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Appendix A Data Sources and Methods T he Committee on a Review of the California Institute for Regenera- tive Medicine (CIRM) was asked to assess CIRM’s initial processes, programmatic and scientific scope, organizational and management systems, funding model, and intellectual property policies. To respond com- prehensively to its charge, the committee examined data from a variety of sources. These sources included documents pertaining to the establishment and initial operation of CIRM and numerous evaluation reports commis- sioned during the Institute’s initial 5- to 6-year period of operation. Valu- able input also was obtained through CIRM’s responses to the committee’s data requests, three public meetings, two adjunct meetings, site visits to CIRM-funded research facilities, telephone interviews, and written public comments in response to online questionnaires targeting various stakehold- ers. The study was conducted over a 17-month period. STUDY COMMITTEE The study committee comprised 13 individuals with expertise in stem cell research, developmental biology, bioethics, research administration, financial structures for biomedical research, program evaluation, economics and finance, business administration, and intellectual property. Appendix E provides biographical sketches of the committee members. The committee convened for a total of 10 days on five different occasions in October 2011, January 2012, April 2012, June 2012, and August 2012. 131

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132 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE DOCUMENT REVIEW The committee reviewed background documents and evaluation reports pertaining to CIRM, including Proposition 71 and Senate Bill (SB) 1064; 2004 Economic Impact Analysis reports; the 2006 CIRM Scientific Strate- gic Plan; CIRM annual reports from 2007 through 2010; the 2008 Interim Economic Impact Review and addendum; the 2009 Strategic Plan Update; the 2009 Little Hoover Commission Review of CIRM; 2010 External Advisory Panel (EAP) evaluation reports; the 2011 CIRM publication on the economic impact of research funded by CIRM; and the 2012 CIRM transition plan. The committee also reviewed published reports on stem cell research. DATA REQUESTS TO CIRM The committee made a total of 79 requests for data, reports, and information from CIRM. These requests were on topics related to CIRM’s initial processes and planning, grants and programs, grant management and resources, reviews and evaluations, governance and management, finances, collaborations, intellectual property policies, and conflict of interest policies. PUBLIC MEETINGS The committee hosted three public meetings to obtain additional infor- mation on specific aspects of the study charge. These meetings were held in conjunction with the committee’s October (Washington, DC), January (San Francisco, California), and April (Irvine, California) meetings. The committee determined the topics and speakers for these public meetings. As part of the two public meetings in California, the committee held open forums at which members of the public were invited to provide testimony on any topics related to the study charge. At the first public meeting, CIRM delivered the charge to the commit- tee and provided a general overview of its structure and programs. At the second meeting, representatives from CIRM’s leadership and governing board provided information on the Institute’s scientific priorities and tran- sition plan, standards working group, and intellectual property policies. In addition, CIRM principal investigators provided their perspectives on the Institute. The third public meeting included presentations from individuals with varying perspectives on CIRM—investigators who applied for but did not receive funding from the Institute, current Disease Team grantees, industry representatives, and a technology transfer officer. The agendas for the public meetings are presented in Boxes A-1 through A-3.

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APPENDIX A 133 ADJUNCT MEETINGS A subgroup of the committee hosted two meetings with stem cell scien- tists outside of California to obtain their perspectives on the work funded by CIRM and the Institute’s scientific impact. Discussions centered on the scientists’ view of CIRM’s accomplishments and goals as articulated in the 2012 Strategic Plan. The first meeting was held May 30, 2012, in Toronto, Canada, and the second in Boston, Massachusetts, on June 26, 2012. These sites were selected as the committee considered them to be major hubs of activity in stem cell research. Scientists participating in these meeting are listed below. Toronto Mick Bhatia, Stem Cell and Cancer Research Institute, McMaster University (by phone) James Ellis, Department of Molecular Genetics, University of Toronto Andras Nagy, Samuel Lunenfeld Research Institute, Mount Sinai Hospital Janet Rossant, Department of Medical Genetics and Microbiology, University of Toronto Molly Shoichet, Department of Chemical Engineering and Applied Chemistry, University of Toronto (by phone) William Stanford, Department of Cellular and Molecular Medicine, University of Ottawa  Peter Zandstra, Institute of Biomaterials and Biomedical Engineering, University of Toronto Boston Fernando Camargo, Stem Cell and Regenerative Biology, Harvard University Konrad Hochedlinger, Stem Cell and Regenerative Biology, Harvard University Jerome Ritz, Connell O’Reilly Cell Manipulation and Gene Transfer Laboratory, Dana-Farber Cancer Institute, Harvard Medical School Anthony Rosenzweig, Beth Israel Deaconess Medical Center, Harvard Medical School David Scadden, Stem Cell and Regenerative Biology, Harvard University Ramesh Shivdasani, School of Medicine, and Dana-Farber Cancer Institute, Harvard Medical School Les Silberstein, Joint Program in Transfusion Medicine, Harvard Medical School Amy Wagers, Stem Cell and Regenerative Biology, Harvard University

