About the Company
Ultragenyx is an American biopharmaceutical company involved in the research and development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small molecule, gene therapies, and ASO and mRNAs in the disease categories of bone, endocrine, metabolic, muscle and CNS diseases. Ultragenyx is based in Novato, CA and Brisbane, CA and has a presence in the Boston area, including a gene therapy plant under construction as of 2021. The company’s Latin American headquarters is located in Miami. Ultragenyx collaborates on product development with other companies including GeneTX, Kyowa Hakko Kirin, Mereo Biopharma and Daiichi Sankyo. Ultragenyx has three products Burosumab, Triheptanoin and Vestronidase alfa that have received FDA approval and several others currently in clinical trials. The company also holds the non-US commercial rights to Regeneron’s evinacumab-dgnb, which is approved by the FDA and EMA. The company also has therapies approved outside the U.S. in Canada, Latin America, Europe, and Japan. In 2020 and 2021, Ultragenyx was named one of Deloitte's fasting growing technology and life sciences companies in North America and one of the best companies to work for by BioSpace. Also in 2021, the company’s CEO, Emil Kakkis, was awarded the California Life Sciences Pantheon Leadership award.
Exchange
Nasdaq
Fundamental Data and Insider Transactions (Quarterly data) Full Screen
Loading the fundamentals chart...
Latest $RARE News
Forecasting The Future: 11 Analyst Projections For Ultragenyx Pharmaceutical
Providing deeper insights, analysts have established 12-month price targets, indicating an average target of $82.55, along ...
Ultragenyx Pharmaceutical gets grant for method of developing stable cell line for producing RAAV
Develop a stable cell line for high-titer rAAV production with Ultragenyx's patented method involving HDAC inhibitors and AAV rep proteins. Explore the groundbreaking patent now!
Why Ultragenyx Pharmaceutical Shares Are Trading Lower By Around 9%? Here Are Other Stocks Moving In Monday's Mid-Day Session
Shares of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) fell sharply during Monday’s session after the company revealed new ...
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
NOVATO, Calif., April 19, 2024 (GLOBE NEWSWIRE) -- (NASDAQ: RARE), a biopharmaceutical company focused on the development ...
Ultragenyx Announces Update to Conference Call Timing to Discuss New Data from GTX-102 in Patients with Angelman Syndrome
Conference call now scheduled for Monday, April 15 at 8:00 a.m. ET Updated registration link below NOVATO, Calif., April 12, ...
Ultragenyx Pharmaceutical Inc RARE
We sell different types of products and services to both investment professionals and individual investors. These products and services are usually sold through license agreements or subscriptions ...
Ultragenyx reports serious adverse effects in rare disease therapy trial
Shares of Ultragenyx Pharmaceutical (RARE) dropped 7% after serious adverse effects were reported in a gene therapy trial for ...
Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102
Expansion Cohorts showed rapid, clinically meaningful improvement across multiple domains; improvements consistent or exceeding Dose-escalation ...
Ultragenyx (RARE) Meets Efficacy Goal in Neuro Disease Study
Ultragenyx (RARE) reports positive interim data from the phase I/II study of GTX-102 for Angelman syndrome. However, the ...
Ultragenyx Pharmaceutical Inc.
For most investors, the best investment strategy is to buy good stocks and hold them for the long run.
Loading the latest forecasts...