Ultragenyx Pharmaceutical Inc (Ultragenyx) discovers, develops, and commercializes therapies for rare and ultra-rare genetic diseases. It operates as a single reportable segment and markets four products: Crysvita, Dojolvi, Evkeeza, and Mepsevii. The company’s pipeline candidates are sourced through licenses from academic institutions and collaborations with pharmaceutical and biotechnology partners. Across both approved medicines and the clinical pipeline, Ultragenyx focuses on four technology areas: biologics, small molecules, gene therapy, and nucleic acid–based candidates.

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Ultragenyx has undertaken various R&D initiatives focused on identifying, acquiring, and developing products and product candidates, including conducting clinical studies and supporting commercialization efforts. The company is engaged in substantial research and development activities, particularly in gene therapy programs, biologic and nucleic acid programs, and translational research. The R&D unit of Ultragenyx Pharmaceutical Inc is responsible for managing the company's research and development activities, including clinical studies, manufacturing of drug substances and products, and translational research. The unit also oversees strategic licensing agreements and infrastructure costs related to laboratory, IT, and equipment depreciation. Ultragenyx's biologics pipeline features UX143 (setrusumab), a human monoclonal antibody that inhibits sclerostin and is in Phase III trials for the treatment of osteogenesis imperfecta (OI). Ultragenyx has a collaboration and license agreement with Mereo BioPharma Limited for the development and commercialization of UX143 in the US. Its AAV gene therapy candidates include UX111, DTX301, and DTX401. DTX301 is in Phase III trials for treating ornithine transcarbamylase (OTC) deficiency, while DTX401 is in Phase III trials for glycogen storage disease type Ia (GSDIa), a condition caused by a defective G6Pase enzyme, crucial for glucose and glycogen metabolism. UX701 is in Phase II trials for treating Wilson disease. The nucleic acid pipeline includes GTX-102, an antisense oligonucleotide (ASO) for Angelman syndrome, developed in collaboration with GeneTx. Additionally, UTX055 is in preclinical trials for the treatment of CDKL5 deficiency disorder. In FY2025, the company spent US$750 million on its R&D activities, which grew 7.4% YoY. As of December 31, 2025, the company held over 300 active patents covering its products and methods, including more than 50 from the US Patent and Trademark Office. Additionally, it had over 300 pending patent applications, with more than 50 in the US. As of December 2025, the company had 916 employees working on its research and development.