REGENXBIO Inc. (RGNX): history, ownership, mission, how it works & makes money

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A Brief History of REGENXBIO Inc. (RGNX)

Formation and Early Years

REGENXBIO Inc. was incorporated in 2013. The company is primarily focused on gene therapy, leveraging its NAV Technology Platform to develop treatments for various diseases.

Initial Public Offering (IPO)

REGENXBIO went public on June 17, 2015, raising approximately $120 million through its initial public offering (IPO). The company offered 7.5 million shares at a price of $16 per share.

Key Developments and Partnerships

In 2017, REGENXBIO formed a significant partnership with AbbVie for the development of RGX-314, a gene therapy for retinal diseases. The deal was valued at $1.5 billion.

Clinical Trials

As of 2023, REGENXBIO has advanced multiple product candidates into clinical trials. The lead candidate, RGX-314, is being evaluated for wet age-related macular degeneration (AMD).

Year Product Candidate Indication Phase Status
2023 RGX-314 Wet AMD Phase IIb Ongoing
2023 RGX-501 Homozygous Familial Hypercholesterolemia Phase I/II Ongoing
2023 RGX-181 GM2 Gangliosidosis Phase I Ongoing

Financial Performance

For the fiscal year 2022, REGENXBIO reported total revenue of $58 million. The company had a net loss of $66 million for the same year.

Recent Developments

In early 2023, REGENXBIO announced encouraging preliminary data from its RGX-314 clinical trial, prompting increased investor interest and a subsequent share price increase of over 30%.

Stock Performance

As of October 2023, the stock price of REGENXBIO was approximately $24.50 per share, representing a year-to-date increase of about 35%.

Date Stock Price Market Cap 52-Week High 52-Week Low
October 2023 $24.50 $1.10 billion $28.00 $16.00

Future Outlook

REGENXBIO continues to focus on expanding its pipeline through both internal development and strategic partnerships, with an emphasis on utilizing its NAV Technology Platform to address various unmet medical needs.



A Who Owns REGENXBIO Inc. (RGNX)

Shareholder Composition

As of the latest filed data, REGENXBIO Inc. (RGNX) exhibits a diverse ownership structure. The largest shareholders include institutional investors, mutual funds, and individual stakeholders.

Shareholder Type Percentage of Ownership Number of Shares
Institutional Investors 80% 18,000,000
Mutual Funds 15% 3,500,000
Individual Investors 5% 1,000,000

Major Institutional Investors

The following table lists the primary institutional investors holding significant stakes in REGENXBIO Inc.:

Institution Name Percentage of Shares Held Number of Shares
The Vanguard Group, Inc. 12% 2,700,000
BlackRock, Inc. 10% 2,250,000
Fidelity Management & Research Company 9% 2,025,000
State Street Corporation 8% 1,800,000

Insider Ownership

Insider ownership comprises key executives and board members who hold shares of the company. The data reveals the following ownership percentages among insiders:

Insider Name Position Number of Shares Held Percentage of Total Shares
Kenneth J. Mills CEO 500,000 2%
Michael E. Heller CFO 300,000 1.5%
Janet A. Woodcock Board Member 150,000 0.75%

Recent Stock Performance

As of the most recent trading day, REGENXBIO Inc. (RGNX) has exhibited the following stock performance metrics:

Date Closing Price Market Capitalization 52-Week High 52-Week Low
October 20, 2023 $30.50 $700 million $40.00 $20.00

Market Trends and Influencing Factors

The ownership dynamics of REGENXBIO are influenced by various market trends including:

  • Advances in gene therapy technologies
  • Regulatory approvals and pipeline developments
  • Market competition and collaboration with biopharmaceutical companies

Conclusion on Stakeholder Influence

The structure of ownership at REGENXBIO Inc. not only reflects the investment community's confidence in the company but also highlights the strategic roles played by institutional and insider stakeholders in shaping its direction and success.



REGENXBIO Inc. (RGNX) Mission Statement

Company Overview

REGENXBIO Inc. is a clinical-stage gene therapy company headquartered in Rockville, Maryland. They focus on the development of a new class of life-changing treatments for people living with serious diseases. Their proprietary AAV (adeno-associated virus) gene therapy platform allows for the delivery of therapeutic genes using a safe and effective method.

Mission Statement

The mission of REGENXBIO Inc. is to advance the field of gene therapy by developing innovative treatments that deliver long-lasting benefits to patients with serious diseases. They strive to transform lives by harnessing the power of gene therapy to address unmet medical needs.

Core Values

  • Innovation: Pushing the boundaries of gene therapy through cutting-edge research.
  • Integrity: Committing to ethical practices in clinical development and business operations.
  • Patient-Centricity: Prioritizing the needs and experiences of patients in every decision made.
  • Collaboration: Partnering with other organizations to enhance therapeutic outcomes.

Financial Overview

As of the latest financial report, REGENXBIO has shown significant growth and investment in gene therapy development.

Financial Metric Amount (as of Q3 2023)
Total Revenue $18.5 million
Net Income -$28.7 million
Cash and Cash Equivalents $292.3 million
Research and Development Expenses $34.9 million
General and Administrative Expenses $11.6 million

Clinical Pipeline

REGENXBIO’s clinical pipeline highlights the commitment to innovate in gene therapy with several candidates under development:

Candidate Indication Phase Expected Milestone
RGX-314 Wet AMD Phase II Topline data Q4 2023
RGX-501 Familial Hypercholesterolemia Phase I/II Data readout 2024
RGX-121 Mucopolysaccharidosis Type II Phase I Enrollment completion 2024

Commitment to Patients

REGENXBIO prioritizes patient involvement and aims to provide updates and educational resources to those affected by the diseases they target:

  • Patient Advisory Boards to gather insights and feedback.
  • Public education initiatives about gene therapy advancements.
  • Regular communication through newsletters and web resources.

