Develops treatments for rare and ultra-rare genetic diseases, focusing on unmet medical needs.
Ultragenyx Pharmaceutical Inc. is a dynamic biopharmaceutical company dedicated to advancing the field of rare and ultra-rare genetic diseases through a comprehensive approach encompassing product identification, acquisition, development, and global commercialization. Operating across North America, Europe, and beyond, the company focuses on pioneering biologic therapies that address significant unmet medical needs.
Among its portfolio of innovative biologic products is Crysvita (burosumab), an antibody designed to target fibroblast growth factor 23, offering treatment solutions for conditions like X-linked hypophosphatemia and tumor-induced osteomalacia. Additionally, Ultragenyx Pharmaceutical Inc. offers Mepsevii, an enzyme replacement therapy crucial for managing Mucopolysaccharidosis VII, alongside Dojolvi, which addresses long-chain fatty acid oxidation disorders. Evkeeza (evinacumab) represents another milestone, serving patients with homozygous familial hypercholesterolemia.
The company's robust pipeline includes promising candidates such as DTX401 and DTX301, both utilizing adeno-associated virus 8 (AAV8) gene therapy for glycogen storage disease type Ia and ornithine transcarbamylase deficiency, respectively. Other noutable developments include UX143, a human monoclonal antibody for osteogenesis imperfecta, GTX-102 targeting Angelman syndrome with antisense oligonucleotide therapy, and UX701 and UX053 for Wilson disease and glycogen storage disease type III, respectively. Collaborations with esteemed partners like Kyowa Kirin Co., Ltd., REGENXBIO Inc., and Bayer Healthcare LLC underscore Ultragenyx Pharmaceutical Inc.'s commitment to pioneering treatments that make a profound difference in patients lives.