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Ultragenyx Pharmaceutical Inc   (RARE)
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Price: $47.3700 $0.72 1.543%
Day's High: $47.63 Week Perf: -2.35 %
Day's Low: $ 46.22 30 Day Perf: 3.75 %
Volume (M): 1,792 52 Wk High: $ 54.98
Volume (M$): $ 84,887 52 Wk Avg: $42.62
Open: $46.61 52 Wk Low: $31.52



 Market Capitalization (Millions $) 3,395
 Shares Outstanding (Millions) 72
 Employees 1,000
 Revenues (TTM) (Millions $) 410
 Net Income (TTM) (Millions $) -635
 Cash Flow (TTM) (Millions $) -318
 Capital Exp. (TTM) (Millions $) 72

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
   


   

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Shares

Ultragenyx's Strategic Move: Unlocking Employee Potential and Driving Company Growth through Restricted Stock Units)

Published Fri, Feb 23 2024 9:05 PM UTC

Ultragenyx Awards Restricted Stock Units to New Non-Executive Officers: A Strong Signal of Growth and Attractiveness to Top Talent
NOVATO, Calif., Feb. 23, 2024 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company on the forefront of developing and commercializing novel therapies for rare and ultrarare diseases, announced today the issuance of 10,...

Clinical Study

UX111: Advancements in Reducing Heparan Sulfate Exposure for Improved Cognitive Function in Sanfilippo Syndrome Type A

Published Tue, Feb 6 2024 1:00 PM UTC

Abstract:Sanfilippo Syndrome Type A, also known as Mucopolysaccharidosis type IIIA (MPS IIIA), is a rare genetic disorder characterized by the accumulation of heparan sulfate within the body, leading to severe neurological impairment and cognitive decline. Ultragenyx, a biopharmaceutical company, has recently introduced UX111, a potential groundbreaking treatment for MPS III...

Clinical Study

Unveiling the Promising Frontier in Wilson Disease Therapy: UX701 Gene Therapy Raises Hope for Novel Treatment Approaches

Published Thu, Jan 25 2024 9:05 PM UTC

Wilson Disease (WD) is a rare, genetic disorder characterized by the body's inability to properly metabolize copper, leading to its accumulation in vital organs such as the liver, brain, and other tissues. Current treatment options for WD, namely medical therapy and liver transplantation, have limitations, emphasizing the need for innovative therapeutic strategies. In this a...

Shares

Ultragenyx Pharmaceutical Grants Restricted Stock Units to Newly Hired Officers, Demonstrating Commitment to Employee Retention

Published Fri, Jan 19 2024 9:00 PM UTC



NOVATO, Calif., Jan. 19, 2024 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a leading biopharmaceutical company specializing in the development and commercialization of groundbreaking therapies for rare and ultrarare diseases, recently announced the issuance of 12,130 restricted stock units (RSUs) to eight recently recruited non-executive officers. The decision ...

Product Service News

Ultragenyx's Evkeeza receives NICE Recommendation as Breakthrough Treatment for Homozygous Familial Hypercholesterolemia in the UK

Published Thu, Jan 4 2024 1:02 PM UTC

Ultragenyx's Evkeeza Receives Positive Recommendation for Homozygous Familial Hypercholesterolemia Treatment in the UKJanuary 4, 2024Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has recently announced that the National Institute for Health and Care Excellence (NICE) in the UK has issued a final draft guidance recommending the use of Evkeeza (evinacumab) to NHS England. This...






 

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