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) is 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 gene therapy portfolios.
We have a robust clinical pipeline of active gene therapy programs, as well as some undisclosed programs in early research:
- DTX401, an AAV8 gene therapy, advancing to a Phase 3 clinical trial for the treatment of glycogen storage disease type Ia (GSDla)
- DTX301, an AAV8 gene therapy, advancing to a Phase 3 clinical trial for the treatment of ornithine transcarbamylase (OTC) deficiency
- UX701, an AAV9 gene therapy, in a seamless Phase 1/2/3 study for the treatment of Wilson disease
With our clonal producer cell line (PCL) technology, we have established a new manufacturing system that can produce AAV vectors with increased efficiency and lower material costs than traditional methods, and at a scale that can help meet the rising demands of today’s gene therapy landscape. We are building a gene therapy manufacturing facility in Bedford, Massachusetts, to provide us with a flexibility and control of process that can further optimize the clonal PCL platform, ultimately broadening our future partnerships and advancing our own gene therapy pipeline.
Nucleic acid therapy (ASO/mRNA)
Nucleic acid (ASO/mRNA) 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 is an investigational ASO therapy in development 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