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Ultragenyx Pharmaceutical Inc. is a clinical-stage biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of products for the treatment of genetic diseases. Its clinical-stage pipeline consists of two product categories: biologics (including a monoclonal antibody and an enzyme replacement therapy), and small-molecule substrate replacement therapies. Its product candidates under biologics category include KRN23 (UX023) and recombinant human beta-glucuronidase (rhGUS) (UX003). Its product candidates under small-molecule category include UX007 and aceneuramic acid extended-release (Ace-ER) (UX001). It is also developing recombinant human protective protein cathepsin-A (rhPPCA). KRN23 is a fully human monoclonal antibody. rhGUS is an intravenous (IV) enzyme replacement therapy. UX007 is a substrate replacement therapy. It is developing Ace-ER for the treatment of GNE myopathy. rhPPCA is in preclinical development.
Number of employees : 520 people.
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| 2016 | 2017 | Delta | | USD (in Million) | % | USD (in Million) | % | | Rare Diseases Treatment Products | 0.133 | 100% | - | - | - | | Collaboration and License | - | - | 2.14 | 81.8% | - | | Product | - | - | 0.476 | 18.2% | - |
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| 2016 | 2017 | Delta | | USD (in Million) | % | USD (in Million) | % | | United States | 0.133 | 100% | 2.61 | 100% | +1863.91% |
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| Vote  | Quantity | Float | Company-owned shares | Total Float |
| Share A |
1 |
50,579,619 |
47,314,969 |
93.5% |
0 |
0.0% |
93.5% |
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| Name | Equities | % | | Capital Research & Management Co. (Global Investors) | 5,856,138 |
11.6% | | Wellington Management Co. LLP | 5,274,908 |
10.4% | | Capital Research & Management Co. (International Investors) | 4,359,891 |
8.62% | | The Vanguard Group, Inc. | 3,951,864 |
7.81% | | BlackRock Fund Advisors | 3,546,199 |
7.01% | | Fidelity Management & Research Co. | 3,310,512 |
6.55% | | Emil D. Kakkis MD, PhD | 3,010,213 |
5.95% | | T. Rowe Price Associates, Inc. | 2,778,029 |
5.49% | | SSgA Funds Management, Inc. | 1,712,524 |
3.39% | | Capital Research & Management Co. (World Investors) | 1,461,390 |
2.89% |
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| Company contact information |
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Ultragenyx Pharmaceutical, Inc. 60 Leveroni Court Novato, CA 94949 Phone : +1.415.483.8800 Fax : +1.415.483.8810 Web : http://www.ultragenyx.com |
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Sector Bio Therapeutic Drugs
Connections : Ultragenyx Pharmaceutical |
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American Society of Hematology | |
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Global Alliance for TB Drug Development |
Blackthorn Therapeutics, Inc. |
American Society of Nephrology | |
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American Society of Clinical Oncology |
Emil Kakkis & Jenny Soriano Living Trust | |
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Everylife Foundation for Rare Diseases |
Dimension Therapeutics, Inc. |
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United Kingdom
France
Deutschland
Schweiz (DE)
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