Appendix A
Workshop Agenda
Navigating the Manufacturing Process and Ensuring the Quality of
Regenerative Medicine Therapies:
A Workshop
June 26, 2017
National Academy of Sciences Building
Lecture Room
2101 Constitution Avenue NW
Washington, DC 20418
| 8:30 a.m. | Opening Remarks |
|
R. ALTA CHARO, Forum Co-Chair |
|
|
Warren P. Knowles Professor of Law |
|
|
University of Wisconsin–Madison |
|
|
JAY P. SIEGEL, Forum Co-Chair |
|
|
Chief Biotechnology Officer |
|
|
Head, Scientific Strategy and Policy |
|
|
Johnson & Johnson |
|
| 8:35 a.m. | Charge to Workshop Speakers and Participants |
|
CLAUDIA ZYLBERBERG, Workshop Co-Chair |
|
|
Founder and Chief Executive Officer |
|
|
Akron Biotech |
|
|
STEVEN OH, Workshop Co-Chair |
|
|
Acting Deputy Director, Division of Cellular and Gene Therapies |
|
|
Office of Tissues and Advanced Therapies |
|
|
Center for Biologics Evaluation and Research |
|
|
U.S. Food and Drug Administration |
| 8:45 a.m. | Opening Keynote |
|
ADRIAN GEE |
|
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Professor of Cell and Gene Therapy |
|
|
Baylor College of Medicine |
SESSION I: TRANSITIONING FROM DISCOVERY AND DEVELOPMENT TO MANUFACTURING FOR REGENERATIVE THERAPIES
Session Objectives:
- To discuss challenges and opportunities associated with bringing new discoveries from the lab to manufacturing and navigating the process of scaling up the manufacturing of new therapies.
- To learn about methods and capabilities for manufacturing and quality control data collection for the purpose of informing the transition from research and development (R&D) to the implementation of good manufacturing practices (GMPs).
- To illuminate potential opportunities and models to reach scale and commercialization with current infrastructure and to assess probable future needs.
| Moderator: | Krishanu Saha, Assistant Professor, University of Wisconsin–Madison |
| 9:10–9:55 a.m. | Speakers: |
|
BRUCE LEVINE |
|
|
Barbara and Edward Netter Professor in Cancer Gene Therapy |
|
|
University of Pennsylvania Perelman School of Medicine |
|
|
LAURA NIKLASON |
|
|
Professor of Anesthesiology and Biomedical Engineering |
|
|
Yale University |
|
|
BOB PRETI |
|
|
President and Chief Executive Officer |
|
|
PCT Cell Therapy Services, LLC |
| 9:55–10:30 a.m. | Discussion with Workshop Participants |
| 10:30–10:45 a.m. | BREAK |
SESSION II: IDENTIFYING AND MEASURING CRITICAL QUALITY ATTRIBUTES OF REGENERATIVE MEDICINE PRODUCTS AND SOURCE CELLS
Session Objectives:
- To examine methods and processes used to identify and measure critical quality attributes for raw materials and regenerative medicine products.
- To discuss measurement methodology and how to ensure that measurements are accurate and reproducible.
- To survey technologies and future needs in the measurement of critical quality attributes.
| Moderator: | Martha Somerman, Director, National Institute of Dental and Craniofacial Research, National Institutes of Health |
| 10:45–11:30 a.m. | Speakers: |
|
ANNE PLANT |
|
|
Chief of the Biosystems and Biomaterials Division |
|
|
National Institute of Standards and Technology |
|
|
LINDA KELLEY |
|
|
Director, Cell Therapies Processing Facility |
|
|
Moffitt Cancer Center |
|
|
ROBERT DEANS |
|
|
Chief Technology Officer |
|
|
BlueRock Therapeutics |
|
| 11:30 a.m.–noon | Discussion with Workshop Participants |
| Noon–1:00 p.m. | WORKING LUNCH |
SESSION III: DESIGNING TECHNOLOGIES TO MEET THE MANUFACTURING NEEDS OF NEW REGENERATIVE THERAPIES
Session Objectives:
- To explore existing technologies that facilitate the efficient and cost-effective development of products that meet manufacturing and regulatory standards.
