Emerging Technologies for Rare Diseases: Clinical and Regulatory Case Studies and Approval Pathways
Tuesday, September 12, 2017
Willard Intercontinental Hotel, Washington, DC
The rapid pace of innovation in the biotechnology industry has created a number of opportunities to bring cutting edge treatments to patients with rare diseases. However, some of these technologies are not well-understood by the public and have posed new challenges for the regulatory agencies charged with approving new treatments. Examples of specific technologies that could have broad applicability to rare disease patients include gene editing, gene therapy, epigenetic gene regulation, immunotherapy, RNAi, exon skipping and cell-based therapies. These technologies could be transformative and, in some cases, curative for rare disease patients, but must have viable regulatory and approval pathways to reach those in need of treatment.
The EveryLife Foundation for Rare Diseases convened our 9th annual Scientific Workshop to explore this topic. The goal of the workshop was to gather key thought-leaders from industry, Food and Drug Administration (FDA) and patient organizations to learn about the latest technologies. In addition, when possible, companies were invited to present case studies highlighting clinical and regulatory pathways. Senior leadership from FDA was invited to present an overview of resources and guidance available to industry and patient organizations. Our sponsors made it possible for us to open this event to the public free of charge.
Click here to view the agenda.
Welcome and EveryLife Foundation Introduction
Welcome Remarks, Max Bronstein, MPP, Senior Director, Health Policy and Corporate Affairs, Audentes Therapeutics
EveryLife Foundation Overview, Emil Kakkis, MD, PhD, President, EveryLife Foundation for Rare Diseases | Watch Presentation
The Curative and Transformative Potential of Novel Therapies for Rare Diseases in the Age of Precision Medicine, Christopher Austin, MD, Director, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH) | Watch Presentation
Unique Challenges of Clinical Development for Emerging Therapies
Sara Nochur, PhD, SVP Regulatory Affairs, Alnylam | Watch Presentation
Timothy Miller, PhD, CEO, Abeona Therapeutics | Watch Presentation
Nick Leschly, CEO, bluebird bio | Watch Presentation
Beyond the Clinic: Bringing Emerging Therapies to Market
John Gray, PhD, Senior Vice President and Chief Scientific Officer, Audentes Therapeutics | Watch Presentation
Gilmore O’Neill, Senior Vice President, Drug Innovation Units, Biogen | Watch Presentation
The Role of Patient Organizations in Advancing Emerging Therapies
Lori Sames, Co-Founder and CEO, Hannah’s Hope Fund | Watch Presentation
Next Generation Gene Editing
Harry Malech, MD, Chief, Laboratory of Host Defenses and the Genetic Immunotherapy Section National Institute of Allergy and Infectious Diseases, NIH | Watch Presentation
Gerry Cox, MD, PhD, Chief Medical Officer, Editas Medicine | Watch Presentation
Regulatory Pathways for Rare Diseases
Celia Witten, MD, PhD, Deputy Director, Center for Biologics Evaluation and Research (CBER), Food and Drug Administration (FDA) | Watch Presentation
Rich Moscicki, MD, Deputy Center Director for Science Operations, Center for Drug Evaluation and Research (CDER), FDA | Watch Presentation
Reimbursement, Public Perception, and Governance of Emerging Technologies
Richard Bankowitz, MD, MBA, Executive Vice President, Clinical Affairs, America’s Health Insurance Plans (AHIP) | Watch Presentation
Cary Funk, Associate Director of Research on Science and Society, Pew Research Center | Watch Presentation
Scientific Workshops
Thank you to our 2017 sponsors!
Please contact Carol Kennedy, Chief Development Officer, at ckennedy@rareadvocates.org for information on sponsorship opportunities.
