Ultragenyx

Ultragenyx Pharmaceutical Inc.
Company typePublic
Industry
Founded2010; 14 years ago (2010)
FounderEmil Kakkis
Headquarters
Novato, California
,
U.S.
Areas served
International
Key people
Emil Kakkis (president)
Products
RevenueIncrease US$271 million (2020)[1]
Number of employees
893 (2020)
Websiteultragenyx.com

Ultragenyx Pharmaceutical Inc. 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[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]

  1. ^ "Ultragenyx Pharmaceutical Revenue 2012-2021 | RARE".
  2. ^ "Ultragenyx reports nearly $73M Q3 loss as R&D spending up 30%". The North Bay Business Journal. 2021-11-03. Retrieved 2022-02-23.
  3. ^ "Rare disease drug maker Ultragenyx plans to hire 100-150 at new gene therapy facility near Boston". www.bizjournals.com. Retrieved 2022-02-23.
  4. ^ Bartucci, Camila (2021-11-23). "Ultragenyx abre central en Miami, Latam". PharmaBiz.NET (in Spanish). Retrieved 2022-03-03.
  5. ^ Denworth, Lydia (2020). "Is gene therapy ready to treat some forms of autism?". Science. doi:10.1126/science.abf2497. S2CID 242454742.
  6. ^ "Novato Firm Partners With Rival To Treat Rare Illness". Novato, CA Patch. 2022-01-21. Retrieved 2022-03-15.
  7. ^ "Health Canada Approves Ultragenyx' Drug for Group of Rare Metabolic Disorders". Global Genes. 2021-02-17. Retrieved 2022-03-18.
  8. ^ "2021 Technology Fast 500 Award Winners". Deloitte United States. Retrieved 2022-03-18.
  9. ^ "BioSpace | Best Places to Work jobs | Choose from 3,724 live job openings". www.biospace.com. Retrieved 2022-03-22.
  10. ^ "Marin County pharmaceuticals CEO wins lifetime achievement award". The North Bay Business Journal. 2021-11-17. Retrieved 2022-03-25.