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Regenxbio Inc (RGNX)

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Upturn Advisory Summary
10/29/2025: RGNX (1-star) has a low Upturn Star Rating. Not recommended to BUY.
1 Year Target Price $29.27
1 Year Target Price $29.27
| 6 | Strong Buy |
| 5 | Buy |
| 1 | Hold |
| 0 | Sell |
| 0 | Strong Sell |
Analysis of Past Performance
Type Stock | Historic Profit -28.58% | Avg. Invested days 27 | Today’s Advisory Consider higher Upturn Star rating |
Upturn Star Rating ![]() | Upturn Advisory Performance | Stock Returns Performance |
Key Highlights
Company Size Small-Cap Stock | Market Capitalization 664.77M USD | Price to earnings Ratio - | 1Y Target Price 29.27 |
Price to earnings Ratio - | 1Y Target Price 29.27 | ||
Volume (30-day avg) 12 | Beta 1.17 | 52 Weeks Range 5.04 - 13.93 | Updated Date 10/29/2025 |
52 Weeks Range 5.04 - 13.93 | Updated Date 10/29/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) -3.44 |
Analyzing Revenue: Products, Geography and Growth
Revenue by Products
Product revenue - Year on Year
Earnings Date
Report Date 2025-11-05 | When - | Estimate -1.3 | Actual - |
Profitability
Profit Margin -112.7% | Operating Margin (TTM) -296.26% |
Management Effectiveness
Return on Assets (TTM) -17.89% | Return on Equity (TTM) -62.49% |
Valuation
Trailing PE - | Forward PE - | Enterprise Value 288838405 | Price to Sales(TTM) 4.27 |
Enterprise Value 288838405 | Price to Sales(TTM) 4.27 | ||
Enterprise Value to Revenue 1.85 | Enterprise Value to EBITDA -3.37 | Shares Outstanding 50514216 | Shares Floating 41716660 |
Shares Outstanding 50514216 | Shares Floating 41716660 | ||
Percent Insiders 7.66 | Percent Institutions 85.62 |
Upturn AI SWOT
Regenxbio Inc

Company Overview
History and Background
REGENXBIO Inc. was founded in 2009. It's a clinical-stage biotechnology company focused on developing, commercializing, and licensing adeno-associated virus (AAV) gene therapy. The company utilizes its proprietary NAV Technology Platform to create AAV vectors for gene delivery.
Core Business Areas
- Gene Therapy Development: REGENXBIO focuses on developing gene therapies for a range of genetic diseases, including neurodegenerative, metabolic, and retinal diseases.
- NAV Technology Platform Licensing: The company licenses its NAV Technology Platform to other companies for their gene therapy programs, generating revenue through upfront payments, milestone payments, and royalties.
Leadership and Structure
Kenneth T. Mills is the President and CEO. The company has a typical biotechnology organizational structure with departments focused on research, development, manufacturing, clinical trials, and commercialization.
Top Products and Market Share
Key Offerings
- RGX-314 (Wet AMD): RGX-314 is an AAV8 vector-based gene therapy being developed for the treatment of wet age-related macular degeneration (AMD). It aims to provide a one-time treatment to reduce the need for frequent anti-VEGF injections. Competitors include Regeneron (EYLEA), Roche (Vabysmo), and biosimilars. Market share data is not yet applicable as the product is still in clinical development.
- NAV Technology Platform Licensing Agreements: REGENXBIO's NAV Technology Platform is licensed to various pharmaceutical and biotechnology companies. Revenue from these licensing agreements varies year to year, depending on new deals, milestone payments, and royalties. Competitors are other companies with gene therapy vector platforms such as Voyager Therapeutics.
Market Dynamics
Industry Overview
The gene therapy industry is rapidly growing, driven by advancements in vector technology and increasing success in clinical trials. There is significant unmet medical need for genetic diseases, creating substantial market opportunity.
Positioning
REGENXBIO is positioned as a leading company in the AAV gene therapy space, particularly due to its NAV Technology Platform. Its competitive advantage lies in the breadth and versatility of its vector library and its ability to generate new AAV vectors.
Total Addressable Market (TAM)
The overall gene therapy market is expected to reach tens of billions of dollars in the coming years. REGENXBIO, with its diverse pipeline and licensing agreements, is positioned to capture a significant portion of this market.
Upturn SWOT Analysis
Strengths
- Proprietary NAV Technology Platform
- Strong intellectual property portfolio
- Experienced management team
- Diverse pipeline of gene therapy candidates
- Established licensing agreements
Weaknesses
- High research and development costs
- Regulatory hurdles and approval timelines
- Manufacturing complexities
- Dependence on clinical trial outcomes
- Reliance on collaborations for certain programs
Opportunities
- Expanding gene therapy applications to new disease areas
- Advancing clinical trials and securing regulatory approvals
- Strategic partnerships and collaborations
- Increasing adoption of gene therapy by healthcare providers
- Developing next-generation AAV vectors
Threats
- Competition from other gene therapy companies
- Adverse events or safety concerns in clinical trials
- Changes in regulatory landscape
- Patent challenges
- Pricing and reimbursement pressures
Competitors and Market Share
Key Competitors
- BMY
- SNY
- VRTX
- BLUE
Competitive Landscape
REGENXBIO's advantages include its NAV Technology Platform and diverse pipeline. Disadvantages may include financial constraints compared to larger pharmaceutical companies.
Growth Trajectory and Initiatives
Historical Growth: Historical growth trends are characterized by increasing R&D spending and potential revenue from licensing agreements.
Future Projections: Analyst projections generally anticipate revenue growth driven by advancements in its pipeline programs and potential commercialization of RGX-314.
Recent Initiatives: Recent initiatives include advancing RGX-314 into late-stage clinical trials and expanding its NAV Technology Platform licensing agreements.
Summary
Regenxbio is a gene therapy company with a strong technology platform. Its NAV Technology Platform gives them a competitive advantage, and they have several promising therapies. Clinical trial successes are critical. Competition and regulatory hurdles are also major challenges.
Similar Stocks
Sources and Disclaimers
Data Sources:
- Company website
- SEC filings
- Analyst reports
Disclaimers:
This analysis is based on publicly available information and should not be considered financial advice. Market share estimates are approximate.
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 Regenxbio Inc
Exchange NASDAQ | Headquaters Rockville, MD, United States | ||
IPO Launch date 2015-09-17 | President, CEO & Director Mr. Curran M. Simpson M.S. | ||
Sector Healthcare | Industry Biotechnology | Full time employees 353 | Website https://www.regenxbio.com |
Full time employees 353 | Website https://www.regenxbio.com | ||
REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase III clinical trial for the treatment of Duchenne muscular dystrophy, as well as RGX-121 for the treatment of mucopolysaccharidosis type II. It also develops RGX-111 for the treatment of mucopolysaccharidosis type I, RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2, and RGX-381 for the treatment of the ocular manifestations of CLN2 disease. In addition, the company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. Further, it has a collaboration and license agreement with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States. REGENXBIO Inc. was incorporated in 2008 and is headquartered in Rockville, Maryland.

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