Ultragenyx

American biopharmaceutical company

Ultragenyx company logo

Ultragenyx Pharmaceutical Inc. is an American biopharmaceutical company focused on the development of novel products for the treatment of rare and ultra-rare genetic diseases. The company is headquartered in Novato, California, and was founded in 2010 by Dr. Emil D. Kakkis, a physician and scientist with extensive experience in the field of rare diseases.

History

Ultragenyx was established with the mission to address the unmet medical needs of patients with rare genetic disorders. The company was founded by Dr. Emil Kakkis, who previously worked on the development of enzyme replacement therapies for rare diseases. Since its inception, Ultragenyx has focused on developing therapies for conditions that have limited or no treatment options.

Research and Development

Ultragenyx's research and development efforts are centered around several key therapeutic areas, including metabolic disorders, bone diseases, and neurological disorders. The company employs a variety of approaches, including gene therapy, enzyme replacement therapy, and small molecule drugs, to develop treatments for these conditions.

Gene Therapy

Gene therapy is a major focus for Ultragenyx, as it offers the potential to address the underlying genetic causes of many rare diseases. The company is actively developing gene therapies for conditions such as X-linked hypophosphatemia and glycogen storage disease type Ia.

Enzyme Replacement Therapy

Ultragenyx has developed enzyme replacement therapies for several rare metabolic disorders. These therapies aim to replace deficient or defective enzymes in patients, thereby alleviating symptoms and improving quality of life.

Small Molecule Drugs

In addition to biologics, Ultragenyx is also exploring small molecule drugs as potential treatments for rare diseases. These drugs are designed to modulate specific biochemical pathways involved in disease pathogenesis.

Products

Ultragenyx has developed and commercialized several products for the treatment of rare diseases. Some of the key products include:

• Crysvita (burosumab-twza): A monoclonal antibody for the treatment of X-linked hypophosphatemia, a rare genetic disorder that affects bone mineralization.
• Mepsevii (vestronidase alfa-vjbk): An enzyme replacement therapy for the treatment of mucopolysaccharidosis VII, also known as Sly syndrome.
• Dojolvi (triheptanoin): A synthetic triglyceride used as a source of calories and fatty acids for patients with long-chain fatty acid oxidation disorders.

Collaborations and Partnerships

Ultragenyx has established several collaborations and partnerships with other biopharmaceutical companies and research institutions to advance its pipeline of therapies. These collaborations are aimed at leveraging complementary expertise and resources to accelerate the development of new treatments.

Corporate Social Responsibility

Ultragenyx is committed to corporate social responsibility and actively engages in initiatives to support patients with rare diseases. The company works closely with patient advocacy groups and participates in efforts to raise awareness about rare diseases and the challenges faced by affected individuals and families.

See also

• Biopharmaceutical company
• Rare disease
• Gene therapy
• Enzyme replacement therapy

Related pages

• List of pharmaceutical companies
• Orphan drug
• Genetic disorder