UNC Catalyst initiative aims to create, share tools to fight rare diseases

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UNC Catalyst initiative aims to create, share tools to fight rare diseases


Eshelman Institute for Innovation commits $2 million to pharmacy school effort to create, share tools to jump start studying rare diseases typically ignored in research funding


(Chapel Hill, N.C. — Jan. 5, 2017) — Freely giving researchers the tools and knowledge to tackle rare and orphaned diseases is the mission of UNC Catalyst, a new endeavor the University of North Carolina at Chapel Hill has launched with a $2 million grant from the Eshelman Institute for Innovation. UNC Catalyst will provide patient groups and rare-disease organizations with the knowledge and research tools to train scientists to create new treatments.


“Science has cracked the human genome, but translating that knowledge into new medicines has been painfully slow,” said Bob Blouin, director of the Eshelman institute and dean of the Eshelman School of Pharmacy. “This is especially true for rare diseases, which suffer from a lack of visibility, resources and research expertise. UNC Catalyst will create and freely share the tools and the basic expertise currently missing in the study of many rare conditions.”


According to the National Institutes of Health, a rare or orphan disease in the U.S. affects fewer than 200,000 people. There are more than 6,800 rare diseases. Many are genetic, often caused by a single-gene mutation and include conditions such as cystic fibrosis, Huntington’s disease and muscular dystrophy.


Over the past decade, the DNA mutations associated with many rare diseases have been identified, but there has been little success moving from knowledge of the gene to a treatment. Other factors include a lack of high-quality research tools available for these diseases and too few researchers trained to work in the field.


UNC Catalyst will partner with the international Structural Genomics Consortium and rare disease groups, such as the Genetic Alliance, to recruit, train and fund research scientists. These scientists will create tools needed to study the physical effects these genetic mutations have on the body and create a framework for designing a new treatment. To magnify and accelerate the impact of this initiative, researchers across the globe will have unrestricted access to the research tools generated by UNC Catalyst.


“The hundreds of rare disease advocacy organizations in Genetic Alliance’s network will benefit greatly from this partnership,” said Sharon Terry, president and CEO of Genetic Alliance. “We have long worked for an open-science scalable approach to build research tools and support the necessary talent to accelerate solutions to ultimately ameliorate suffering in the millions of individuals affected by rare diseases. This answers that need, and we are delighted to work with these partners.”


Working in partnership with the Structural Genomics Consortium and Genetic Alliance, the UNC Catalyst for Rare Diseases will create a dedicated laboratory and data hub at UNC-Chapel Hill.




About the University of North Carolina at Chapel Hill

The University of North Carolina at Chapel Hill, the nation’s first public university, is a global higher education leader known for innovative teaching, research and public service. A member of the prestigious Association of American Universities, Carolina regularly ranks as the best value for academic quality in U.S. public higher education. Now in its third century, the University offers 77 bachelor’s, 110 master’s, 64 doctorate and seven professional degree programs through 14 schools and the College of Arts and Sciences. Every day, faculty – including two Nobel laureates – staff and students shape their teaching, research and public service to meet North Carolina’s most pressing needs in every region and all 100 counties. Carolina’s 317,000-plus alumni live in all 50 states and 156 other countries. More than 167,000 live in North Carolina.


About the Eshelman Institute

The Eshelman Institute for Innovation provides a mechanism for faculty, staff and students at the UNC Eshelman School of Pharmacy to seek funding for bold, transformative ideas and also provides opportunities to educate and train students and postdoctoral fellows; foster collaboration, creativity and innovation; and stimulate commercialization of intellectual property and entrepreneurial development. The institute was created by a $100 million gift from school alumnus Fred Eshelman made in December 2014.


About the UNC Eshelman School of Pharmacy

The UNC Eshelman School of Pharmacy at UNC-Chapel Hill is one of the world’s leading schools of pharmacy and pharmaceutical sciences. The school is ranked number one by U.S. News & World Report and ranks second in total research funding among the nation’s pharmacy schools. The school’s research enterprise has spawned 20 companies in the past 20 years.


About Genetic Alliance

Genetic Alliance engages individuals, families and communities to transform health. Founded in 1986, it is the world’s largest nonprofit health advocacy organization network. Genetic Alliance’s network includes more than 1,200 disease-specific advocacy organizations, as well as thousands of universities, private companies, government agencies and public policy organizations. For more information about Genetic Alliance, visit www.geneticalliance.org.


About the Structural Genomics Consortium

The SGC is a non-profit precompetitive public-private partnership that accelerates research in human biology and drug discovery by making all of its research output freely available to the scientific community. To achieve its mission, the organization is building an open and collaborative network of scientists: the SGC has active research facilities at six leading academic institutions across the globe including UNC-Chapel Hill. SGC scientists collaborate with more than 300 researchers in academia and industry. For more information, visit www.thesgc.org.


Communications and Public Affairs contact: Thania Benios, (919) 962-8596, thania_benios@unc.edu

Eshelman School of Pharmacy contact: David Etchison, (919) 966-7744, david_etchison@unc.edu