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ProQR Therapeutics BV (PRQR)



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Upturn Advisory Summary
06/27/2025: PRQR (1-star) has a low Upturn Star Rating. Not recommended to BUY.
Year Target Price $8.88
Year Target Price $8.88
6 | Strong Buy |
2 | Buy |
0 | Hold |
0 | Under performing |
0 | Sell |
Analysis of Past Performance
Type Stock | Historic Profit -53.38% | Avg. Invested days 25 | Today’s Advisory Consider higher Upturn Star rating |
Upturn Star Rating ![]() ![]() | Upturn Advisory Performance ![]() | Stock Returns Performance ![]() |
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Key Highlights
Company Size Small-Cap Stock | Market Capitalization 214.90M USD | Price to earnings Ratio - | 1Y Target Price 8.88 |
Price to earnings Ratio - | 1Y Target Price 8.88 | ||
Volume (30-day avg) - | Beta 0.31 | 52 Weeks Range 1.07 - 4.62 | Updated Date 06/29/2025 |
52 Weeks Range 1.07 - 4.62 | Updated Date 06/29/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) -0.39 |
Earnings Date
Report Date - | When - | Estimate - | Actual - |
Profitability
Profit Margin -153.8% | Operating Margin (TTM) -228.14% |
Management Effectiveness
Return on Assets (TTM) -15.07% | Return on Equity (TTM) -53.07% |
Valuation
Trailing PE - | Forward PE - | Enterprise Value 79792727 | Price to Sales(TTM) 10.95 |
Enterprise Value 79792727 | Price to Sales(TTM) 10.95 | ||
Enterprise Value to Revenue 3.6 | Enterprise Value to EBITDA -2.1 | Shares Outstanding 105343000 | Shares Floating 66028969 |
Shares Outstanding 105343000 | Shares Floating 66028969 | ||
Percent Insiders 18.06 | Percent Institutions 50.95 |
Analyst Ratings
Rating 4.75 | Target Price 8.88 | Buy 2 | Strong Buy 6 |
Buy 2 | Strong Buy 6 | ||
Hold - | Sell - | Strong Sell - | |
Strong Sell - |
Upturn AI SWOT
ProQR Therapeutics BV

Company Overview
History and Background
ProQR Therapeutics BV was founded in 2012 and focuses on developing RNA therapies for genetic eye diseases. They aim to correct genetic defects using oligonucleotide-based therapeutics.
Core Business Areas
- RNA-Based Therapeutics Development: ProQR's primary focus is on designing and developing RNA-based therapeutics to treat genetic eye disorders. This includes target identification, drug discovery, and clinical development.
- Clinical Trials and Regulatory Approval: Conducting clinical trials to evaluate the safety and efficacy of their therapeutic candidates and pursuing regulatory approvals for commercialization.
- Collaboration and Partnerships: Entering into strategic collaborations and partnerships with other pharmaceutical companies and research institutions to advance their pipeline and expand their market reach.
Leadership and Structure
The company has a management team led by a CEO and various functional heads (e.g., R&D, Clinical Development, etc.). The organizational structure includes research, development, and commercial teams.
Top Products and Market Share
Key Offerings
- Sepofarsen: Sepofarsen is ProQR's lead product candidate in clinical development for the treatment of CEP290-mediated Leber congenital amaurosis 10 (LCA10), a rare inherited retinal disease. Its market share is currently non-existent as it awaits FDA approval or trials. Competitors are companies developing gene therapies or other treatments for LCA, such as Editas Medicine (EDIT).
- QR-421a: QR-421a is another investigational RNA-based therapy being developed for Usher syndrome type 2A. Clinical trials are ongoing. Its market share is also nonexistent, awaiting potential future FDA approval. Competitors exist in the broader inherited retinal disease therapeutic space.
Market Dynamics
Industry Overview
The biopharmaceutical industry, specifically RNA therapeutics, is rapidly growing with increasing investment and technological advancements. There's a significant unmet need for treatments for rare genetic diseases, including inherited retinal disorders.
Positioning
ProQR positions itself as a leader in RNA-based therapies for genetic eye diseases, focusing on precision medicine approaches. Their competitive advantage lies in their proprietary RNA technology and focus on a specific therapeutic area.
Total Addressable Market (TAM)
The TAM for genetic eye diseases is substantial, estimated to be in the billions of dollars, with a growing prevalence of these conditions globally. ProQR is positioned to capture a portion of this market with successful clinical trials and regulatory approvals. Based on prevalence, the LCA TAM alone could be hundreds of millions of dollars.
Upturn SWOT Analysis
Strengths
- Proprietary RNA therapy platform
- Focus on a specific therapeutic area (genetic eye diseases)
- Advanced clinical programs for lead candidates
- Experienced management team
Weaknesses
- Dependence on successful clinical trial outcomes
- High cash burn rate
- Regulatory approval risks
- Relatively small company size
Opportunities
- Expanding pipeline with new therapeutic targets
- Partnering with larger pharmaceutical companies
- Receiving orphan drug designation and priority review
- Positive clinical trial results driving market value
Threats
- Competition from larger pharmaceutical companies
- Clinical trial failures
- Regulatory hurdles
- Patent disputes
Competitors and Market Share
Key Competitors
- EDIT
- CRSP
- AGEN
Competitive Landscape
ProQR faces competition from larger, more established pharmaceutical companies with greater resources. Their advantage is their specialized focus on RNA therapies for genetic eye diseases. However, EDIT, CRSP and AGEN have significantly larger market caps and more diverse pipelines.
Growth Trajectory and Initiatives
Historical Growth: Historical growth has been limited due to the early stage of the company and lack of approved products. Growth strategy focused on clinical trials
Future Projections: Future growth depends on successful clinical trial results and regulatory approvals. Analyst estimates are unavailable after delisting.
Recent Initiatives: Recent initiatives have centered around advancing clinical trials for Sepofarsen and QR-421a.
Summary
ProQR Therapeutics BV is a biopharmaceutical company focused on RNA-based therapies for genetic eye diseases, particularly in phase 2/3 clinical trials for inherited disorders such as Leber congenital amaurosis 10 (LCA10). The company has faced significant challenges recently, including financial difficulties and a delisting from the NASDAQ stock exchange. Success is highly dependent on positive outcomes from ongoing clinical trials, regulatory approval, and securing financial resources. The company's expertise in RNA-based therapeutics and specific focus on ophthalmology are its key strengths, but it must overcome substantial financial and regulatory hurdles.
Peer Comparison
Sources and Disclaimers
Data Sources:
- Company filings
- Financial news sources
- Industry reports
Disclaimers:
The information provided is for informational purposes only and does not constitute financial advice. Investment decisions should be made based on individual circumstances and consultation with a financial advisor. Market share estimates are approximate and based on available data.
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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