Traditional biologics
CRYSVITA® (burosumab-twza) is a traditional biologic approved in the U.S. for X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO)
MEPSEVII® (vestronidase alfa-vjbk) is the first and only enzyme replacement therapy approved in the U.S. for MPS VII
UX143 (setrusumab), an investigational, anti-sclerostin, fully human monoclonal antibody for the treatment of osteogenesis imperfecta
Small molecules
DOJOLVI® (triheptanoin) oral liquid, a substrate replacement therapy, is a small molecule drug approved in the U.S. for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD)
Gene therapy
For certain rare genetic diseases, gene therapy offers the potential for a durable treatment that could have a significant impact on patients’ lives. Our expertise in adeno-associated virus (AAV)—widely considered an ideal vector for gene therapy—has enabled us to develop one of the industry’s largest advanced clinical gene therapy portfolios.
- UX111, an investigational AAV9 gene therapy, for the treatment of Sanfilippo syndrome type A (MPS IIIA)
- DTX401, an investigational AAV8 gene therapy, for the treatment of glycogen storage disease type Ia (GSDIa)
- DTX301, an investigational AAV8 gene therapy, being evaluated in a Phase 3 clinical trial for the treatment of ornithine transcarbamylase (OTC) deficiency
- UX701, an investigational AAV9 gene therapy, being evaluated in a seamless Phase 1/2/3 study for the treatment of Wilson disease
We employ a variety of platforms and technologies across our gene therapy clinical programs, coupled with our HEK manufacturing platform that supports our most advanced clinical-stage gene therapy programs.
Our gene therapy manufacturing facility in Bedford, Massachusetts, provides us with flexibility and process control that enables us to broaden our future partnerships and advance our own gene therapy pipeline.
Nucleic acid therapy (ASO)
Nucleic acid (ASO) has unique properties that allow us to tackle diseases that are untreatable with other modalities. For certain diseases, nucleic acid is the best option to successfully develop a therapeutic. It can be delivered in a lipid nanoparticle (LNP) to express a functional protein.
- GTX-102, an investigational antisense oligonucleotide (ASO) therapy being evaluated in the Phase 3 Aspire study and in the Aurora study for the treatment of Angelman syndrome
Discover more about Ultragenyx:
Pipeline
See the progress we have made with our diverse portfolio of investigational therapies.
Explore our pipeline