Track B

B8 It Takes a Global Village: An Orphan Drug Development Case Study

Track: Licensing Successful Practices

Target Audience: All Audiences

Moderator
Claire Driscoll, National Human Genome Research Institute (NHGRI); National Institutes of Health (NIH)

Speakers
Marjan Huizing, National Human Genome Research Institute (NHGRI); National Institutes of Health (NIH)
Hootan Khatami, Escala Therapeutics

Uncovering the genetic basis of various rare diseases and studying the complex biology that underlie these disorders is a focus of the in-house research program at the National Human Genome Research Institute (NHGRI) at National Institutes of Health (NIH). What was once an esoteric field of study that attracted few researchers, few dollars and little commercial interest has evolved into one of the hottest niches of the biotechnology/biopharma sector. This field is also embroiled in controversy, a consequence of the sometimes exorbitant prices charged for drugs which treat small numbers of patients. Even so, companies of all sizes have rare disease drug development projects in their pipelines, and these companies are aggressively scouting for new candidate drugs within government and academic labs. In this session, we will present a case study illustrating how a project can be successfully put together by academic physician-scientists and university technology transfer offices then licensed for commercial development. You’ll hear details how the various scientific, regulatory, intellectual property/licensing, clinical, patient and commercial aspects came together over the course of 15 years in an ongoing quest to develop the first drug to treat GNE myopathy, a rare neuromuscular disease.
 

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