Ultragenyx Pharmaceutical Inc.

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company dedicated to the research, development, and commercialization of novel products for serious rare and ultra-rare genetic diseases. Its mission is to bring life-altering treatments to patients with conditions that often have limited or no approved therapeutic options. The company's headquarters are located in Novato, California, USA.

The company's portfolio includes several approved therapies such as Crysvita (burosumab) for X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO), Mepsevii (vestronidase alfa) for mucopolysaccharidosis VII (MPS VII), and Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders (LC-FAOD). Ultragenyx also markets Evkeeza (evinacumab) outside the U.S. for homozygous familial hypercholesterolemia (HoFH). The company focuses on genetically defined rare diseases, utilizing diverse drug modalities including biologics, small molecules, gene therapies (AAV), and antisense oligonucleotides (ASO).

Led by Founder, President, and CEO Emil Kakkis, Ultragenyx is a commercial-stage biopharmaceutical company with a robust clinical pipeline. In April 2026, the U.S. FDA accepted the Biologics License Application (BLA) resubmission for UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA), with a PDUFA action date set for September 19, 2026. Additionally, the company reported positive 36-week data from a Phase 3 study of DTX301 AAV8 gene therapy for Ornithine Transcarbamylase (OTC) Deficiency in March 2026. Ultragenyx has also outlined a restructuring plan in early 2026 to reduce operating expenses and target profitability in 2027, and operates its own gene therapy manufacturing facility in Bedford, Massachusetts.