Ultragenyx Pharmaceutical Inc. (RARE)

Overview

Ultragenyx Pharmaceutical Inc. is a pioneering biopharmaceutical firm dedicated to the discovery, acquisition, development, and commercialization of innovative products for the diagnosis and treatment of rare and ultra-rare genetic disorders across North America, Latin America, Japan, Europe, and globally. Founded in 2010 and based in Novato, California, Ultragenyx operates at the forefront of medical research into genetic diseases, collaborating with esteemed institutions and companies such as Kyowa Kirin Co., Ltd., Saint Louis University, Baylor Research Institute, REGENXBIO Inc., and others to advance its mission. This fruitful collaboration ensures a robust pipeline of therapeutic candidates designed to address some of the most challenging and underserved medical needs.

Products and Services

The innovation at Ultragenyx spans several biologic products targeting a variety of rare genetic diseases, alongside a diverse pipeline of product candidates in various stages of development:

• Crysvita (burosumab) - This antibody targets fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia and tumor-induced osteomalacia, offering new hope to patients suffering from these rare bone disorders.
• Mepsevii - An enzyme replacement therapy designed for children and adults with Mucopolysaccharidosis VII, Mepsevii represents a critical advancement in treating this lysosomal storage disorder.
• Dojolvi - Tailored for individuals with long-chain fatty acid oxidation disorders, Dojolvi provides a novel dietary management solution to support those affected by this metabolic condition.
• Evkeeza (evinacumab) - For patients grappling with homozygous familial hypercholesterolemia, Evkeeza offers a groundbreaking monoclonal antibody treatment aimed at reducing LDL cholesterol levels.
• DTX401 - This adeno-associated virus 8 (AAV8) gene therapy clinical candidate aims to cure glycogen storage disease type Ia, demonstrating Ultragenyx's commitment to gene therapy solutions.
• DTX301 - Another AAV8 gene therapy, designed for patients with ornithine transcarbamylase deficiency, showcases the company's innovative approach to genetic metabolic disorders.
• UX143 - A human monoclonal antibody in development for the treatment of osteogenesis imperfecta, illustrating Ultragenyx's focus on genetic disorders affecting bone density and growth.
• GTX-102 - An antisense oligonucleotide aimed at treating Angelman syndrome, this prospective therapy offers hope for a condition currently lacking specific treatments.
• UX111 - An AAV9 gene therapy product candidate for Sanfilippo syndrome type A, or MPS IIIA, represents a potential breakthrough for this rare lysosomal storage disease.
• UX701 - Focused on Wilson disease, UX701 is a gene therapy candidate that could offer a significant improvement over current treatment options for this genetic disorder affecting copper metabolism.
• UX053 - For patients with glycogen storage disease type III, UX053 represents another critical gene therapy effort by Ultragenyx to address unmet medical needs in metabolic disorders.