Ultragenyx Pharmaceutical Inc. (RARE)

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of various products for the treatment of rare and ultra-rare genetic diseases in the United States. Its biologics product candidate includes Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII. The company is developing various biologics product candidates, including Burosumab, a human monoclonal antibody that is in Phase III study to bind and reduce the biological activity of fibroblast growth factor 23 to enhance abnormally low phosphate levels in patients with X-linked hypophosphatemia, as well as for the treatment of tumor-induced osteomalacia. It is also developing a range of small-molecule product candidates, such as UX007, a substrate replacement therapy that completed Phase II study for patients with fatty acid oxidation disorders, as well as for patients with glucose transporter type-1 deficiency syndrome. In addition, the company is developing adeno-associated virus 8 (AAV8) gene therapy products comprising DTX301, an AAV8 for the treatment of OTC deficiency; DTX401, an AAV8 gene therapy program for the treatment of patients with GSDIa; and DTX201, a FVIII gene therapy program for the treatment of hemophilia A. It has a collaboration agreement with Rentschler Fill Solutions GmbH. Ultragenyx Pharmaceutical Inc. was founded in 2010 and is headquartered in Novato, California.