- To illuminate opportunities for new technologies and manufacturing models to increase efficiency and quality.
- To discuss novel and more precise in-process and final release testing technologies.
- To review existing infrastructure such as GMP facilities in academic centers and the commercial sector.
- To understand the open and closed systems models applicable to the manufacturing setting and information technology support.
| Moderator: | Thomas Petersen, Vice President, Regenerative Medicine, United Therapeutics Corporation |
| 1:00–1:45 p.m. | Speakers: |
|
ISABELLE RIVIÈRE |
|
|
Director, Cell Therapy and Cell Engineering Facility |
|
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Memorial Sloan Kettering Cancer Center |
|
|
RODNEY RIETZE |
|
|
Lead, cGMP Process Automation for Cell and Gene Therapies |
|
|
Novartis |
|
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PHILIP VANEK |
|
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General Manager, Cell Therapy Technologies |
|
|
GE Healthcare |
|
| 1:45–2:15 p.m. | Discussion with Workshop Participants |
SESSION IV: REGULATORY CHALLENGES AND OPPORTUNITIES FOR REGENERATIVE MEDICINE THERAPIES
Session Objective:
- To consider the regulatory landscape for regenerative medicine, including
- developing standards
- enforcing regulation
- meeting the needs of patients
| Moderator: | Jiwen Zhang, Senior Director, Regulatory Affairs, Cell Therapy and Regenerative Medicine, GE Healthcare |
| 2:15–3:00 p.m. | Speakers: |
|
ROBERT MCBURNEY |
|
|
President and Chief Executive Officer |
|
|
Accelerated Cures Project for Multiple Sclerosis |
|
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Co-Principal Investigator |
|
|
iConquerMS Patient-Powered Research Network |
|
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STEVEN BAUER |
|
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Chief, Cellular and Tissue Therapy Branch |
|
|
Center for Biologics Evaluation and Research |
|
|
U.S. Food and Drug Administration |
|
|
KATHERINE TSOKAS |
|
|
Senior Director, Global Regulatory Affairs |
|
|
Johnson & Johnson |
|
| 3:00–3:35 p.m. | Discussion with Workshop Participants |
| 3:35–3:50 p.m. | BREAK |
SESSION V: CLOSING KEYNOTE AND PANEL
Session Objectives:
- To summarize the lessons learned and topics discussed throughout the workshop day.
- To discuss ways forward to support the development, manufacture, and regulation of safe and effective regenerative medicine therapies.
| Moderator: | Krishnendu Roy, Robert A. Milton Chair and Professor and Technical Lead, National Cell Manufacturing Consortium, Georgia Institute of Technology |
| 3:50–4:10 p.m. | Closing Keynote: |
|
DEAN KAMEN |
|
|
Advanced Regenerative Manufacturing Institute |
|
|
DEKA Research & Development Corporation |
|
| 4:10–4:30 p.m. | Panelist Reflections |
|
BOB PRETI |
|
|
President and Chief Executive Officer |
|
|
PCT Cell Therapy Services, LLC |
|
|
ANNE PLANT |
|
|
Chief of the Biosystems and Biomaterials Division |
|
|
National Institute of Standards and Technology |
|
|
PHILIP VANEK |
|
|
General Manager, Cell Therapy Technologies |
|
|
GE Healthcare |
|
|
ROBERT MCBURNEY |
|
|
President and Chief Executive Officer |
|
|
Accelerated Cures Project for Multiple Sclerosis |
|
|
Co-Principal Investigator |
|
|
iConquerMS Patient-Powered Research Network |
|
| 4:30–5:00 p.m. | Discussion with Workshop Participants |
| 5:00 p.m. | Final Remarks from Workshop Co-Chairs |
|
CLAUDIA ZYLBERBERG, Workshop Co-Chair |
|
|
Founder and Chief Executive Officer |
|
|
Akron Biotech |
|
|
STEVEN OH, Workshop Co-Chair |
|
|
Acting Deputy Director, Division of Cellular and Gene Therapies |
|
|
Office of Tissues and Advanced Therapies |
|
|
Center for Biologics Evaluation and Research |
|
|
U.S. Food and Drug Administration |
|
| 5:15 p.m. | Adjourn |
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