The University of Pittsburgh Partners with Major BioPharm Co. on Rare Diseases

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To advance treatments for rare diseases with significant unmet need, Pitt is collaborating with biopharmaceutical company Shire.

This 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.

The University of Pittsburgh today announced a new research collaboration with global biopharmaceutical company Shire plc (LSE: SHP, NASDAQ: SHPG) designed to advance potential treatments for rare diseases, where sizable unmet need exists.

The collaboration will combine the expertise of Pitt’s top-ranked health sciences schools with Shire’s research, development and commercialization experience.

“We’re very excited to enter into a partnership with Shire Pharmaceuticals,” said Patrick D. Gallagher, Ph.D., chancellor of Pitt. “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.”

Pitt has an extensive legacy of delivering groundbreaking health solutions, such as creating the first safe, 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 offers an enormous enterprise consisting of thousands of scientists who have made significant contributions to biomedical research.

“Shire is committed to enabling people with life-altering conditions to lead better lives,” said Philip J. Vickers, Ph.D., head of research and development at Shire. “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.”

“This 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,” said Arthur S. Levine, M.D., Pitt’s senior vice chancellor for the health sciences and John and Gertrude Petersen Dean of Medicine. “We can and very much want to make a difference for these families and are glad to have an opportunity to do so.”

This kind of partnership is relatively new for Pitt but is the type of relationship the university has identified 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.”

According to patient advocacy organization Global Genes, rare diseases affect more than 320 million people worldwide – 10 times the number affected by all cancers combined and approximately the same number 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, cause significant suffering and very often result in death early in life. While each individual disease generally affects fewer than several hundred thousand people, collectively they account for a massive global burden of underserved patients.

“Our scientific teams have been among the most prolific in determining the fundamental causes of rare diseases over the past decades,” said Dietrich Stephan, Ph.D., 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.”

Examples of diseases that Pitt scientists already have been investigating independent of the collaboration with Shire include cutis laxa, Huntington’s disease, demyelinating brain disorders and vascular malformations, such as hereditary hemorrhagic telangiectasia.

“Given the scale of resources and level of expertise within this partnership, I am hopeful many new life-changing therapies will be developed,” said Dr. Stephan. “Only the creativity of our scientists will limit the number of new drugs we can potentially deliver.”

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