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Neurogene Inc (NGNE)



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Upturn Advisory Summary
09/17/2025: NGNE (1-star) is currently NOT-A-BUY. Pass it for now.
1 Year Target Price $47.4
1 Year Target Price $47.4
4 | Strong Buy |
2 | Buy |
1 | Hold |
0 | Sell |
0 | Strong Sell |
Analysis of Past Performance
Type Stock | Historic Profit -39.07% | Avg. Invested days 26 | Today’s Advisory PASS |
Upturn Star Rating ![]() ![]() | Upturn Advisory Performance ![]() | Stock Returns Performance ![]() |
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Key Highlights
Company Size Small-Cap Stock | Market Capitalization 248.90M USD | Price to earnings Ratio - | 1Y Target Price 47.4 |
Price to earnings Ratio - | 1Y Target Price 47.4 | ||
Volume (30-day avg) 7 | Beta - | 52 Weeks Range 6.88 - 74.49 | Updated Date 09/17/2025 |
52 Weeks Range 6.88 - 74.49 | Updated Date 09/17/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) -4.31 |
Analyzing Revenue: Products, Geography and Growth
Revenue by Products
Earnings Date
Report Date - | When - | Estimate - | Actual - |
Profitability
Profit Margin - | Operating Margin (TTM) - |
Management Effectiveness
Return on Assets (TTM) -25.07% | Return on Equity (TTM) -39.41% |
Valuation
Trailing PE - | Forward PE - | Enterprise Value -12763785 | Price to Sales(TTM) 313.77 |
Enterprise Value -12763785 | Price to Sales(TTM) 313.77 | ||
Enterprise Value to Revenue 480.94 | Enterprise Value to EBITDA -8.02 | Shares Outstanding 14271900 | Shares Floating 4799645 |
Shares Outstanding 14271900 | Shares Floating 4799645 | ||
Percent Insiders 9.27 | Percent Institutions 108.24 |
Upturn AI SWOT
Neurogene Inc
Company Overview
History and Background
Neurogene Inc. is a biotechnology company focused on developing genetic medicines for neurological diseases. Founded in 2018, it aims to address unmet needs in treating rare and devastating neurological disorders through innovative gene therapy approaches. Significant milestones include advancing multiple gene therapy programs into clinical trials and securing strategic partnerships.
Core Business Areas
- Gene Therapy Development: Focuses on developing and commercializing gene therapies for neurological diseases.
- Research and Discovery: Invests in research to identify novel therapeutic targets and gene delivery technologies.
- Manufacturing: Develops and maintains in-house GMP capabilities for manufacturing gene therapy products.
Leadership and Structure
The company is led by a team of experienced biotechnology executives and scientists. The organizational structure includes research and development, clinical operations, manufacturing, and commercial functions.
Top Products and Market Share
Key Offerings
- NG-101 (Aspa-AAV Gene Therapy for Canavan Disease): An AAV-based gene therapy designed to deliver a functional copy of the ASPA gene to patients with Canavan disease. This is currently in clinical trials. Competitors include companies developing supportive care treatments for Canavan disease, but no direct competitors with a gene therapy in the same stage of development. Market share is not yet applicable as there is no revenue.
- NG-401 (AAV Gene Therapy for Rett Syndrome): An AAV-based gene therapy designed to treat Rett Syndrome. This is in pre-clinical development. Competitors are companies in pre-clinical stages as well, no direct competitive gene therapy treatments have entered human clinical trials.
Market Dynamics
Industry Overview
The gene therapy industry is experiencing rapid growth driven by technological advancements and increasing regulatory approvals. The neurological disease segment represents a significant market opportunity due to the high unmet medical need and limited treatment options.
Positioning
Neurogene Inc. is positioned as a leader in developing gene therapies for neurological diseases, focusing on rare and severe disorders. Their competitive advantage lies in their proprietary gene delivery technologies and experienced management team.
Total Addressable Market (TAM)
The TAM for gene therapies targeting neurological diseases is estimated to be billions of dollars, given the number of potential target diseases and patient populations. Neurogene Inc. is focused on capturing a significant portion of this market through its targeted approach.
Upturn SWOT Analysis
Strengths
- Strong intellectual property portfolio
- Experienced management team
- Proprietary gene delivery technologies
- Advanced clinical-stage programs
- In-house manufacturing capabilities
Weaknesses
- High R&D expenses
- Clinical trial risks
- Reliance on regulatory approvals
- Limited commercial infrastructure
- Cash burn rate
Opportunities
- Expanding pipeline to new neurological diseases
- Securing strategic partnerships
- Acquiring complementary technologies
- Accelerated regulatory pathways
- Increasing market adoption of gene therapies
Threats
- Competition from other gene therapy companies
- Adverse clinical trial results
- Regulatory hurdles
- Pricing and reimbursement challenges
- Manufacturing complexities
Competitors and Market Share
Key Competitors
- REGENXBIO (RGNX)
- Voyager Therapeutics (VYGR)
- uniQure (QURE)
Competitive Landscape
Neurogene Inc. faces competition from established gene therapy companies with broader pipelines and greater financial resources. However, Neurogene's focus on specific neurological diseases and its proprietary technologies provide a competitive edge. Given the company's private status, it does not have public market share data; market share data provided is an approximate estimate of other competitors in the AAV gene therapy space.
Growth Trajectory and Initiatives
Historical Growth: Historical growth is difficult to evaluate, as the company is still relatively new.
Future Projections: Future growth projections depend on the success of its clinical trials and regulatory approvals. Analyst estimates are unavailable due to the company's private status.
Recent Initiatives: Recent initiatives include advancing its lead gene therapy programs into later-stage clinical trials and expanding its manufacturing capabilities.
Summary
Neurogene is a promising gene therapy company focused on neurological diseases, but it is still an early stage company. Its pipeline of gene therapy programs is promising, and its in-house manufacturing is an advantage, but these programs also need to succeed in clinical trials. The company has strong intellectual property but requires substantial investment. Neurogene faces competition from larger, more established companies and needs to manage regulatory and manufacturing challenges effectively to achieve long-term success.
Peer Comparison
Sources and Disclaimers
Data Sources:
- Neurogene Inc. Website
- Industry Reports
- Company Presentations
- Analyst Reports (if available)
Disclaimers:
This analysis is based on publicly available information and estimates. The information provided is for informational purposes only and does not constitute investment advice. Market share data is an estimate.
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 Neurogene Inc
Exchange NASDAQ | Headquaters New York, NY, United States | ||
IPO Launch date 2014-03-07 | Founder, Executive Chair & CEO Dr. Rachel L. McMinn Ph.D. | ||
Sector Healthcare | Industry Biotechnology | Full time employees 107 | Website https://www.neurogene.com |
Full time employees 107 | Website https://www.neurogene.com |
Neurogene Inc., a clinical stage biotechnology company, develops genetic medicines for rare neurological diseases. The company's product candidates include NGN-401 that is packaged in an adeno-associated virus 9, which is in Phase 1/2 clinical trial for the treatment of Rett syndrome; and NGN-101, a conventional gene therapy candidate that is in Phase 1/2 clinical trial to treat CLN5 Batten disease. It has a license agreements with The University of Edinburgh, Virovek, Inc., Sigma-Aldrich Co. LLC., and Leland Stanford Junior University. The company was founded in 2018 and is headquartered in New York, New York.

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