Ultragenyx Pharmaceutical Inc.

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing treatments for rare and ultra-rare genetic diseases. Its approved biologic products include Crysvita (burosumab) for X-linked hypophosphatemia and tumor-induced osteomalacia, Mepsevii for Mucopolysaccharidosis VII, Dojolvi for long-chain fatty acid oxidation disorders, and Evkeeza (evinacumab) for homozygous familial hypercholesterolemia. The company also has a robust pipeline with candidates in Phase 3 trials, such as UX143 for osteogenesis imperfecta, and gene therapies like UX111 for Sanfilippo syndrome type A, DTX401 for glycogen storage disease type Ia, DTX301 for ornithine transcarbamylase deficiency, and GTX-102 for Angelman syndrome. Additionally, UX701, an AAV9 gene therapy for Wilson disease, is in Phase 2 trials.

Ultragenyx Pharmaceutical Inc. is listed on the Nasdaq Global Select Market (NasdaqGS) under the ticker symbol RARE. Its shares are traded in US dollars (USD), reflecting its primary listing in the United States. The company is headquartered in the United States and operates in the US region.

Ultragenyx Pharmaceutical Inc. operates in the Healthcare sector, specifically within the Biotechnology industry. As a biotechnology company, it focuses on the identification, acquisition, development, and commercialization of novel therapies for rare and ultra-rare genetic diseases. This places it at the intersection of advanced biological research and pharmaceutical development, targeting conditions with high unmet medical needs.

Ultragenyx Pharmaceutical Inc. operates across multiple regions globally, including North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. This broad geographic presence allows the company to commercialize its approved products, such as Crysvita, Mepsevii, Dojolvi, and Evkeeza, in various international markets. The company's headquarters is in the United States, and its operations are coordinated from there to serve patients with rare genetic diseases worldwide.

Ultragenyx has several promising product candidates in clinical development. UX143, a human monoclonal antibody for osteogenesis imperfecta, is in Phase 3 trials. Gene therapies include UX111 (AAV9) for Sanfilippo syndrome type A, DTX401 (AAV8) for glycogen storage disease type Ia, DTX301 (AAV8) for ornithine transcarbamylase deficiency, and GTX-102, an antisense oligonucleotide for Angelman syndrome. Additionally, UX701, an AAV9 gene therapy for Wilson disease, is in Phase 2. These candidates target severe genetic disorders with limited treatment options, highlighting the company's focus on innovative therapies.