Ultragenyx
Ultragenyx is a biopharmaceutical company dedicated to developing innovative treatments for rare and ultrarare genetic diseases.
Description
Ultragenyx Pharmaceutical Inc. is an American biopharmaceutical company dedicated to developing novel therapies for rare and ultra-rare genetic diseases.
Founded in 2010 by Dr. Emil Kakkis, Ultragenyx focuses on serious rare and ultra-rare genetic conditions that often lack approved treatments. The company employs a diverse range of therapeutic modalities, including biologics, small molecules, gene therapies, and nucleic acid products, to address diseases in areas such as bone, endocrine, metabolic, muscle, and central nervous system disorders. Headquartered in Novato, California, Ultragenyx has expanded its presence with facilities in Brisbane, California, and the Boston area, as well as a Latin American headquarters in Miami.
Key Products and Services:
- Crysvita (burosumab): A fully human monoclonal antibody approved for the treatment of X-linked hypophosphatemia (XLH), a rare inherited disorder that causes low levels of phosphate in the blood.
- Dojolvi (triheptanoin): A highly purified, synthetic, 7-carbon fatty acid triglyceride approved for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare metabolic conditions.
- Mepsevii (vestronidase alfa): An enzyme replacement therapy approved for the treatment of Mucopolysaccharidosis VII (Sly syndrome), a rare lysosomal storage disorder.
- Evkeeza (evinacumab): A monoclonal antibody for the treatment of homozygous familial hypercholesterolemia (HoFH), a genetic condition characterized by extremely high cholesterol levels.
- Gene Therapy Programs: Including DTX401 for glycogen storage disease type Ia (GSDIa), DTX301 for ornithine transcarbamylase (OTC) deficiency, and UX701 for Wilson disease, all in various stages of clinical development.
- Collaborative Research: Partnerships with academic institutions and biotechnology companies to advance product candidates, exemplified by collaborations with GeneTx for Angelman syndrome and Mereo BioPharma for osteogenesis imperfecta.
Ultragenyx is committed to transforming the lives of patients with rare and ultra-rare diseases by delivering innovative therapies where none previously existed. Their dedication to scientific excellence and patient-centric approach has established them as a leader in the biopharmaceutical industry, continually striving to address unmet medical needs and improve patient outcomes worldwide.