Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) is a biopharmaceutical company dedicated to the development and commercialization of therapies for rare and ultra-rare genetic diseases. Since its founding in 2010 and headquartered in Novato, California, Ultragenyx has focused on leveraging advanced genetic, enzymatic and biologic approaches to address high unmet medical needs. The company’s mission centers on translating cutting-edge science into treatments that improve quality of life for patients with serious inherited disorders.
Ultragenyx’s commercial portfolio includes therapies that target specific metabolic and bone disorders. Its lead products address conditions such as X-linked hypophosphatemia and mucopolysaccharidosis type VII, while its pipeline features candidates for fatty acid oxidation disorders, congenital disorders of glycosylation, and other rare diseases. The company applies a variety of modalities—enzyme replacement, gene therapy and antibody approaches—to build a diversified pipeline aimed at long-term disease modification and symptom management.
Operating globally, Ultragenyx pursues regulatory approvals and market launches across North America, Europe, Asia and other regions. The company has established partnerships and collaborations with academic institutions, patient advocacy groups and industry peers, facilitating clinical development and broadening access to therapies. By maintaining manufacturing capabilities and distribution networks in key markets, Ultragenyx works to ensure timely supply and support for patients and healthcare providers worldwide.
Leadership at Ultragenyx is spearheaded by a team of industry veterans with deep expertise in drug development and regulatory affairs. Emil Kakkis, M.D., Ph.D., serves as Chief Executive Officer and brings extensive experience in rare disease research and patient advocacy. Under his guidance, the company has grown from an early-stage biotech to a commercial organization, building a robust pipeline and forging strategic alliances. With a commitment to scientific innovation and patient-centricity, Ultragenyx continues to expand its reach in the rare disease community.
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