Скачать или смотреть Panel: Bespoke Therapies for Ultra-Rare Disease

This session will explore the National Institutes of Health (NIH)’s public-private partnership to help individualized gene therapies reach patients despite their lack of commercial viability. Panelists will provide an overview of this initiative including how it will maximize benefits to patients, as well as explore the specific opportunities and challenges related to its success.

Chair:
Yael Weiss, M.D., Ph.D., VP, Business Development, Ultragenyx

Speakers:
Philip J. Brooks, Ph.D., Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)
Barry Byrne, M.D., Ph.D., Professor and Associate Chair of Pediatrics; Director of the Powell Gene Therapy Center, University of Florida
Joseph Menetski, Ph.D., Associate VP of Research Partnerships, Foundation for the National Institutes of Health
Bob Smith, SVP, Global Gene Therapy Business, Pfizer Inc.