University Forms Research Partnership with Shire

The University of Pittsburgh has agreed to a research collaboration with Shire PLC, a global biopharmaceutical company, to advance potential treatments for rare diseases. 

The collaboration will combine the expertise of Pitt’s top-ranked health sciences schools with Shire’s research, development, and commercialization experience. Shire, which has headquarters in Lexington, Mass., and Dublin, Ireland, works to discover and provide therapies to patients with rare diseases and other specialty conditions. 

“We’re very excited to enter into a partnership with Shire Pharmaceuticals,” said Chancellor Patrick Gallagher. “For decades, Pitt has been a leading research university, and as we move into our next phase, finding external partnerships with companies like Shire is critical for our future and a priority for Pitt.”

The University has identified the formation of such partnerships as a strategic priority. Under the agreement, Pitt and Shire will issue joint request for proposals to University scientists. Selected projects will be funded by Shire and could ultimately result in a licensing agreement.

“Public funding for higher education is changing,” Chancellor Gallagher added. “For Pitt to continue to engage in research of impact, including research into rare diseases, we must seek out external partnerships.”

Arthur S. Levine, Pitt’s John and Gertrude Petersen Dean of Medicine and senior vice chancellor for the health sciences, said “the collaboration has enormous potential to take what our scientists have already learned—and will discover—about rare illnesses and apply this new knowledge to improve the care of patients who have otherwise garnered little attention. We can and very much want to make a difference for these families and are glad to have an opportunity to do so.”

Rare diseases affect more than 320 million people worldwide, according to patient advocacy organization Global Genes. That is 10 times the number of people affected by all cancers combined and about the same number of those who suffer from the global epidemic of diabetes. Scientists have identified thousands of rare diseases, often having origins in genetic mutations that can be passed from one generation to the next. These diseases are usually extremely severe, causing significant suffering, and very often resulting in death early in life. While each individual disease generally affects fewer than several hundred thousand people, collectively the rare diseases account for a massive global burden of underserved patients.

Examples of such diseases that Pitt scientists already have been investigating—independent of the collaboration with Shire—include cutis laxa, a connective-tissue disorder characterized by wrinkled and sagging inelastic skin; Huntington’s disease, a neurodegenerative genetic disorder that affects muscle coordination and leads to mental decline and behavioral symptoms; and demyelinating brain disorders and vascular malformations, such as hereditary hemorrhagic telangiectasia, a disorder that causes multiple abnormalities in the blood vessels.

Pitt has an extensive legacy of discovering groundbreaking health solutions, including creating the first safe and effective polio vaccine, pioneering organ transplantation, and developing imaging technology to identify the protein plaques characteristic of Alzheimer’s disease in the living brain. Ranked among the top five universities in National Institutes of Health research funding, Pitt comprises thousands of scientists who have made significant contributions to biomedical research.

“Our scientific teams have been among the most prolific in determining the fundamental causes of rare diseases over the past decades,” said Dietrich Stephan, who is leading the collaboration for Pitt and is professor and chair of the Department of Human Genetics at Pitt’s Graduate School of Public Health. “Based on these new insights into the core pathogenesis of rare diseases, we have, in many cases, developed new pre-clinical models of disease and lead compounds to fuel new drug development—the essential ingredients for this broad-based partnership in the rare disease area.”

Philip J. Vickers, head of research and development at Shire, said the company “is committed to enabling people with life-altering conditions to lead better lives. Collaboration is key to meeting this objective, and partnership with the University of Pittsburgh is an excellent step toward uncovering potential new avenues for treating patients.”