Ultragenyx (RARE)
Health Care · Rare disease biopharmaceuticals · NASDAQ
A biopharmaceutical company developing and commercializing novel therapies for rare and ultra-rare genetic diseases with high unmet medical need.
What Ultragenyx does
Ultragenyx is a biopharmaceutical company focused on discovering, developing, and commercializing novel therapies for serious rare and ultra-rare genetic diseases. The company's portfolio spans multiple modalities including biologics, small molecules, adeno-associated virus (AAV) gene therapy, and nucleic acid product candidates. Ultragenyx has approved products such as Crysvita for X-Linked Hypophosphatemia and Tumor-Induced Osteomalacia, along with a pipeline of clinical-stage candidates targeting genetic disorders with significant unmet medical need and clear biology. The company retains global commercialization rights to its products and operates its own commercial organization across developed markets including North America, the EU, UK, Latin America, and select international markets.
Themes: ["Rare genetic diseases","Ultra-rare disease therapeutics","Gene therapy (AAV)","Monoclonal antibodies","Unmet medical need","Orphan drugs"]
Fundamentals
- Price$34.61 as of 2026-07-09 close
- Market cap$3.4B as of 2026-07-10
- 1-year return-16.5% as of 2026-07-09 close
- Net margin-91.0% as of 2026-07-10
- Gross margin+83.6% as of 2026-07-10
- ROE-414.2% as of 2026-07-10
- Debt / equity90.54 as of 2026-07-10
- Revenue growth (YoY)+13.3% as of 2026-07-10
- Revenue CAGR (3y)+22.8% SEC XBRL
- Beta0.27 as of 2026-07-10
Key risks (from latest filing)
["Company is deeply unprofitable with negative 90.96% net margin and high R&D burn, dependent on future product commercialization success and market adoption.","Rare disease markets are inherently small with limited patient populations, creating revenue ceiling constraints and heavy reliance on pricing power and reimbursement decisions.","Gene therapy and AAV-based products face significant regulatory uncertainty, manufacturing complexity, and potential safety concerns that could delay or prevent approvals."]
Competitors & peers
- Biomarin Pharmaceutical (BMRN)
- Alexion Pharmaceuticals (now part of AstraZeneca)
- Spark Therapeutics (now part of Roche)
- Sesen Bio
- Regenxbio
- Sangamo Therapeutics (SGMO)
Fundamentals: Finnhub, as of 2026-07-10. Filings: SEC EDGAR. Prices are delayed daily-close data.
Last updated 2026-07-09.
Informational only — NOT financial advice. All figures are delayed daily-close data from SEC EDGAR & Finnhub, shown with their as-of date.