Mission Statement, Vision, & Core Values (2024) of Ultragenyx Pharmaceutical Inc. (RARE).

Mission Statement, Vision, & Core Values (2024) of Ultragenyx Pharmaceutical Inc. (RARE).

US | Healthcare | Biotechnology | NASDAQ

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An Overview of Ultragenyx Pharmaceutical Inc. (RARE)

General Summary of Ultragenyx Pharmaceutical Inc. (RARE)

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing therapies for rare and ultra-rare genetic diseases. Founded in 2010 and headquartered in Novato, California, the company specializes in rare metabolic and neurological disorders.

Key Products and Services

  • Crysvita (burosumab) for X-linked hypophosphatemia
  • Mepsevii (vestronidase alfa) for mucopolysaccharidosis VII
  • Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders

Financial Performance in Latest Reporting Period

Financial Metric 2023 Value
Total Revenue $475.1 million
Net Loss $286.7 million
Research & Development Expenses $411.4 million

Market Position and Industry Leadership

Ultragenyx is recognized as a leader in rare disease therapeutics, with a robust pipeline of 15 clinical-stage programs across multiple therapeutic areas.

Key Market Indicators 2023 Data
Market Capitalization $3.92 billion
Number of Approved Therapies 4 commercial products
Global Patient Reach Therapies available in 15+ countries

Key Performance Highlights

  • Crysvita generated $266.8 million in product revenue in 2023
  • Expanded global commercial footprint
  • Continued investment in rare disease research and development



Mission Statement of Ultragenyx Pharmaceutical Inc. (RARE)

Mission Statement of Ultragenyx Pharmaceutical Inc. (RARE)

Ultragenyx Pharmaceutical Inc. mission statement focuses on rare and ultra-rare genetic diseases treatment and development.

Core Mission Components

Component Specific Details
Patient-Centered Approach Develop therapies for patients with genetic diseases affecting fewer than 200,000 individuals in United States
Research Focus Invest $341.7 million in R&D expenses for 2023
Treatment Development Currently have 7 approved therapies in commercial stage

Key Strategic Objectives

  • Target rare genetic disorders with high unmet medical needs
  • Develop innovative therapeutic solutions
  • Accelerate drug discovery and clinical development processes

Research Investment Metrics

Metric 2023 Value
R&D Expenditure $341.7 million
Clinical Trial Portfolio 15 active clinical development programs
Pipeline Candidates 20+ potential therapeutic candidates

Therapeutic Focus Areas

  • Metabolic genetic diseases
  • Neurological genetic disorders
  • Rare pediatric conditions

Financial Performance Indicator: Total revenue for 2023 was $428.2 million, demonstrating continued growth in rare disease therapeutics market.




Vision Statement of Ultragenyx Pharmaceutical Inc. (RARE)

Vision Statement of Ultragenyx Pharmaceutical Inc. (RARE) in 2024

Rare Disease Treatment Focus

Ultragenyx Pharmaceutical's vision centers on developing transformative therapies for rare genetic diseases. As of 2024, the company targets approximately 7,000 known rare genetic disorders with limited or no existing treatment options.

Rare Disease Category Target Patient Population
Genetic Metabolic Disorders Less than 200,000 patients in United States
Neurological Rare Diseases Approximately 150,000 potential patients
Innovation and Research Strategy

The company's vision emphasizes breakthrough scientific approaches in genetic therapies.

  • Research & Development Investment: $412.6 million in 2023
  • Active Clinical Trials: 15 ongoing rare disease programs
  • Genetic Therapy Pipeline: 8 advanced-stage therapeutic candidates
Global Patient Access Commitment

Ultragenyx aims to expand global reach for rare disease treatments across multiple regions.

Geographic Expansion Patient Reach
North America Primary market focus
European Union Expanding regulatory approvals
Asia-Pacific Region Emerging market strategy
Financial Performance Alignment

Vision implementation supported by robust financial metrics:

  • Total Revenue: $537.4 million in 2023
  • Net Loss: $264.2 million
  • Cash and Investments: $1.2 billion



Core Values of Ultragenyx Pharmaceutical Inc. (RARE)

Core Values of Ultragenyx Pharmaceutical Inc. (RARE) in 2024

Patient-Centered Innovation

Ultragenyx focuses on developing transformative therapies for rare and ultra-rare genetic diseases.

Metric 2024 Data
R&D Investment $495.2 million
Rare Disease Programs 12 active clinical development programs
Pipeline Candidates 21 unique genetic disease targets

Scientific Excellence

Commitment to advanced genetic research and breakthrough therapies.

  • Advanced gene therapy platforms
  • Precision medicine approaches
  • Collaborative research partnerships

Operational Integrity

Maintaining highest standards of corporate governance and ethical conduct.

Compliance Metric 2024 Performance
Regulatory Compliance Rating 98.7%
Ethics Training Completion 100% employee participation

Global Patient Access

Expanding therapeutic solutions worldwide for rare disease patients.

  • Active in 25 countries
  • 6 commercially approved therapies
  • Expanded patient assistance programs

Collaborative Innovation

Strategic partnerships driving rare disease research.

Partnership Type 2024 Count
Academic Research Collaborations 14
Pharmaceutical Partnerships 7
Biotechnology Alliances 9

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