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Neurogene Inc (NGNE)



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Upturn Advisory Summary
08/28/2025: 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 268.88M 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 08/29/2025 |
52 Weeks Range 6.88 - 74.49 | Updated Date 08/29/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) -4.31 |
Analyzing Revenue: Products, Geography and Growth
Revenue by Products
Earnings Date
Report Date 2025-08-11 | When - | Estimate -1.11 | Actual -1.05 |
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 7216897 | Price to Sales(TTM) 313.77 |
Enterprise Value 7216897 | 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 clinical-stage biotechnology company focused on developing genetic medicines to treat neurological diseases. Founded in 2018, Neurogene aims to develop treatments for rare neurological disorders, focusing on programs that address the underlying genetic causes.
Core Business Areas
- Gene Therapy Development: Focuses on creating and advancing novel AAV-based gene therapies for various neurological disorders.
- Neurological Disease Research: Conducts research to identify and validate therapeutic targets in neurological diseases with high unmet need.
Leadership and Structure
The leadership team includes seasoned executives with experience in gene therapy and drug development. The company structure typically involves research, development, clinical, and regulatory functions.
Top Products and Market Share
Key Offerings
- NG-101 (for Batten disease): An AAV-based gene therapy in clinical development for CLN5 Batten disease. Market share is currently 0% as the product is not yet approved. Competitors: Passage Bio, BioMarin (for other types of Batten disease).
- NG-401 (for Rett syndrome): An AAV-based gene therapy in clinical development for Rett Syndrome. Market share is currently 0% as the product is not yet approved. Competitors: Various companies exploring gene therapy approaches for Rett syndrome.
Market Dynamics
Industry Overview
The gene therapy market is rapidly growing, driven by advancements in vector technology and increasing regulatory approvals. Focus areas include rare genetic diseases, neurological disorders, and cancer.
Positioning
Neurogene is positioned as a player in the rare neurological disease gene therapy space. Its competitive advantage lies in its focus on specific genetic mutations and AAV delivery technology.
Total Addressable Market (TAM)
The TAM for gene therapies targeting neurological disorders is estimated to be in the billions of dollars annually. Neurogene is positioned to capture a portion of this market through its pipeline of gene therapy candidates.
Upturn SWOT Analysis
Strengths
- Focus on rare neurological diseases with high unmet need
- Proprietary AAV-based gene therapy platform
- Experienced leadership team in gene therapy development
- Strong preclinical and early clinical data for lead programs
Weaknesses
- Reliance on successful clinical trial outcomes
- High R&D expenses and capital requirements
- Regulatory and manufacturing risks associated with gene therapy
- Limited commercial infrastructure
Opportunities
- Expansion of pipeline to include additional neurological targets
- Strategic partnerships with larger pharmaceutical companies
- Accelerated regulatory pathways for rare disease therapies
- Positive clinical trial results leading to market approval
Threats
- Competition from other gene therapy companies
- Adverse safety events in clinical trials
- Reimbursement challenges for expensive gene therapies
- Changes in regulatory landscape
Competitors and Market Share
Key Competitors
- BMRA
- VRTX
- BLUE
Competitive Landscape
Neurogene faces competition from larger and more established gene therapy companies. Its advantages include a focus on specific rare diseases and AAV technology. Disadvantages include limited resources compared to larger competitors.
Growth Trajectory and Initiatives
Historical Growth: Historical growth has been marked by progress in preclinical and clinical development programs and capital raises.
Future Projections: Future growth depends on successful clinical trial outcomes, regulatory approvals, and commercial launch of gene therapy products. Analyst estimates vary based on these factors.
Recent Initiatives: Recent initiatives include advancing clinical trials for NG-101 and NG-401 and expanding the pipeline through strategic partnerships.
Summary
Neurogene is a clinical-stage biotech company with a focus on rare neurological diseases. Its strength lies in its innovative AAV gene therapy platform and targeted approach. The company is currently pre-revenue and relies on financing to advance its programs. Success depends heavily on clinical trial outcomes and regulatory approvals.
Peer Comparison
Sources and Disclaimers
Data Sources:
- Company website
- SEC filings
- Analyst reports
Disclaimers:
This analysis is based on publicly available information and is not financial advice. The gene therapy sector is dynamic, and information may change rapidly.
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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