Top 50 Thought-Leaders in Orphan Drugs and Rare Disease: New Report

December 8, 2013


This report, created by Terrapinn and the World Orphan Drug Conference recognizes the top 50 thought-leaders in orphan drugs and rare disease.

Chris Austin, Director, National Center for Advancing Translational Sciences, NIH

In September 2012, Christopher P. Austin, M.D., was appointed the first permanent director of the National Center for Advancing Translational Sciences (NCATS). Austin is leading NCATS in its mission to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions.

Currently, many costly, time-consuming bottlenecks exist in the translational process. Austin is applying his experience across the spectrum of the research pipeline to develop, demonstrate, and disseminate innovative technologies and paradigms that increase the efficiency of translation, and thus speed the discovery and delivery of interventions that improve human health.

Austin came to NIH in 2002 from Merck, where his work focused on genome-based discovery of novel targets and drugs. He began his NIH career as the senior advisor to the director for translational research at the National Human Genome Research Institute, where he initiated the Knockout Mouse Project and the Molecular Libraries Roadmap Initiative. Other NIH roles have included serving as director of the Therapeutics for Rare and Neglected Diseases program as well as the NIH Chemical Genomics Center and as scientific director of the NIH Center for Translational Therapeutics.

Austin earned an A.B. summa cum laude in biology from Princeton University and an M.D. from Harvard Medical School. He completed clinical training in internal medicine and neurology at Massachusetts General Hospital, and a research fellowship in genetics at Harvard.

Why we chose Dr. Austin:

As the first Director of NIH/NCATS, and having previously served as the Director for the agency’s TRND program which bridges the gap between basic research discovery and testing of new drugs in humans, Dr. Austin has led multiple initiatives to expedite orphan drugs to patients. The division has recently announced its first Federal Register notice concerning a novel selective series of non-inhibitory chaperones of glucocerebrosidase (GCase) for the treatment of Gaucher disease.

Want to read the full report to see the other 49 individuals chosen? You can download a copy of the report here:

The thought-leaders include representatives from industry, patient advocacy groups, research institutions, government, and media from around the world. They were selected based on the following criteria:

  • Research & methodology
  • Leadership
  • Awards & recognition
  • Community engagement & impact

Download a copy of the report today to find out who is pioneering orphan drug research, awareness, and legislation.

You may also be interested in participating at World Orphan Drug Congress USA on April 23-25, 2014. World Orphan Drug Congress USA is the largest international, commercially-focused event for the advancement of rare disease research and orphan drug development.

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