Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company founded in 2010 that is focused on the development of novel therapies for rare and ultra-rare genetic diseases. The company’s headquarters is located in Novato, California, United States, and it has additional locations in Brisbane and South San Francisco. Ultragenyx Pharmaceutical has a pipeline of approximately ten product candidates in clinical development, with a focus on developing transformative therapies for serious and rare diseases such as lysosomal storage disorders, gene therapy, and hematologic and metabolic disorders. In late 2019, Ultragenyx Pharmaceutical received FDA approval for its first product, MEPSEVII® (vestronidase alfa-vjbk), an enzyme replacement therapy for the treatment of children and adults with mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome. Ultragenyx’s product development strategy focuses on leveraging its internal research and development capabilities, along with external collaborative relationships, to identify and advance promising new therapies for patients with rare and ultra-rare diseases. Ultragenyx Pharmaceutical is committed to advancing the field of rare disease research and improving the lives of patients affected by these debilitating conditions. The company’s mission is to become the world’s leading rare disease company by developing and delivering transformative therapies to patients in need.