Ultragenyx Pharmaceutical Inc. (UP0)
Summary
UP0 Chart
RARE's Fayuvi Approval Spurs Second PRV Sale Worth $203M in a Week
Ultragenyx agrees to sell its Fayuvi-linked PRV for $203 million, marking its second voucher sale in one week and adding non-dilutive funding for rare-disease programs.
Can RARE's Approved Product Portfolio Drive Its Next Growth Phase?
Ultragenyx's established rare-disease treatments continue to support revenue growth, while two newly approved gene therapies offer additional commercial opportunities.
Ultragenyx to Sell Rare Pediatric Disease PRV for $210M to Boost Cash
RARE enters into an agreement to sell a Rare Pediatric Disease PRV for $210M, adding non-dilutive cash as it advances rare-disease therapies.
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Ultragenyx Pharmaceutical Inc. Profile
| Biotechnology Industry | Healthcare Sector | Emil D. Kakkis CEO | XSTU Exchange | 90400D108 CUSIP |
| US Country | 1,371 Employees | - Last Dividend | - Last Split | 31 Jan 2014 IPO Date |
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.
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