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ProQR Therapeutics BV (PRQR)

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Upturn Advisory Summary
12/05/2025: PRQR (1-star) is currently NOT-A-BUY. Pass it for now.
1 Year Target Price $8.88
1 Year Target Price $8.88
| 6 | Strong Buy |
| 2 | Buy |
| 0 | Hold |
| 0 | Sell |
| 0 | Strong Sell |
Analysis of Past Performance
Type Stock | Historic Profit -64.68% | Avg. Invested days 26 | Today’s Advisory PASS |
Upturn Star Rating ![]() | Upturn Advisory Performance | Stock Returns Performance |
Key Highlights
Company Size Small-Cap Stock | Market Capitalization 220.17M USD | Price to earnings Ratio - | 1Y Target Price 8.88 |
Price to earnings Ratio - | 1Y Target Price 8.88 | ||
Volume (30-day avg) 8 | Beta 0.17 | 52 Weeks Range 1.07 - 3.52 | Updated Date 12/7/2025 |
52 Weeks Range 1.07 - 3.52 | Updated Date 12/7/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) -0.48 |
Analyzing Revenue: Products, Geography and Growth
Revenue by Products
Product revenue - Year on Year
Earnings Date
Report Date - | When - | Estimate - | Actual - |
Profitability
Profit Margin -264.83% | Operating Margin (TTM) -387.1% |
Management Effectiveness
Return on Assets (TTM) -24.02% | Return on Equity (TTM) -103.76% |
Valuation
Trailing PE - | Forward PE - | Enterprise Value 114936235 | Price to Sales(TTM) 13.7 |
Enterprise Value 114936235 | Price to Sales(TTM) 13.7 | ||
Enterprise Value to Revenue 6.36 | Enterprise Value to EBITDA -2.1 | Shares Outstanding 105345192 | Shares Floating 65453075 |
Shares Outstanding 105345192 | Shares Floating 65453075 | ||
Percent Insiders 18.04 | Percent Institutions 50.14 |
Upturn AI SWOT
ProQR Therapeutics BV

