Ultragenyx Pharmaceutical Inc. (RARE) Bundle
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 FocusUltragenyx 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 |
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
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 |
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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