Ultragenyx Pharmaceutical Inc (Ultragenyx) identifies, develops and markets novel products for the treatment of various rare and ultra-rare genetic diseases. The company operates through single reportable segment and offers four marketed products: Crysvita, Dojolvi, Evkeeza, and Mepsevii. Its pipeline product candidates are either in-licensed from academic institutions or derived from partnerships with other pharmaceutical and biotechnology companies. Ultragenyx’s approved therapies and clinical-stage pipeline consist of four categories: biologics, small molecules, gene therapy, nucleic acid product candidates.

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Ultragenyx conducts research to advance its pipeline including biologics that include a monoclonal antibody and an enzyme replacement therapy; and small-molecule substrate replacement therapies for the treatment of various serious rare and ultra-rare genetic diseases with unmet medical needs. The company’s product pipeline has been either in-licensed from academic institutions or derived from partnerships with other pharmaceutical companies. Ultragenyx’s biologics pipeline includes UX143 (setrusumab), a human monoclonal antibody that inhibits sclerostin in Phase III for the treatment of osteogenesis imperfecta (OI). The company entered into collaboration and license agreement with Mereo BioPharma Limited to develop and commercialize UX143 (setrusumab) in the US. The AAV gene therapy candidates include UX111, DTX301, and DTX401. Ultragenyx is investigating DTX301 in Phase III trials for the treatment of ornithine transcarbamylase (OTC) deficiency; DTX401 in Phase III clinical trials for the treatment of glycogen storage disease type Ia (GSDIa), a disease that is caused by a defective G6Pase enzyme, an essential enzyme for glucose and glycogen metabolism; and UX701 in Phase II trials for the treatment of Wilson disease. Its nucleic acid pipeline includes GTX-102, an antisense oligonucleotide (ASO) for the treatment of Angelman syndrome. The company entered into collaboration agreement with GeneTx to develop GeneTx’s GTX-102. In FY2024, the company spent US$697.9 million on its R&D activities, which grew 7.6% YoY. As of December 31, 2024, the company held over 275 active patents covering its products and methods, including more than 50 from the US Patent and Trademark Office. Additionally, it had over 325 pending patent applications, with more than 50 in the US. As of December 2024, the company had 875 employees working on its research and development.