Company Overview
History and Background
ProQR Therapeutics BV is a biopharmaceutical company founded in 2012, dedicated to developing RNA therapeutics for rare genetic diseases. The company has focused on developing oligonucleotide-based therapies to address the underlying cause of diseases. Significant milestones include progressing its lead candidates through clinical trials and establishing strategic partnerships.
Core Business Areas
- RNA Therapeutics Development: ProQR Therapeutics BV focuses on the discovery, development, and commercialization of RNA-based medicines. Their platform aims to correct gene defects at the RNA level, offering potential treatments for a range of rare genetic disorders.
Leadership and Structure
ProQR Therapeutics BV is led by a management team with expertise in biotechnology and drug development. The organizational structure is typical of a clinical-stage biopharmaceutical company, with departments dedicated to research and development, clinical operations, regulatory affairs, and business development.
Top Products and Market Share
Key Offerings
- QR-414 (now Ulsa): A topically administered RNA therapeutic designed to restore functional CEP290 in individuals with Leber congenital amaurosis (LCA10). Competitors in the LCA space include other gene therapy and small molecule developers. Market share data is not yet available as the product is in clinical development and recently received orphan drug designation.
- QR-421a: An investigational RNA therapeutic targeting a specific mutation in the USH2A gene for Usher syndrome type 2A and non-syndromic Retinitis Pigmentosa. Competitors include companies developing gene therapies and other oligonucleotide-based approaches. Market share data is not applicable as it is in clinical development.
Market Dynamics
Industry Overview
The rare genetic diseases market is characterized by high unmet medical needs and significant scientific innovation. The development of RNA therapeutics is a rapidly growing segment, offering targeted approaches to diseases previously considered untreatable. Regulatory pathways for rare diseases are often expedited.
Positioning
ProQR Therapeutics BV positions itself as a pioneer in RNA therapeutics for rare genetic diseases. Its strength lies in its proprietary RNA-targeting platform and a pipeline focused on diseases with clear genetic causes. Competitive advantages include its focus on specific genetic mutations and its clinical-stage assets.
Total Addressable Market (TAM)
The total addressable market for rare genetic diseases is substantial and growing, driven by increased diagnosis, genetic screening, and therapeutic advancements. ProQR Therapeutics BV is positioned to address specific segments within this broad market with its targeted therapies. Quantifying ProQR's specific TAM for its pipeline candidates requires further analysis of patient populations for each indicated disease.
Upturn SWOT Analysis
Strengths
- Proprietary RNA-targeting platform
- Focus on rare genetic diseases with high unmet needs
- Clinical-stage pipeline with promising candidates
- Experienced management team
Weaknesses
- Reliance on a few key pipeline candidates
- Long and costly drug development process
- Potential for clinical trial failures
- Limited commercialization experience
Opportunities
- Advancements in RNA therapeutic technology
- Expansion into new rare disease indications
- Strategic partnerships and collaborations
- Favorable regulatory pathways for rare diseases
Threats
- Competition from other biotechnology companies
- Changes in regulatory requirements
- Reimbursement challenges for novel therapies
- Patent expirations and generic competition (in the long term)
Competitors and Market Share
Key Competitors
- Editas Medicine (EDIT)
- Intellia Therapeutics (NTLA)
- CRISPR Therapeutics (CRSP)
- Alnylam Pharmaceuticals (ALNY)
Competitive Landscape
ProQR Therapeutics BV competes in the gene editing and RNA therapeutics space. Its advantages lie in its specific focus on oligonucleotide-based therapies and a curated pipeline for rare genetic diseases. However, it faces strong competition from companies with more advanced gene editing platforms and larger development pipelines. The competitive landscape is dynamic, with significant investment and rapid scientific advancement.
Growth Trajectory and Initiatives
Historical Growth: Historical growth for ProQR Therapeutics BV has been characterized by the progression of its pipeline, expansion of its research capabilities, and securing funding. Revenue growth has been minimal to non-existent as it is pre-commercial.
Future Projections: Future growth projections are heavily dependent on the successful clinical development and regulatory approval of its lead drug candidates, particularly QR-414 and QR-421a. Analyst estimates would focus on potential peak sales of these therapies and the expansion of the pipeline into new indications.
Recent Initiatives: Recent initiatives likely include advancing its lead candidates into later-stage clinical trials, forging strategic partnerships to support development or commercialization, and potentially seeking regulatory designations or approvals.
Summary
ProQR Therapeutics BV is a promising player in the rare genetic disease RNA therapeutics space. Its core strength lies in its proprietary technology and a focused pipeline addressing significant unmet needs. However, the company faces the inherent risks and long development timelines typical of biopharmaceutical development, with significant competition from established players in gene editing and RNA therapeutics. Continued clinical success and strategic partnerships will be crucial for its future growth.
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Sources and Disclaimers
Data Sources:
- Company SEC Filings (e.g., 10-K, 10-Q)
- Industry Research Reports
- Financial News Outlets
- Biopharmaceutical Databases
Disclaimers:
This JSON output is for informational purposes only and does not constitute financial advice. Data is based on publicly available information and may not be exhaustive or completely up-to-date. Investment decisions should be made in consultation with a qualified financial advisor.
AI Summarization is directionally correct and might not be accurate.
Summarized information shown could be a few years old and not current.
Fundamental Rating based on AI could be based on old data.
AI-generated summaries may have inaccuracies (hallucinations). Please verify the information before taking action.
About ProQR Therapeutics BV
Exchange NASDAQ | Headquaters - | ||
IPO Launch date 2014-09-18 | Founder, CEO & Member of Management Board Mr. Daniel Anton de Boer | ||
Sector Healthcare | Industry Biotechnology | Full time employees 166 | Website https://www.proqr.com |
Full time employees 166 | Website https://www.proqr.com | ||
ProQR Therapeutics N.V., a biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company's products pipeline includes AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP); and AX-1412 for cardiovascular diseases (CVDs) targeting Beta-1,4-galactosyltransferase 1 (B4GALT1). It also develops various other early-stage research programs, including AX-1005 for undisclosed targets in CVDs; AX-2402, which focuses on Rett syndrome; AX-2911 for metabolic dysfunction-associated steatohepatitis (MASH) targeting patatin-like phospholipase domain-containing protein 3 (PNPLA3); AX-0601 for obesity and Type 2 diabetes; and AX-9115 for rare metabolic condition, as well as additional programs in Rett Syndrome, additional CNS programs, and multiple other unnamed targets and programs. In addition, the company develops Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center; Inserm Transfert SA; Ionis Pharmaceuticals, Inc.; Vico Therapeutics B.V.; University of Rochester; and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands.

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