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:

  • Increase awareness of rare diseases and their impact
  • Revive promising therapies and encourage research to uncover new ones
  • Promote drug development and commercialization
  • Train a workforce prepared to find solutions for treating rare diseases

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.

Advocating for Treatments

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:

  • Promotes the search for new therapies and helps to identify those with the most promise
  • Reaches out to drug companies, government agencies, research institutions, and patient advocacy groups to encourage them to communicate with one another and work together to combat rare diseases
  • Assesses the potential usefulness of research and orphan drugs that companies didn’t pursue or make available for sale
  • Works with patient advocacy groups to document the prevalence of rare diseases and devise strategies to promote the development and commercialization of treatments
  • Helps to prepare and file orphan drug designations
  • Heightens awareness of rare diseases to gain broad public support for the development of new, more affordable therapies

Educating Students and Others About Rare Diseases

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:

  • Provides summer internships for students with patient advocacy groups, pharmaceutical and biotech companies, nonprofit organizations and government agencies such as the U.S. Food & Drug Administration’s Office of Orphan Products Development (OOPD)
  • Teaches students about all aspects of rare diseases, including the market size, orphan drug discovery, the orphan drug application process, regulation and public policy
  • Develops case studies on rare diseases that bring together science, business and ethics
  • Presents workshops and symposia
  • Disseminates key findings and other information through conferences and papers on topics such as the economics of rare diseases and strategies to address cost issues

Tackling Rare Diseases Through Science

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.