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Uniqure NV (QURE)



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Upturn Advisory Summary
08/28/2025: QURE (1-star) has a low Upturn Star Rating. Not recommended to BUY.
1 Year Target Price $37.22
1 Year Target Price $37.22
9 | Strong Buy |
2 | Buy |
2 | Hold |
0 | Sell |
0 | Strong Sell |
Analysis of Past Performance
Type Stock | Historic Profit -55.93% | Avg. Invested days 19 | 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 895.98M USD | Price to earnings Ratio - | 1Y Target Price 37.22 |
Price to earnings Ratio - | 1Y Target Price 37.22 | ||
Volume (30-day avg) 13 | Beta 0.1 | 52 Weeks Range 4.45 - 19.18 | Updated Date 08/29/2025 |
52 Weeks Range 4.45 - 19.18 | Updated Date 08/29/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) -3.9 |
Analyzing Revenue: Products, Geography and Growth
Revenue by Products
Product revenue - Year on Year
Earnings Date
Report Date - | When - | Estimate - | Actual - |
Profitability
Profit Margin - | Operating Margin (TTM) -790.78% |
Management Effectiveness
Return on Assets (TTM) -15.39% | Return on Equity (TTM) -427.47% |
Valuation
Trailing PE - | Forward PE 27.93 | Enterprise Value 581859281 | Price to Sales(TTM) 62.49 |
Enterprise Value 581859281 | Price to Sales(TTM) 62.49 | ||
Enterprise Value to Revenue 40.58 | Enterprise Value to EBITDA 0.99 | Shares Outstanding 54866900 | Shares Floating 38737699 |
Shares Outstanding 54866900 | Shares Floating 38737699 | ||
Percent Insiders 6.08 | Percent Institutions 96.91 |
Upturn AI SWOT
Uniqure NV

