Ultragenyx is a rare disease biopharmaceutical company that develops and commercializes treatments for serious rare and ultra-rare genetic diseases. The company has four approved products: Crysvita (burosumab) for X-Linked Hypophosphatemia, Dojolvi (triheptanoin) for Long-chain Fatty Acid Oxidation Disorders, Evkeeza (evinacumab) for Homozygous Familial Hypercholesterolemia, and Mepsevii (vestronidase alfa) for MPS VII. Crysvita accounts for roughly 70% of total revenue and is the only approved therapy targeting the underlying cause of XLH. Ultragenyx sells directly to specialist physicians — metabolic disease specialists, medical geneticists, and rare disease experts — across more than 35 countries through its own commercial organizations. Most products are in-licensed from academic institutions or other pharma companies. Revenue is driven by patients on therapy and premium per-patient pricing, typical in rare disease where treatments address conditions with no alternatives. Crysvita's revenue structure varies by geography: in the U.S. and Canada, Kyowa Kirin books sales and pays Ultragenyx a revenue share; in Europe, Ultragenyx receives royalties, a portion of which have been sold to OMERS and Royalty Pharma; in Latin America, Ultragenyx books sales directly. The company is currently unprofitable, and management targets GAAP profitability in 2027, driven by commercial revenue growth and R&D expense reduction as late-stage trials complete. The pipeline includes five programs near or at BLA submission, spanning AAV gene therapies, an antisense oligonucleotide, and biologics across multiple rare disease indications.