offering is subject to market conditions, and there is no assurance of its completion or terms.

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ProQR Therapeutics NV (Nasdaq: PRQR), a company focused on transformative RNA therapies using its proprietary Axiomer™ RNA editing technology platform, has announced its participation in the 2024 Cantor Global Healthcare Conference. The event is scheduled for Tuesday, September 17 at 9:10 am ET in New York City. Company management will present in a fireside chat format, which will be accessible via live webcast from the 'Investors & Media' section of ProQR's website under 'Events'. For those unable to attend live, a replay of the webcast will be available for approximately 30 days following the presentation.

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ProQR Therapeutics NV (Nasdaq: PRQR) reported its Q2 2024 financial and operating results. Key highlights include:

- AX-0810 (for cholestatic diseases) and AX-1412 (for cardiovascular diseases) programs advancing to clinic in late 2024/early 2025
- $4.5 million in milestones achieved in H1 from Lilly partnership
- €96.2 million cash position as of Q2 end, providing runway into mid-2026
- Net loss of €2.7 million (€0.03 per diluted share) for Q2 2024
- R&D costs increased to €7.1 million in Q2 2024 from €5.9 million in Q2 2023
- General and administrative costs decreased to €3.0 million in Q2 2024 from €4.1 million in Q2 2023

ProQR plans to announce translational data and clinical trial designs for AX-0810 and AX-1412 in H2 2024.

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FAQ

What is the current stock price of Proqr Therapeuti (PRQR)?

The current stock price of Proqr Therapeuti (PRQR) is $1.13 as of April 8, 2025.

What is the market cap of Proqr Therapeuti (PRQR)?

The market cap of Proqr Therapeuti (PRQR) is approximately 102.1M.

What is the core focus of ProQR Therapeutics?

ProQR Therapeutics is dedicated to developing RNA therapies targeting severe genetic disorders by correcting disease-causing mutations using its Axiomer RNA editing platform.

How does the Axiomer RNA editing platform work?

The Axiomer platform leverages the ADAR enzyme to perform precise single nucleotide edits in RNA. This process can correct mutations by converting adenine to inosine, thereby modulating protein expression.

Which medical conditions does ProQR target?

The company focuses on rare and genetic diseases, including forms of cystic fibrosis, Leber's congenital amaurosis, dystrophic epidermolysis bullosa, and Rett syndrome, among others.

How does ProQR Therapeutics generate value in its business model?

ProQR generates value through its advanced RNA editing technology, strategic research collaborations, and a robust intellectual property portfolio that underpins its diverse therapeutic pipeline.

What main advantages does the Axiomer platform offer?

Axiomer offers targeted and precise RNA editing by utilizing endogenous cellular machinery, which may lead to effective treatments for genetic disorders through accurate correction of RNA mutations.

How does the company differentiate itself from competitors?

ProQR stands out through its proprietary RNA editing technology, extensive IP portfolio, and key partnerships with established industry leaders, positioning it uniquely in the biopharmaceutical landscape.

What role do strategic partnerships play at ProQR?

Partnerships with entities like Eli Lilly bolster ProQR’s research capabilities and financial resources, enabling accelerated development and validation of its RNA editing programs.

How does ProQR ensure the quality and stability of its RNA therapeutics?

By integrating chemically modified editing oligonucleotides and rigorous preclinical studies, ProQR ensures its treatments are both precise and stable, reinforcing the potential for clinical impact.
Proqr Therapeuti

Nasdaq:PRQR

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PRQR Stock Data

102.08M
86.21M
18.06%
53.87%
0.85%
Biotechnology
Healthcare
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Netherlands
Leiden