Ultragenyx Pharmaceutical Inc.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) is a biopharmaceutical company dedicated to developing and commercializing novel therapies for patients with serious rare and ultra-rare genetic diseases. The company maintains its corporate presence at www.ultragenyx.com and operates a diversified portfolio spanning multiple therapeutic modalities including biologics, small molecules, adeno-associated virus gene therapies, and nucleic acid therapeutics.

The company's approved product portfolio consists of four marketed therapies. Crysvita, a monoclonal antibody targeting fibroblast growth factor 23, treats X-Linked Hypophosphatemia and Tumor-Induced Osteomalacia. Mepsevii is an enzyme replacement therapy for Mucopolysaccharidosis VII, while Dojolvi addresses Long-chain Fatty Acid Oxidation Disorders through a synthetic fatty acid triglyceride formulation. Evkeeza, a monoclonal antibody blocking angiopoietin-like 3, treats Homozygous Familial Hypercholesterolemia and is commercialized outside the United States through a partnership with Regeneron Pharmaceuticals.

The clinical pipeline includes multiple late-stage programs. UX111, an AAV9 gene therapy for Sanfilippo syndrome type A, underwent resubmission to the FDA in January 2026 following receipt of a Complete Response Letter. DTX401, an AAV8 gene therapy for Glycogen Storage Disease Type Ia, completed Phase 3 data collection with a rolling BLA submission completed in December 2025. GTX-102, an antisense oligonucleotide for Angelman syndrome, achieved full enrollment in its Phase 3 Aspire study with data expected in the second half of 2026. Additional programs in development include DTX301 for Ornithine Transcarbamylase deficiency, UX701 for Wilson disease, and UX143 for Osteogenesis Imperfecta.

Ultragenyx employs a strategy focused on in-licensing promising candidates from academic institutions and pharmaceutical partners while retaining global commercialization rights. The company maintains commercial operations across North America, the European Union, United Kingdom, Latin America, Turkey, and select Asian markets. Founded in April 2010, Ultragenyx has established itself as a specialized rare disease-focused biopharmaceutical company with expertise in patient identification, clinical study design, and regulatory strategy across multiple development programs conducted in parallel.