Ultragenyx is a commercial-stage biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.
The company's strategy focuses on multiple therapeutic modalities, including small molecules, biologics, and gene therapies. Ultragenyx is recognized for its aggressive development timelines and its ability to navigate complex regulatory pathways for orphan indications, maintaining a global presence with products marketed in over 35 countries.
CLASSIFICATION
SIZE & FINANCIALS
Employees:1001-5000
Revenue:$673M (2025)
Founded:2010
Ownership:public
Status:operating
FUNDING
Stage:Public
Total Raised:$135M (Pre-IPO)
Investors:TPG, Adage Capital Management, Fidelity Management & Research Company
STOCK
Exchange:NASDAQ
Ticker:RARE
Market Cap:$1.98B
PIPELINE
Stage:Commercial
Lead Drug Stage:Commercial
Modalities:Gene therapy, mAb, Small molecule, ASO, Enzyme replacement therapy
Active Trials:15
Trial Phases:Phase 1: 4 | Phase 2: 5 | Phase 3: 6
FDA Approvals:4
EMA Approvals:3
CORPORATE STRUCTURE
Subsidiaries:Dimension Therapeutics, GeneTx Biotherapeutics
Key Partnerships:Kyowa Kirin (Crysvita co-development), Regeneron (Evkeeza ex-US commercialization), Daiichi Sankyo (Gene therapy manufacturing technology)
COMPETITION
Position:Leader
Competitors:BioMarin Pharmaceutical, Sarepta Therapeutics, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, Amgen (Horizon Therapeutics)
LEADERSHIP
Key Executives:
Emil D. Kakkis, M.D., Ph.D. - CEO & President
Howard Horn - CFO
Eric Crombez, M.D. - Chief Medical Officer
Scientific Founders:Emil D. Kakkis
Board Members:Daniel G. Welch (Chair), Deborah Dunsire, Matthew K. Fust, Shehnaaz Suliman
LINKS
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