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Ultragenyx Pharmaceutical Inc   (RARE)
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Price: $50.3600 $-0.07 -0.139%
Day's High: $51.45 Week Perf: -1.37 %
Day's Low: $ 49.96 30 Day Perf: -6.52 %
Volume (M): 1,247 52 Wk High: $ 60.37
Volume (M$): $ 62,784 52 Wk Avg: $47.11
Open: $50.80 52 Wk Low: $34.06



 Market Capitalization (Millions $) 4,809
 Shares Outstanding (Millions) 95
 Employees 1,276
 Revenues (TTM) (Millions $) 523
 Net Income (TTM) (Millions $) -559
 Cash Flow (TTM) (Millions $) 82
 Capital Exp. (TTM) (Millions $) 21

Ultragenyx Pharmaceutical Inc
Ultragenyx Pharmaceutical Inc is a biopharmaceutical company that focuses on the development and commercialization of innovative therapies for the treatment of rare and ultra-rare genetic diseases. The company's mission is to provide novel treatments for these rare diseases where there is unmet medical need.

The company was founded in 2010 by Emil Kakkis, who is a pioneer in the field of rare diseases. Ultragenyx started as a small startup and has grown to become one of the leading rare disease biotech companies in the industry. It is headquartered in Novato, California, USA, and has operations in Europe as well.

Ultragenyx is known for its expertise in developing and commercializing rare disease therapies. The company has a robust pipeline of programs that are aimed at addressing a range of rare diseases. Some of the key therapeutic areas the company is focused on include metabolic disorders, skeletal disorders, neurological disorders, and immunology.

One of the company's top products is Mepsevii (vestronidase alfa), which is an enzyme replacement therapy for the treatment of a rare genetic disorder called mucopolysaccharidosis VII (MPS VII). This condition is caused by a deficiency in beta-glucuronidase, which leads to the accumulation of certain complex sugars in the body tissues and organs. Mepsevii works by replacing the missing enzyme in the patient's body, thereby reducing the accumulation of complex sugars and improving the patient's symptoms.

Another product under development by Ultragenyx is DTX301, a gene therapy product for the treatment of ornithine transcarbamylase (OTC) deficiency. OTC deficiency is a rare genetic disorder that affects the body's ability to break down ammonia, leading to its build-up in the blood and tissues. DTX301 works by introducing a healthy copy of the OTC gene into the patient's liver cells, which helps to restore the body's ability to break down ammonia.

Ultragenyx has a strong commitment to research and innovation, and it is constantly exploring new approaches to treat rare diseases. The company has a team of highly experienced professionals who are dedicated to bringing innovative medicines to patients with rare diseases. It has also established partnerships with other companies and organizations in the industry to advance its research and development programs.

In summary, Ultragenyx Pharmaceutical Inc is a biopharmaceutical company that is dedicated to the development and commercialization of innovative therapies for the treatment of rare and ultra-rare genetic diseases. The company has a broad pipeline of rare disease programs, and it is committed to advancing its research and development efforts to bring new treatments to patients in need.


   Company Address: 60 Leveroni Court Novato 94949 CA
   Company Phone Number: 483-8800   Stock Exchange / Ticker: NASDAQ RARE


   

Stock Performances by Major Competitors

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FOLD   -3%    
INSM        8% 
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PRAX        11.22% 
VRTX        9.29% 
• View Complete Report
   



Shares

Ultragenyx Cultivates Growth Through Strategic Inducement Grants Amidst Market Fluctuations

Published Fri, Oct 18 2024 8:30 PM UTC

Ultragenyx Pharmaceutical Inc.: Navigating Employee Incentives and Market Dynamics in the Rare Disease SectorIn a strategic move aimed at fortifying its talent base and enhancing employee retention, Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) recently announced the grant of 22,405 restricted stock units (RSUs) to six newly hired non-executive officers. This decision, made ...

Management Announcement

Ultragenyx Achieves Breakthrough Therapy Status for Osteogenesis Imperfecta as OKX Explorer Enhances Web3 Analytics with Bitlayer Integration

Published Mon, Oct 7 2024 12:00 PM UTC

In a significant stride for patients with rare bone disorders, Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a company committed to innovating treatments for rare and ultra-rare diseases, has proudly announced that it has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for its investigational drug, setrusumab (UX143). This therap...

Clinical Study

Ultragenyx Provides Update on Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for...

Published Thu, Oct 3 2024 8:30 PM UTC

Novel Developments in Gene Therapy: Ultragenyx Updates on UX701 for Wilson Disease in Cyprus2+ Study Recent updates from Ultragenyx Pharmaceutical Inc. regarding their pivotal Phase 1/2/3 Cyprus2+ study highlight significant advancements in the development of UX701, a novel gene therapy designed for the treatment of Wilson Disease. This breakthrough comes at a crucial time ...

Product Service News

OKX Explorer Expands Capabilities with Bitlayer Support, Enhancing Web3 Analytics

Published Tue, Sep 3 2024 6:00 AM UTC

On September 3, 2024, OKX Explorer, a prominent blockchain search engine and analytics platform, announced the integration of support for Bitlayer, a platform designed for the management and analysis of blockchain data. This new addition aims to bolster the capabilities of OKX Explorer, allowing users to leverage advanced tools for navigating and understanding the complexiti...

Announcement

Ultragenyx Induces Growth 12 New Executives Boost Talent Pool with 37,975 Restricted Stock Units,

Published Wed, Aug 21 2024 8:30 PM UTC

Ultragenyx Welcomes New Talent with Stock Incentives: A Strategic Move in the Biopharmaceutical SectorIn a significant move to enhance its workforce, Ultragenyx Pharmaceutical Inc., a leading biopharmaceutical company dedicated to developing novel therapies for rare and ultrarare diseases, has announced the grant of 37,975 restricted stock units (RSUs) to 12 newly hired non...







Ultragenyx Pharmaceutical Inc's Segments
Product Revenue    55.38 % of total Revenue
Product Revenue Crysvita    25.52 % of total Revenue
Product Revenue Dojolvi    15.32 % of total Revenue
Product Revenue Mepsevii    6.89 % of total Revenue
Product Revenue Evkeeza    7.64 % of total Revenue
Royalty revenue    44.62 % of total Revenue
Royalty revenue Crysvita    44.62 % of total Revenue
North America    55.14 % of total Revenue
Latin America    27.28 % of total Revenue
Europe Middle East and Africa    15.93 % of total Revenue
Asia-Pacific    1.66 % of total Revenue

  Ultragenyx Pharmaceutical Inc Outlook

On January 7 2024 the Ultragenyx Pharmaceutical Inc provided following guidance

Ultragenyx Pharmaceutical Inc. has released a preliminary report on its estimated 2023 revenue, as well as provided guidance for its 2024 revenue and cash usage, along with updates on its pipeline and upcoming milestones for 2024.

According to the preliminary report, Ultragenyx expects its total revenue for 2023 to range between $430 million and $435 million. This figure includes the expected revenue from two of its key products, Crysvita and Dojolvi. Crysvita is projected to contribute revenue in the range of $325 million to $330 million, while Dojolvi is expected to generate revenue of approximately $70 million to $71 million in 2023.

Looking ahead to 2024, Ultragenyx has shared its guidance ...





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