Unlike organizations that focus on a single disease, KGI’s Center for Rare Disease Therapies functions as a nonprofit think tank addressing the problems arising from all rare diseases. The Center partners with the pharmaceutical and biotech industry, government agencies, patient advocacy groups, research institutions, and nonprofit organizations to:
The Center for Rare Disease Therapies draws on KGI’s strengths, talents and distinctions. KGI’s extensive industry relationships, faculty with industry experience and classes focused on industry’s business, scientific and ethical challenges help the center perform its work and fulfill its mission.
The center ensures that rare diseases receive the attention that can lead to finding therapies for people who have no remedy available to them today. In advocating for more treatments, the center:
The Center for Rare Disease Therapies is an integral part of a KGI education. It ensures KGI students become professionals who understand rare diseases. It also enables those already working in the biotech industry and in research to learn more about rare diseases. Specifically, the center involves student teams in industry-sponsored, year-long projects with companies specializing in rare disease therapies
Teams of four to five students work with an industry liaison and a faculty mentor to complete their Team Master’s Project by examining the technical, regulatory, and business aspects of rare diseases. It also:
Being part of KGI enables the Center for Rare Disease Therapies to go beyond scientific research to also focus on how scientific discovery leads to a commercial product.
The center benefits from its relationship with KGI scientists whose research may be instrumental in finding new treatments for rare diseases. KGI faculty and researchers are examining promising gene therapies as well as biomarkers, the chemicals or proteins that signal the presence of disease. They are also actively engaged in drug discovery.By turning to scientific colleagues at KGI and other university research institutions, the Center for Rare Disease Therapies moves closer to its goal of developing a clinically approved therapy for a rare disease within the next three to five years.