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.
Quick Facts Company type, Traded as ...
Ultragenyx Pharmaceutical Inc. |
Company type | Public company |
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Industry | |
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Founded | 2010; 14 years ago (2010) |
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Headquarters | , U.S. |
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Areas served | International |
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Key people | Emil Kakkis (president & founder) |
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Products | |
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Revenue | US$271 million (2020)[1] |
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Number of employees | 893 (2020) |
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Website | ultragenyx.com |
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Ultragenyx is based in Novato, CA and Brisbane, CA[2] and has a presence in the Boston area, including a gene therapy plant under construction as of 2021.[3] The company’s Latin American headquarters is located in Miami.[4]
Ultragenyx collaborates on product development with other companies including GeneTX, Kyowa Hakko Kirin, Mereo Biopharma and Daiichi Sankyo.[5] 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.[6] The company also has therapies approved outside the U.S. in Canada, Latin America, Europe, and Japan.[7]
In 2020 and 2021, Ultragenyx was named one of Deloitte's fasting growing technology and life sciences companies in North America[8] and one of the best companies to work for by BioSpace.[9] Also in 2021, the company’s CEO, Emil Kakkis, was awarded the California Life Sciences Pantheon Leadership award.[10]
Ultragenyx Pharmaceutical Inc. was founded in 2010 by Emil Kakkis based on his history of developing therapies for rare disease starting at Harbor-UCLA Medical Center, continuing through his role as chief medical officer of BioMarin and as founder of the EveryLife Foundation.[11] He became the chief executive officer and president, focusing the company to meet the treatment needs for rare diseases understood as affecting fewer than 200,000 people in the U.S.[12][13] In 2014 the USA-based company went public with an IPO that raised $126 million.[14] In 2015 Ultragenyx began collaborating with Arcturus Therapeutics to develop mRNA products.[15]
Ultragenyx acquired Dimension Therapeutics in 2017[16] to obtain adeno-associated virus (AAV)-based gene therapy manufacturing technology as well as three internal candidates and one partnered candidate with Bayer for hemophilia A.[17]
In 2017, the company discontinued development of Ace-ERs treatment for GNE myopathy after the product didn't meet the study's primary endpoint which was significant improvement in arm strength. It also failed to meet three key secondary endpoints that evaluated patients' physical functions, leg muscle strength and knee extension force.[18] Previous trials in mice had shown efficacy.[19]
In 2020 Ultragenyx announced a new gene therapy plant being built near Boston.[20]
In July 2022 Ultragenyx announced that it would exercise its option to acquire GeneTx Biotherapeutics for an up-front cash payment of $75 million, plus another $115 million in potential milestone-dependent payments. Ultragenyx will add the antisense oligonucleotide therapy GTX-102 to its broad pipeline of therapies indicated for various rare diseases. GTX-102 is currently in early-stage development for Angelman syndrome.[21] The deal was completed in August 2022.