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Appendix A Workshop Agenda Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: A Workshop October 18, 2018 National Academy of Sciences Building Lecture Room 2101 Constitution Avenue, NW Washington, DC 20418 8:30 a.m. Opening Remarks Jay Siegel, Forum Co-Chair Scientific Advisor Tycho Therapeutics, Inc. 8:35 a.m. Charge to Workshop Speakers and Participants Martha Lundberg, Workshop Co-Chair Program Director, Division of Cardiovascular Sciences Advanced Technologies and Surgery Branch National Heart, Lung, and Blood Institute National Institutes of Health 87 PREPUBLICATION COPYÂ Uncorrected Proofs â
88 EXPLORING VARIABILITY IN REGENERATIVE ENGINEERING PRODUCTS Kathy Tsokas, Workshop Co-Chair Regulatory Head of Regenerative Medicine and Advanced Therapy Johnson & Johnson 8:45 a.m. Stage SettingâThe Impact of Variability on Regenerative Engineering Products Guillermo Ameer Daniel Hale Williams Professor of Biomedical Engineering and Surgery Director, Center for Advanced Regenerative Engineering Northwestern University SESSION I: USING CASE STUDIES TO IDENTIFY THE SOURCES OF VARIABILITY ASSOCIATED WITH REGENERATIVE THERAPIES Session Objective: â¢ To gain a better understanding of the sources of variability associ- ated with regenerative engineering products through a series of case studies. Session Moderator: Cato Laurencin, University Professor, Director, Â nstitute I for Regenerative Engineering, University of Connecticut 9:05 a.m. Case Study 1: Variability in the Use of Mesenchymal Stem Cells for Treating Cardiomyopathy Ivonne Hernandez Schulman Professor of Clinical Medicine University of Miami Miller School of Medicine 9:20 a.m. Case Study 2: Sources of Variability in Preclinical and Clinical Research on Stem Cell Therapies for Amyotrophic Lateral Sclerosis Clive Svendsen Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine CedarsâSinai Medical Center PREPUBLICATION COPYÂ Uncorrected Proofs â
APPENDIX A 89 9:35 a.m. Case Study 3: Variability in the Development of Cellular Therapies David Stroncek Chief, Cell Processing Section Department of Transfusion Medicine National Institutes of Health Clinical Center 9:50 a.m. Panel Discussion with Speakers and Workshop Participants 10:20 a.m. Break SESSION II: CONSIDERING THE FACTORS THAT CONTRIBUTE TO PATIENT VARIABILITY AND APPROACHES TO ADDRESSING THOSE DIFFERENCES Session Objectives: â¢ Discuss factors that contribute to patient variability such as a patientâs genetics, the severity of their condition, past treatments, the placebo effect, and the patientâs built environment/geography. â¢ Examine the feasibility of a precision medicine approach that would target the right patient with the right regenerative engineer- ing therapy. Session Moderator: Brian Fiske, Senior Vice President, Research Programs, The Michael J. Fox Foundation for Parkinsonâs Research 10:35 a.m. Jennifer Elisseeff Morton Goldberg Professor Wilmer Eye Institute and Biomedical Engineering, Translational Tissue Engineering Center Johns Hopkins University 10:50 a.m. Joseph Wu Director Stanford Cardiovascular Institute Simon H. Stertzer Professor of Cardiovascular Medicine and Radiology Stanford University School of Medicine 11:05 a.m. Steve Badylak Professor of Surgery McGowan Institute for Regenerative Medicine University of Pittsburgh PREPUBLICATION COPYÂ Uncorrected Proofs â
90 EXPLORING VARIABILITY IN REGENERATIVE ENGINEERING PRODUCTS 11:20 a.m. Flagg Flanagan Chief Executive Officer and Chairman of the Board Discgenics 11:35 a.m. Panel Discussion with Speakers and Audience Members 12:05 p.m. Working Lunch SESSION III: THE IMPORTANCE OF ADDRESSING VARIABILITY IN DONOR TISSUES AND CELLS Session Objectives: â¢ Consider the sources of variability among donor tissues and cells such as the source (e.g., bone marrow, adipose, cord blood), the dose, route of administration, and culture conditions, among other factors. â¢ Discuss methods to address the variability among source tissues and cells so that patients receive a consistent and effective product. Session Moderator: Martha Lundberg, Program Director, Division of C Â ardiovascular Sciences, Advanced Technologies and Surgery Branch, National Heart, Lung, and Blood Institute 1:00 p.m. Andrew Fesnak Assistant Professor of Clinical Pathology and Laboratory Medicine University of Pennsylvania Perelman School of Medicine 1:15 p.m. George Muschler Staff Member Department of Biomedical Engineering Cleveland Clinic 1:30 p.m. Allison Hubel Professor of Mechanical Engineering University of Minnesota 1:45 p.m. Panel Discussion with Speakers and Audience Members 2:15 p.m. Break PREPUBLICATION COPYÂ Uncorrected Proofs â
APPENDIX A 91 SESSION IV: THE IMPORTANCE OF ADDRESSING VARIABILITY AND MEETING QUALITY EXPECTATIONS IN THE MANUFACTURING SETTING Session Objectives: â¢ Explore the translational research priorities for the maturing of the fields of tissue science and regenerative engineering. â¢ Describe advances in preservation technologies needed to sustain fragile cells and tissues under biologically optimized conditions for storage, shipment and handling. â¢ Discuss metrics for reproducibility, robustness, and user-Â riendliness f that will enable the broad distribution of products. Session Moderator: Krish Roy, Robert A. Milton Endowed Chair and D Â irector, Center for ImmunoEngineering, Georgia Institute of Technology 2:30 p.m. Carl Burke BioTherapeutics Development Johnson & Johnson 2:45 p.m. Michele Myers Senior Director, Cell Process Development Cell and Gene Therapy Platform GlaxoSmithKline 3:00 p.m. Erik Finger Assistant Professor, Department of Surgery University of Minnesota 3:15 p.m. Panel Discussion with Speakers and Workshop Participants SESSION V: EXPLORING OBJECTIVE METRICS AND OUTCOMES FOR CLINICAL TRIALS AND THE REGULATORY APPROVAL PATHWAY Session Objectives: â¢ Discuss ideas for objective metrics and reliable approaches to inter- preting the outcomes of clinical trials of regenerative engineering therapies. â¢ Explore how variability in regenerative engineering products can affect the regulatory approval pathway. PREPUBLICATION COPYÂ Uncorrected Proofs â
92 EXPLORING VARIABILITY IN REGENERATIVE ENGINEERING PRODUCTS Session Moderator: Kathy Tsokas, Regulatory Head of Regenerative Medi- cine and Advanced Therapy, Johnson & Johnson 3:45 p.m. Karen Christman Scientific Co-Founder Ventrix 4:00 p.m. Peter Marks Director Center for Biologics Evaluation and Research U.S. Food and Drug Administration 4:15 p.m. Panel Discussion with Speakers and Workshop Participants 4:35 p.m. Final Panel Discussion Carl Burke Karen Christman Allison Hubel Peter Marks Clive Svendsen 5:00 p.m. Final Remarks from Workshop Co-Chairs Martha Lundberg, Workshop Co-Chair Program Director, Division of Cardiovascular Sciences Advanced Technologies and Surgery Branch National Heart, Lung, and Blood Institute National Institutes of Health Kathy Tsokas, Workshop Co-Chair Regulatory Head of Regenerative Medicine and Advanced Therapy Johnson & Johnson 5:10 p.m. Adjourn PREPUBLICATION COPYÂ Uncorrected Proofs â