Ultragenyx is a privately held, developmental stage biotechnology company committed to bringing life-enhancing therapeutics for patients with rare and ultra-rare genetic diseases, also known as orphan diseases, to market. The company focuses on metabolic and rare diseases that may affect small numbers of patients, but for which the medical need is high and there are no effective treatments. Ultragenyx intends to build a sustainable pipeline of safe and effective therapies to address these clinically underserved diseases.
We select target diseases that have well understood biological mechanisms and potent therapeutic activity. We are focused on programs that are ready for development, and not early stage research. Ultragenyx’ lead program, UX001, is being evaluated as a potential treatment for GNE myopathy; also know as hereditary inclusion body myopathy (HIBM).
Our company is led by an experienced management team in rare disease therapeutics. Recognizing that our primary responsibility is to our patients, we are working with advocacy groups to provide support and outreach to individuals and families affected by these disorders and engage them in the clinical testing process. We are also working with regulatory agencies to design and conduct high quality clinical studies that meet the requirements for approval.
We are creating an improved model for successful rare disease drug development that increases efficiency and effectiveness by changing the way the process is organized and conducted. We believe that we can deliver significant value to our patients by building a diverse and high quality pipeline of rare disease therapeutics and efficiently transforming good science into great medicine.