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Neurogene Inc (NGNE)



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Upturn Advisory Summary
03/11/2025: NGNE (1-star) is currently NOT-A-BUY. Pass it for now.
Analysis of Past Performance
Type Stock | Historic Profit -35.56% | Avg. Invested days 25 | 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 144.15M USD | Price to earnings Ratio - | 1Y Target Price 50.2 |
Price to earnings Ratio - | 1Y Target Price 50.2 | ||
Volume (30-day avg) 236544 | Beta - | 52 Weeks Range 9.22 - 74.49 | Updated Date 04/2/2025 |
52 Weeks Range 9.22 - 74.49 | Updated Date 04/2/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) -4.28 |
Analyzing Revenue: Products, Geography and Growth
Revenue by Products
Earnings Date
Report Date 2025-03-17 | When Before Market | Estimate -1.1283 | Actual -0.99 |
Profitability
Profit Margin - | Operating Margin (TTM) -8930.27% |
Management Effectiveness
Return on Assets (TTM) -18.5% | Return on Equity (TTM) -30.28% |
Valuation
Trailing PE - | Forward PE - | Enterprise Value -154023041 | Price to Sales(TTM) 155.83 |
Enterprise Value -154023041 | Price to Sales(TTM) 155.83 | ||
Enterprise Value to Revenue 480.94 | Enterprise Value to EBITDA -8.02 | Shares Outstanding 14929600 | Shares Floating 5545438 |
Shares Outstanding 14929600 | Shares Floating 5545438 | ||
Percent Insiders 10.16 | Percent Institutions 108.36 |
Analyst Ratings
Rating 4.71 | Target Price 54.6 | Buy 2 | Strong Buy 5 |
Buy 2 | Strong Buy 5 | ||
Hold - | Sell - | Strong Sell - | |
Strong Sell - |
Upturn AI SWOT
Neurogene Inc
Company Overview
History and Background
Neurogene Inc. is a clinical-stage biotechnology company focused on developing genetic medicines for neurological diseases. Founded in 2018, it aims to address unmet medical needs with gene therapy approaches.
Core Business Areas
- Gene Therapy Development: Develops and advances novel AAV-based gene therapies for neurological disorders.
- NG-GTx Platform: Utilizes a proprietary gene therapy platform (NG-GTx) to deliver therapeutic genes directly to the central nervous system.
- Clinical Trials: Conducts clinical trials to evaluate the safety and efficacy of its gene therapy candidates.
Leadership and Structure
The leadership team comprises experienced biotech executives and scientists. The organizational structure is typical for a clinical-stage biotech company, with departments focused on research, development, clinical operations, and business development.
Top Products and Market Share
Key Offerings
- NG-101 (for Rett Syndrome): An AAV9 gene therapy designed to deliver a functional MECP2 gene to treat Rett Syndrome. Currently in clinical trials. Competitors include companies developing small molecule therapies for Rett Syndrome, but direct gene therapy competition is limited at this stage. Specific market share is not yet available due to being in clinical trials.
- NG-401 (for Asparagine Synthetase Deficiency - ASD): An AAV9 gene therapy designed to deliver a functional ASNS gene to treat ASD. Currently in clinical trials. Competitors are limited, as there are no approved therapies for ASD. Specific market share is not yet available due to being in clinical trials.
- NG-201 (for Batten disease): An AAV9 gene therapy designed to deliver a functional CLN5 gene to treat Batten disease. Currently in clinical trials. Competitors include companies developing supportive care for Batten disease, but direct gene therapy competition is limited at this stage. Specific market share is not yet available due to being in clinical trials.
Market Dynamics
Industry Overview
The gene therapy market is rapidly growing, driven by technological advancements and increasing investment in genetic medicines. The neurological disease market represents a significant opportunity due to high unmet needs.
Positioning
Neurogene Inc is positioned as a leader in developing gene therapies for severe neurological disorders with limited or no treatment options. It focuses on rare genetic diseases.
Total Addressable Market (TAM)
The TAM for gene therapies targeting neurological disorders is estimated to be in the billions of dollars. Neurogene Inc. is positioned to capture a portion of this market by addressing specific rare disease indications. The TAM for each target disease varies. Rett Syndrome's TAM is estimated at $500 million annually. The TAM for rare disorders such as ASD is highly variable due to lack of prevalence data.
Upturn SWOT Analysis
Strengths
- Proprietary gene therapy platform (NG-GTx)
- Strong management team with gene therapy expertise
- Focus on high unmet medical needs
- Advanced clinical-stage pipeline
Weaknesses
- High development costs associated with gene therapy
- Regulatory uncertainties surrounding gene therapy approval
- Potential for off-target effects or immune responses
- Relatively small company size
Opportunities
- Expanding pipeline to address additional neurological disorders
- Potential for partnerships with larger pharmaceutical companies
- Positive clinical trial results leading to regulatory approval
- Acquisition by a larger pharmaceutical company
Threats
- Competition from other gene therapy companies
- Setbacks in clinical trials
- Changes in regulatory landscape
- Challenges in manufacturing and scaling up gene therapy production
Competitors and Market Share
Key Competitors
- SPHR
- VRTX
- BLUE
Competitive Landscape
Neurogene's competitive advantage lies in its specialized focus on gene therapies for neurological disorders and its proprietary NG-GTx platform. Disadvantages include limited resources compared to larger pharmaceutical companies.
Major Acquisitions
Growth Trajectory and Initiatives
Historical Growth: Historical growth is defined by the expansion of its pipeline and advancement of clinical programs. Significant milestones include IND clearances and initiation of clinical trials.
Future Projections: Future growth is dependent on successful clinical trial outcomes and regulatory approvals. Analyst estimates depend on the probability of success for each program.
Recent Initiatives: Recent strategic initiatives include advancing clinical trials for NG-101 and NG-401 and securing funding through private placements.
Summary
Neurogene Inc. is a clinical-stage company with a focused approach on gene therapies for rare neurological disorders, demonstrating potential but facing typical biotech challenges such as regulatory hurdles and high R&D costs. Its proprietary platform and advanced pipeline are working well, but setbacks in clinical trials and competitor advancements pose risks. Securing partnerships and positive clinical outcomes are essential for its continued growth and success. The company is highly dependent on future funding rounds.
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Sources and Disclaimers
Data Sources:
- Neurogene Inc. website
- SEC filings
- Analyst reports
- Company Press Releases
- ClinicalTrials.gov
Disclaimers:
The data and analysis provided are for informational purposes only and should not be considered financial advice. Investment decisions should be based on your own research and due diligence.
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 which is packaged in an adeno-associated virus 9 that 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 agreement with The University of Edinburgh, Virovek, Inc., Sigma-Aldrich Co. LLC., and Leland Stanford Junior University. The company is headquartered in New York, New York.
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