Company Overview
History and Background
uniQure N.V. was founded in 1998 and is headquartered in Lexington, Massachusetts. It is a pioneer in gene therapy, focusing on developing and delivering transformative therapies for patients with severe genetic diseases. The company initially focused on developing gene therapies for liver diseases, but has since expanded its pipeline to include treatments for neurological disorders.
Core Business Areas
- Liver Diseases: Focuses on developing gene therapies for liver diseases, including hemophilia B.
- Neurological Disorders: Focuses on gene therapies for neurological disorders like Huntington's disease and epilepsy.
- Manufacturing: Develops and scales up manufacturing processes for gene therapy products.
Leadership and Structure
uniQure N.V. has a leadership team consisting of key executives like the CEO, CFO, and Chief Scientific Officer. The company's organizational structure is based on functional departments, including research and development, manufacturing, clinical development, and commercial operations.
Top Products and Market Share
Key Offerings
- Hemgenix (etranacogene dezaparvovec): Hemgenix is a gene therapy for adults with hemophilia B. As a newly approved product in the US, its market share is still developing, but is expected to capture a significant portion of the hemophilia B market. Competitors include traditional factor IX replacement therapies from companies like Novo Nordisk (NVO) and CSL Behring (CSLLY), and other emerging gene therapies.
- AMT-130: AMT-130 is an investigational gene therapy for Huntington's disease. While not yet approved, it represents a significant potential product. The market for Huntington's disease treatments is currently limited, making uniQure a potential leader in the treatment landscape. Competitors developing Huntington's disease treatments include Wave Life Sciences (WVE) and Roche (RHHBY).
Market Dynamics
Industry Overview
The gene therapy industry is rapidly evolving, with increasing regulatory approvals and growing interest from pharmaceutical companies. The market is driven by advancements in gene delivery technologies and the potential for one-time curative treatments for genetic diseases.
Positioning
uniQure N.V. is positioned as a pioneer in the gene therapy space, with a focus on developing and commercializing innovative therapies. Its competitive advantages include its expertise in AAV-based gene therapy and its established manufacturing capabilities.
Total Addressable Market (TAM)
The total addressable market for gene therapies is estimated to reach tens of billions of dollars in the coming years. UniQure is well-positioned to capture a significant share of this market with its pipeline of gene therapy candidates.
Upturn SWOT Analysis
Strengths
- Pioneering gene therapy company
- AAV-based gene therapy expertise
- Hemgenix (Hemophilia B) already approved
- Established manufacturing capabilities
- Strong intellectual property portfolio
Weaknesses
- High development costs
- Regulatory hurdles
- Manufacturing complexity
- Potential for off-target effects
- Commercialization challenges
Opportunities
- Expanding gene therapy pipeline
- Partnering with pharmaceutical companies
- Acquiring complementary technologies
- Entering new geographic markets
- Expanding into new disease areas
Threats
- Competition from other gene therapy companies
- Regulatory setbacks
- Patent disputes
- Pricing pressures
- Adverse events in clinical trials
Competitors and Market Share
Key Competitors
- Novo Nordisk (NVO)
- CSL Behring (CSLLY)
- Sangamo Therapeutics (SGMO)
- Biogen (BIIB)
- Voyager Therapeutics (VYGR)
Competitive Landscape
uniQure faces competition from established pharmaceutical companies and emerging gene therapy companies. Its advantages include its expertise in AAV-based gene therapy and its approved product, Hemgenix. Its disadvantages include its relatively small size and limited financial resources compared to larger competitors.
Major Acquisitions
InoCell B.V.
- Year: 2020
- Acquisition Price (USD millions): 36
- Strategic Rationale: Acquired InoCell B.V. to enhance its manufacturing capabilities for AAV-based gene therapies.
Growth Trajectory and Initiatives
Historical Growth: uniQure N.V.'s historical growth has been driven by its advancements in gene therapy and clinical trial progress.
Future Projections: Future growth projections are based on analyst estimates for Hemgenix sales, the success of AMT-130, and other pipeline candidates. Analyst estimates should be consulted directly.
Recent Initiatives: Recent initiatives include the commercial launch of Hemgenix, continued clinical development of AMT-130, and expansion of manufacturing capabilities.
Summary
UniQure N.V. is a pioneering gene therapy company with a growing pipeline and an approved product, Hemgenix. Their strength lies in AAV technology and experience, although high development costs and regulatory hurdles remain challenges. The company must continue to innovate, execute clinical trials effectively, and successfully commercialize products to maintain its position in a competitive landscape. Future growth will depend on expansion of the pipeline and successful sales of Hemgenix.
Peer Comparison
Sources and Disclaimers
Data Sources:
- uniQure N.V. Investor Relations
- SEC Filings
- Analyst Reports
- Company Press Releases
Disclaimers:
This analysis is based on publicly available information and analyst estimates, which are subject to change. Investment decisions should be made after consulting with a financial professional.
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 Uniqure NV
Exchange NASDAQ | Headquaters - | ||
IPO Launch date 2007-06-20 | CEO & Executive Director Mr. Matthew Craig Kapusta CPA | ||
Sector Healthcare | Industry Biotechnology | Full time employees 209 | Website https://www.uniqure.com |
Full time employees 209 | Website https://www.uniqure.com |
uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases in the United States. The company offers HEMGENIX that allows people living with hemophilia B to produce factor IX, which can lower the risk of bleeding. Its lead product candidate is AMT-130, a gene therapy candidate, which is in Phase I/II clinical study for the treatment of Huntington's disease. The company also develops AMT-260, which is in Phase I/IIa clinical trial for the treatment of mesial temporal lobe epilepsy; AMT-162, which is in Phase I/IIa clinical trial to treat superoxide dismutase enzyme-amyotrophic lateral sclerosis; and AMT-191, an investigational gene therapy candidate which is in phase I/IIa clinical trial for the treatment of fabry disease. It has a licensing agreement with Apic Bio to develop, manufacture, and commercialize intrathecally administered investigational gene therapy for ALS caused by mutations in SOD-1; and development and commercial supply agreement with CLS Bhering. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

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