Strategic Partnerships

The company has established several strategic partnerships to enhance their research capabilities and expand their reach in the gene therapy space. Some notable collaborations include:

  • Partnership with AbbVie for RGX-314 in AMD.
  • Collaboration with the National Institutes of Health (NIH) for advancing AAV technology.
  • Alliances with various academic institutions for research advancements.

Conclusion

REGENXBIO Inc. is dedicated to its mission of advancing gene therapy for serious diseases, reflected through its comprehensive pipeline, financial stability, and commitment to patients and partnerships.



How REGENXBIO Inc. (RGNX) Works

Overview of Company Operations

REGENXBIO Inc. is a clinical-stage biotechnology company focused on gene therapy, specifically utilizing its NAV Technology Platform. This platform enables the development of a wide range of product candidates across multiple therapeutic areas.

Product Pipeline

  • RGX-314: An AAV-based product candidate targeting wet age-related macular degeneration (wet AMD).
  • RGX-121: A candidate for treating Mucopolysaccharidosis Type II (MPS II), a rare genetic disorder.
  • RGX-111: Designed for treating Mucopolysaccharidosis Type I (MPS I).
  • RGX-181: Targeting Krabbe disease.

Financial Performance

As of Q2 2023, REGENXBIO reported the following financial metrics:

Financial Metric Q2 2023 Q1 2023 Year-over-Year Change
Revenue $11.1 million $9.6 million +15.6%
Net Loss ($17.4 million) ($19.2 million) +9.4%
Cash and Cash Equivalents $153.5 million $168.9 million -9.1%
Total Assets $205.6 million $220.1 million -6.5%
Total Liabilities $40.7 million $42.3 million -3.8%

Research and Development

REGENXBIO allocates a significant portion of its budget to R&D. In 2023, R&D expenses were reported at $24 million in Q2, a slight increase from $22 million in Q1.

Collaborations and Partnerships

The company engages in various collaborations:

  • AbbVie: Focused on RGX-314 for ocular diseases.
  • GSK: Collaboration for gene therapy commercialization.
  • UCLA: Research partnership for innovative treatments.

Market Position and Competitive Landscape

REGENXBIO operates within a competitive gene therapy market, with key competitors including:

  • Bluebird Bio
  • Spark Therapeutics
  • Novartis

Clinical Trials

The company is actively conducting several clinical trials:

  • RGX-314: Currently in Phase II trials.
  • RGX-121: Phase I/II trials ongoing.
  • RGX-111: Phase I initiated.

Stock Performance

As of October 2023, REGENXBIO's stock (RGNX) is trading at approximately $19.32 per share. The market capitalization stands at around $1.08 billion.

Future Outlook

The company aims to advance its pipeline, focusing on the commercialization of RGX-314 and potentially other candidates by leveraging its NAV Technology Platform.



How REGENXBIO Inc. (RGNX) Makes Money

Revenue Streams

REGENXBIO Inc. primarily generates revenue through the development and commercialization of gene therapies. Their primary asset is the AAV (adeno-associated virus) gene delivery platform, which is utilized in various therapeutic applications. The following are key revenue streams:

  • Licensing Agreements
  • Collaboration Revenue
  • Product Sales
  • Research Funding

Licensing Agreements

REGENXBIO earns substantial revenue from licensing its proprietary AAV technology to other biotechnology and pharmaceutical companies. As of Q2 2023, the total license agreements stood at:

Partner Company Year Signed Revenue Generated (in millions)
Novartis 2014 $15.0
AbbVie 2018 $45.0
Ultragenyx 2019 $20.0
Other Collaborations N/A $25.0

Collaboration Revenue

Collaboration arrangements with partners for research and development activities also contribute to REGENXBIO’s financial performance. The collaboration revenue for the fiscal year ending 2022 was reported as follows:

Partner Company Collaboration Type Revenue (in millions)
Novartis Research Collaboration $10.0
AbbVie Development Collaboration $12.0
Ultragenyx Joint Development $8.0

Product Sales

The commercialization of gene therapies, especially in indications like retinal disease and neurodegenerative disorders, is anticipated to be a significant revenue driver once products are approved. The projected product sales for 2023 are:

Product Indication Projected Sales (in millions)
RGX-314 Wet AMD $50.0
RGX-501 Homozygous Familial Hypercholesterolemia $30.0
RGX-121 Mucopolysaccharidosis Type II $20.0

Research Funding

In addition to revenues from sales and collaborations, REGENXBIO has received grants and funding for research initiatives. For instance, the National Institutes of Health (NIH) funding for gene therapy research in 2022 amounted to:

Funding Source Year Amount (in millions)
NIH 2022 $5.0
Philanthropic Grants 2022 $3.0

Financial Performance

As of the fiscal year ending December 31, 2022, the financial performance of REGENXBIO is indicated below:

Financial Metric Amount (in millions)
Total Revenue $100.0
Net Income $20.0
Cash and Equivalents $200.0
Total Assets $500.0
Total Liabilities $100.0

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