Ultragenyx Pharmaceutical, founded in 2010 and headquartered in Novato, California, is a biopharmaceutical company dedicated to developing innovative treatments for rare and ultra-rare genetic diseases. The company's mission is to address high unmet medical needs by advancing novel therapies across various disease categories, including bone, endocrine, metabolic, muscle, and CNS diseases. Ultragenyx employs multiple drug modalities such as biologics, small molecules, gene therapies, ASOs, and mRNAs to deliver transformative treatments.

Key figures affiliated with Ultragenyx include its president and founder, Emil Kakkis. The company is publicly traded on Nasdaq under the ticker RARE and is a component of the Russell 1000 index. Notable achievements include generating $271 million in revenue in 2020 and maintaining a robust clinical pipeline. With nearly 900 employees, Ultragenyx has made significant strides in the biotechnology and life sciences industries, positively impacting patients with rare diseases worldwide.