UConn and Cheshire orphan-drug maker Alexion Pharmaceuticals Inc. are partnering to expand stem-cell research into combating rare diseases.
The research collaboration will focus on the discovery and testing of therapeutic candidates to treat rare and disabling disorders for which there are currently no effective treatments, both announced Thursday.
"This collaboration targets unmet medical needs for patients while demonstrating the vitality of the life science community in Connecticut," said Jeff Seemann, UConn's vice president for research.
It also fits with UConn's and the state's plan "to expand educational opportunities, research, and innovation in science, technology, engineering, and math (STEM) over the next decade," UConn President Susan Herbst said in a statement.
Alexion's highly profitable business model relies on devising treatments, like Soliris, to treat rare a rare form of anemia and other ailments for which mainstream drugs don't currently exist.
Their partnership likely will yield valuable data into diseases for which Alexion could develop new treatments.
The collaboration will expand on the work of cell biologist David J. Goldhamer, who is associate Director of the UConn Stem Cell Institute. Goldhamer has identified the offending progenitor cell type that drives the pathology of a group of diseases and has developed physiologically relevant disease models.
These models will be used to further understand the pathophysiology of these disorders and to test potential therapeutics, the partners said.