Ultragenyx Pharmaceutical Inc., headquartered in Novato, California, is a biopharmaceutical company dedicated to the development and commercialization of novel therapies for rare and ultra-rare genetic diseases. Since its founding in 2010, Ultragenyx has focused on disorders with high unmet medical need, particularly those affecting metabolic or skeletal systems. The company’s portfolio includes several approved products, such as Crysvita (burosumab) for X-linked hypophosphatemia, Mepsevii (vestronidase alfa-vjbk) for mucopolysaccharidosis VII, and Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders. These therapies have been approved in major markets including the United States, European Union, United Kingdom and Canada.
In addition to its commercial treatments, Ultragenyx maintains a robust pipeline of gene therapy candidates targeting conditions such as ornithine transcarbamylase (OTC) deficiency and glycogen storage disease type Ia. The company has formed strategic collaborations, notably with Kyowa Kirin for the development and distribution of Crysvita outside of North America and with academic institutions to accelerate early-stage research. Ultragenyx’s integrated approach combines in-house research capabilities with external partnerships to advance both enzyme replacement and gene editing platforms.
Under the leadership of President and Chief Executive Officer Emil Kakkis, M.D., Ph.D., Ultragenyx emphasizes patient-centric development and regulatory engagement to bring transformative medicines to market. The company’s global operations include commercial and clinical teams across North America, Europe and Asia, supported by manufacturing sites in the United States. Ultragenyx continues to invest in expanding access to its therapies, with ongoing initiatives to broaden indications and geographic reach for patients living with rare genetic disorders.
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