Ultragenyx Pharmaceutical Inc
RARE
Ultragenyx is a 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 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.
$43.85
$-0.43 (-0.971%)Apr 25
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About Ultragenyx Pharmaceutical Inc
Ultragenyx is a 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 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.
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