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Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.


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. 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.. In 2014 the USA-based company went public with an IPO that raised $126 million. In 2015 Ultragenyx began collaborating with Arcturus Therapeutics to develop mRNA products. Ultragenyx acquired Dimension Therapeutics in 2017 to obtain adeno-associated virus -based gene therapy manufacturing technology as well as three internal candidates and one partnered candidate with Bayer for Hemophilia A.In 2017, the company discontinued development of Ace-ER's 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. Previous trials in mice had shown efficacy. In 2020 Ultragenyx announced a new Gene Therapy Plant being built near Boston.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. The deal was completed in August 2022.

A bold mission with great benefits Ultragenyx employees bring their best to work every day, and we honor their dedication by providing a comprehensive benefits program that supports their health care, family, and financial needs. All regular full time employees are eligible for coverage starting with the first day of employment.

Our vision is to be a leader in the development of breakthrough therapeutics and diagnostics for rare and ultra-rare diseases.

Key Team

Mr. Erik Harris (Exec. VP & Chief Commercial Officer)

Mr. Theodore A. Huizenga (Sr. VP, Corp. Controller & Principal Accounting Officer)

Mr. Dennis Karl Huang (Chief Technical Operations Officer & Exec. VP)

Ms. Danielle Keatley (Sr. Director of Investor Relations & Corp. Communications)

Ms. Karah Herdman Parschauer J.D. (Chief Legal Officer & Exec. VP)

Mr. Ernie W. Meyer (Chief HR Officer & Exec. VP)

Mr. Thomas R. Kassberg (Chief Bus. Officer & Exec. VP)

Recognition and Awards
Ultragenyx has been recognized for our commitment to rare and ultra-rare disease treatment, including topping the list of the 2019 Scrip Rare Disease Companies of the Year and for having the top FDA-approved treatment for Fanconi Anemia in 2018.

Leadership team

Dr. Emil D. Kakkis M.D., Ph.D. (Founder, Pres, CEO & Director)

Mr. John Richard Pinion II (Chief Quality Operations Officer & Exec. VP of Translational Sciences)

Dr. Camille L. Bedrosian M.D. (Chief Medical Officer & Exec. VP)

Products/ Services
Biotechnology, Health Care, Pharmaceutical
Number of Employees
1,000 - 20,000
Novato, California, United States
Company Registration
SEC CIK number: 0001515673
100M - 500M
Traded as
Social Media
Thu Feb 29 2024

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