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Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
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A

Workshop Agenda

The State of the Science in the Field of Regenerative Medicine:
Challenges of and Opportunities for Cellular Therapies: A
Workshop

October 13, 2016

National Academy of Sciences Building
Room 125
2101 Constitution Avenue, NW
Washington, DC 20418

MEETING OBJECTIVES
  • To examine the state of the science for therapies that generate, repair, or replace tissues by convening scientists, clinicians, industry, patient experts, and other stakeholders.
  • To highlight the challenges, successes, and lessons learned with respect to translation of regenerative therapies from early discovery into clinical practice with the goal of reaching patients.
  • To illuminate next steps for the field and ways that the forum could be a facilitator of progress.
AGENDA
8:30 a.m. Welcoming Remarks
R. Alta Charo, Forum Co-Chair
Warren P. Knowles Professor of Law and Bioethics
School of Law and School of Medicine and Public Health
University of Wisconsin–Madison
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
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Jay Siegel, Forum Co-Chair
Chief Biotechnology Officer
Head, Scientific Strategy and Policy
Johnson & Johnson
INTRODUCTION: OVERVIEW OF THE FIELD OF REGENERATIVE MEDICINE AND FOCUS OF THE WORKSHOP

Objectives: To describe and explore the broad field of regenerative therapies at a high level and to highlight successful research and clinical applications as well as barriers to scientific and therapeutic advances as the field moves forward.

8:35 a.m. Charge to Workshop Speakers and Participants: Considering the State of the Science in Regenerative Therapies
Cynthia Dunbar, Workshop Co-Chair
President
American Society of Gene and Cell Therapy
Senior Investigator, Molecular Hematopoiesis Section
National Heart, Lung, and Blood Institute
8:45 a.m. Areas of Challenge and Success in Regenerative Therapies
Lorenz Studer
Director, Center for Stem Cell Biology
Memorial Sloan Kettering Cancer Center
9:05 a.m. Clarifying Questions
SESSION I: SKIN AND MUSCULOSKELETAL TISSUES

Objectives: To examine the state of the science in research and novel applications of new technology to repair, regenerate, or renew skin or musculoskeletal tissues; to consider the obstacles that hinder progress in research; and to

Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×

highlight scientific successes, lessons learned, and opportunities to move the field forward with the goal of bringing new therapies to patients.

Moderator: Audrey Kusiak, Scientific Program Manager, Rehabilitation Research and Development Service, Office of Research and Development, Department of Veterans Affairs

9:15 a.m. Anthony Oro
Professor of Dermatology
Member of Program in Epithelial Biology, Institute for Stem Cell Biology and Regenerative Medicine
Stanford University
Anthony Ratcliffe
President and Chief Executive Officer
Synthasome, Inc.
Patricia Furlong
Chief Executive Officer
Parent Project Muscular Dystrophy
10:00 a.m. Discussion with Speakers and Attendees
10:30 a.m. Break
SESSION II: HEMATOLOGY AND IMMUNITY

Objectives: To examine the state of the science in research and novel applications of new technology to repair, regenerate, or renew tissue and function in the blood and the immune system; to consider the obstacles that hinder progress in research; and to highlight scientific successes, lessons learned, and opportunities to move the field forward with the goal of bringing new therapies to patients.

Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×

Moderator: Cynthia Dunbar, President, American Society of Gene and Cell Therapy; Senior Investigator, Molecular Hematopoiesis Section, National Heart, Lung, and Blood Institute

10:45 a.m. Fyodor Urnov
Associate Director
Altius Institute for Biomedical Sciences
Harry Malech
Chief, Laboratory of Host Defenses
Chief, Genetic Immunotherapy Section
National Institute of Allergy and Infectious Diseases
Michel Sadelain
Director, Center for Cell Engineering and Gene
Transfer and Gene Expression Laboratory
Memorial Sloan Kettering Cancer Center
11:30 a.m. Patient Perspective
Jennifer Fields
Patient Advocate
11:35 a.m. Discussion with Speakers and Attendees
12:00 p.m. Working Lunch
SESSION III: NEUROLOGICAL AND OPHTHALMOLOGICAL TISSUES

Objectives: To examine the state of the science in research and novel applications of new technology to repair, regenerate, or renew neurological and ophthalmological tissues; to consider the obstacles that hinder progress in research; and to highlight scientific successes, lessons learned, and opportunities to move the field forward with the goal of bringing new therapies to patients.

Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×

Moderator: Brian Fiske, Senior Vice President, Research Programs, The Michael J. Fox Foundation for Parkinson’s Research

1:00 p.m. Sally Temple
Principal Investigator and Scientific Director
Neural Stem Cell Institute
Ann Tsukamoto
Former Executive Vice President, Scientific and Strategic Alliances
StemCells, Inc.
Peter Coffey
Professor, Neuroscience Research Institute
University of California, Santa Barbara
1:45 p.m. Discussion with Speakers and Attendees
SESSION IV: CARDIOVASCULAR AND LUNG TISSUES

Objectives: To examine the state of the science in research and novel applications of new technology to repair, regenerate, or renew cardiovascular and lung tissues; to consider the obstacles that hinder progress in research; and to highlight scientific successes, lessons learned, and opportunities to move the field forward with the goal of bringing new therapies to patients.

Moderator: Jiwen Zhang, Senior Director, Regulatory Affairs, Cell Therapy and Regenerative Medicine, GE Healthcare

2:15 p.m. Deepak Srivastava
The Younger Family Director, Gladstone
Institute of Cardiovascular Disease; Director, Rodenberry Center for Stem Cell Biology and Medicine; Professor, University of California, San Francisco
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Eduardo Marbán
Director, Heart Institute
Professor of Medicine
Cedars-Sinai

Brigitte Gomperts
Associate Professor, Pulmonary and Pediatric Medicine
University of California, Los Angeles
3:00 p.m. Discussion with Speakers and Attendees
3:30 p.m. Break
SESSION V: RENAL TISSUES

Objectives: To examine the state of the science in research and novel applications of new technology in repairing or regenerating tissue in the kidney, to consider the obstacles that hinder progress in research; and to highlight scientific successes, lessons learned, and opportunities to move the field forward with the goal of bringing new therapies to patients.

Moderator: Deborah Hoshizaki, Program Director, Division of Kidney, Urologic, and Hematologic Diseases, National Institute of Diabetes and Digestive and Kidney Diseases

3:45 p.m. Ben Humphreys
Chief, Renal Diseases Division, Department of Medicine
Washington University School of Medicine
David Baron
Chief Scientific Officer
PKD Foundation
4:15 p.m. Discussion with Speakers and Attendees
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
SESSION VI: FINAL DISCUSSION AND NEXT STEPS

Objectives: To reflect on the current state of the science of regenerative therapies, explore the existing and potential scientific barriers to advancing the field of regenerative medicine, and to discuss strategies and lessons learned for facilitating efficient, effective, and translatable research.

Moderator: Alta Charo, Warren P. Knowles Professor of Law and Bioethics School of Law and School of Medicine and Public Health, University of Wisconsin–Madison

4:35 p.m. Looking Toward the Future: The Promise and Challenges of Regenerative Therapies
George Daley
Director, Stem Cell Transplantation Program
Boston Children’s Hospital and Dana–Farber Cancer Institute
Dean, Harvard Medical School
4:55 p.m. Reflections on the Day/Next Steps
Patricia Furlong
Chief Executive Officer
Parent Project Muscular Dystrophy
Deepak Srivastava
The Younger Family Director, Gladstone
Institute of Cardiovascular Disease; Director, Rodenberry Center for Stem Cell Biology and Medicine; Professor, University of California, San Francisco
Sally Temple
Principal Investigator and Scientific Director
Neural Stem Cell Institute
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Ann Tsukamoto
Former Executive Vice President, Scientific and Strategic Alliances
StemCells, Inc.
5:15 p.m. Discussion with Speakers and Attendees
5:35 p.m. Concluding Remarks
R. Alta Charo, Forum Co-Chair
Warren P. Knowles Professor of Law and Bioethics
School of Law and School of Medicine and Public Health
University of Wisconsin–Madison
Cynthia Dunbar, Workshop Co-Chair
President
American Society of Gene and Cell Therapy
Senior Investigator, Molecular Hematopoiesis Section
National Heart, Lung, and Blood Institute
5:45 p.m. ADJOURN
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 87
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 88
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 89
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 90
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 91
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 92
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 93
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 94
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Regenerative medicine holds the potential to create living, functional cells and tissues that can be used to repair or replace those that have suffered potentially irreparable damage due to disease, age, traumatic injury, or genetic and congenital defects. The field of regenerative medicine is broad and includes research and development components of gene and cell therapies, tissue engineering, and non-biologic constructs. Although regenerative medicine has the potential to improve health and deliver economic benefits, this relatively new field faces challenges to developing policies and procedures to support the development of novel therapies are both safe and effective.

In October 2016, the National Academies of Sciences, Engineering, and Medicine hosted a public workshop with the goal of developing a broad understanding of the opportunities and challenges associated with regenerative medicine cellular therapies and related technologies. Participants explored the state of the science of cell-based regenerative therapies within the larger context of patient care and policy. This publication summarizes the presentations and discussions from the workshop.

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