The Rare and Orphan Disease Center

Working with foundations and their associated team of basic and clinical researchers to generate custom mouse models with clinical mutations to treat and cure Rare and Orphan Disease.

The Rare and Orphan Disease Center at The Jackson Laboratory

The Rare Disease Community needs an infrastructure that that moves them through the diagnostic phase into actionable therapeutics. Preclinical mouse models that precisely model the patient mutations and clinical symptoms, inform us of the effectiveness of new therapies and a fast-track their path to the clinic. The Jackson Laboratory understands the sense of urgency and the financial burden facing the families…. and we are here to help.

By definition, a rare and orphan disease is one that affects fewer than 200,000 individuals in the United States. Because of the small population afflicted by any one illness, funding to investigate causes and treatments tends to be limited, slowing the discovery of potential therapies. Yet with over 7,000 recognized rare diseases, an estimated 350 million people worldwide are affected at any given time.

The Rare and Orphan Disease Center brings together global mouse resources and the expertise of scientists at the Jackson Laboratory for a start-to-finish approach to model development.  A cure for any disease will not happen without concerted effort from people around the world. We partner with foundations, pharmaceutical and biotech companies, and other scientists worldwide to facilitate research into treatments of these less common diseases.