Ultragenyx Pharmaceutical (RARE)
Develops and commercializes therapies for serious rare and ultra-rare genetic diseases.
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Develops and commercializes approved therapies and product candidates for serious rare and ultra-rare genetic diseases. Its operating model combines patient and caregiver engagement with collaborations, acquisitions, and commercial execution.
Identifies, acquires, develops, and commercializes therapies for serious rare and ultra-rare genetic diseases with clear biology and high unmet need. The portfolio focuses on diseases where the biology is clear and unmet need is high. Its approved portfolio includes Mepsevii, Crysvita, and Dojolvi, alongside a broader product-candidate portfolio and patient and caregiver engagement that informs clinical protocol, endpoint, analysis, commercialization, and access approaches.
Founder Emil D. Kakkis has remained President and CEO since the 2010 formation, providing continuity through the public-market and commercial stages. Strategic collaborations and targeted acquisitions add capabilities, while product approvals and collaboration arrangements shape operations.
Company facts
Target rangeAs of Sep 21, 2026
Latest analyst notes
| Date | Firm | Action | Prior | Current |
|---|---|---|---|---|
| Sep 21, 2026 | Evercore ISI | Target | Target: $16 | Target: $18 |
| Sep 21, 2026 | Barclays | Target | Target: $32 | Target: $35 |
| Sep 18, 2026 | Morgan Stanley | Target | Target: $18 | Target: $20 |
| Sep 18, 2026 | Cantor Fitzgerald | Reiterate | Rating: —Target: $33 | Target: $39 |
| Sep 18, 2026 | Canaccord Genuity | Target | Target: $37 | Target: $39 |
EPSReportedEstimate
RevenueReportedEstimate
Balance sheet, Jun 30, 2026Cash & short-term investments $292.0M · Total debt $1.20B · Total assets $1.26B · Shareholders' equity −$291.0M
- Revenue
- $214.0M
- Operating income
- −$75.0M
- Net income
- −$92.0M
No dividends on record.
| Institutions | 109.00% | 107.46M shares | |
|---|---|---|---|
| 329 holders · as of Jun 30, 2026 | |||
| Insiders | 4.22% | 4.16M shares | |
| 16 holders · as of Sep 15, 2026 | |||
| Adds to | 113.22% | ||
98.59M company shares as of Sep 22, 2026 · institutions as of Jun 30, 2026 · holders as of their latest filing
These groups come from separate filings that can overlap, so they add to 113.22%; nothing is left for Other.
Top shareholders13F · 13D/G · Form 4
| Holder | Stake | Shares | Date |
|---|---|---|---|
BlackRock, Inc.Institution | 10.81% | 10.65M | as of Jun 30, 2026 |
RTW Investments, LPInstitution | 7.91% | 7.80M | as of Jun 30, 2026 |
Vanguard Portfolio Management LLCInstitution | 6.89% | 6.79M | as of Jun 30, 2026 |
AQR Capital Management LLCInstitution | 6.65% | 6.56M | as of Jun 30, 2026 |
State Street CorpInstitution | 6.25% | 6.16M | as of Jun 30, 2026 |
Insider tradesForm 4
| Date | Insider | Role | Shares | Avg price |
|---|---|---|---|---|
| Sep 15, 2026 | Theodore Alan Huizenga | officer: SVP, Chief Accounting Officer | −1,283 | $13.57 |
| Sep 3, 2026 | Theodore Alan Huizenga | officer: SVP, Chief Accounting Officer | −161 | $13.99 |
| Aug 3, 2026 | Howard Horn | officer: Chief Financial Officer | −4,696 | $24.92 |
| Aug 3, 2026 | Karah Herdman Parschauer | officer: EVP and Chief Legal Officer | −1,900 | $24.92 |
| Jul 1, 2026 | Howard Horn | officer: Chief Financial Officer | −4,698 | $33.13 |
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Compare
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| Ultragenyx Pharmaceutical | $15.76 | +7.58% | $1.44B | — | — | 2.0 | +28.5% |
| BioMarin | $63.87 | −0.30% | $12.38B | 164.3 | 10.4 | 3.6 | +20.0% |
| Amgen | $409.90 | +4.26% | $212.19B | 24.4 | 17.5 | 5.6 | +9.7% |
| PTC Therapeutics | $65.95 | +1.06% | $5.45B | — | 53.3 | 5.4 | +101.6% |
| Sarepta Therapeutics | $20.86 | +3.57% | $2.13B | — | 36.3 | 1.1 | −34.3% |
| Median | 94.3 | 26.9 | 4.5 | +14.8% |