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134 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE Clifford Woolf, FM Kirby Neurobiology Center, Children’s Hospital, Harvard Medical School Leonard Zon, Stem Cell and Regenerative Biology, Harvard University SITE VISITS Individual members of the committee conducted three site visits to CIRM-funded research facilities to obtain information about stem cell pro- grams funded by the Institute. The committee selected one private univer- sity, one medical school, and one public university to visit. In January 2012, committee members visited CIRM facilities at the University of California, Davis (UC Davis), the University of California, San Francisco (UCSF), and Stanford University. The visits included tours of the stem cell facilities and discussions with principal investigators about the CIRM-funded work being conducted at the institutions. Investigators and university leadership who participated in each site visit are listed below. In addition, in August 2012, the committee chair and vice chair visited Celgene, a biopharmaceutical company in Summit, New Jersey. The purpose of this visit was to help the committee better understand the process of discovery, development, and commercialization of products to treat disease. UC Davis Gerhard Bauer, Stem Cell Research Program, UC Davis Institute for Regenerative Cures (IRC) Paul Knoepfler, Department of Cell Biology and Human Anatomy, School of Medicine Kit Lam, Department of Biochemistry and Molecular Medicine, UC Davis Comprehensive Cancer Center Claire Pomeroy, CEO, UC Davis Health System and Dean of School of Medicine Mark Zern, Transplant Research Institute Min Zhao, Department of Dermatology, School of Medicine UCSF Arturo Alvarez-Bullya, Department of Neurological Surgery, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research Sam Hawgood, Dean of the UCSF School of Medicine and Vice Chancellor for Medical Affairs Diana Laird, Department of Obstetrics, Gynecology and Reproductive Sciences, UCSF Medical Center Daniel Lim, Department of Neurological Surgery, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research

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APPENDIX A 135 Emmanuelle Passegue, Division of Hematology/Oncology, Department of Medicine, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research Holger Willenbring, Department of Surgery, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research Stanford University Michael Longaker, Department of Surgery Ravi Majeti, Division of Hematology, School of Medicine Norma Neff, Research Technical Manager Renee A. Reijo-Pera, Center for Human Embryonic Stem Cell Research and Education Yuval Rinkevich, Institute for Stem Cell Biology and Regenerative Medicine Chris Shay, Space and Renovations Project Management Judy Shizuru, Department of Blood & Marrow Transplantation, School of Medicine Irving Weissman, Department of Pathology, Institute for Stem Cell Biology and Regenerative Medicine, School of Medicine Marius Wernig, Department of Pathology, Institute for Stem Cell Biology and Regenerative Medicine, School of Medicine Joanna Wysocka, Department of Chemical and System Biology INTERVIEWS Committee members spoke with many individuals during the course of this study. These formal and informal conversations, which took place by phone between the committee’s in-person meetings, were intended to gather information to inform the committee’s deliberations and to clarify questions. The individuals who provided this information are listed below. Cindy Bell, Genome Canada and former Director of Cancer Stem Cell Consortium Alan Bernstein, former Executive Director, Global HIV Vaccine Enterprise Genc Bülent, Bundesministerium für Bildung und Forschung (BMBF) (Federal Ministry of Education and Research), Germany George Daley, Stem Cell Transplantation at the Children’s Hospital Kristen Doyle, Cancer Prevention and Research Institute of Texas Bill Gimson, Cancer Prevention and Research Institute of Texas Dan Gincel, Maryland Stem Cell Research Fund Zach W. Hall, former president, CIRM Marianne Horn, Connecticut Department of Public Health

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136 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE Richard Klausner, The Column Group Bob Klein, former board chair, CIRM Bernard Lo, UCSF School of Medicine, CIRM Standards Working Group Jeff Lomax, CIRM Bert Lubin, Children’s Hospital and Research Center Foundation David Magnus, Stanford University School of Medicine Ed Penhoet, Alta Partners, former vice chair of CIRM’s Governing Board Mahendra S. Rao, National Institutes of Health (NIH) Intramural Center for Regenerative Medicine Beth Roxland, New York State Task Force on Life and the Law Christopher Scott, Stanford University School of Medicine Michael Stöcker, BMBF Lawrence S. Sturman, New York State Stem Cell Science Alan Trounson, CIRM BOX A-1 Committee on a Review of the California Institute for Regenerative Medicine (CIRM) The National Academies Keck Building 500 Fifth Street, NW Washington, DC 20001 Room 110 AGENDA FOR PUBLIC SESSION October 19, 2011 11:15 a.m. WELCOME AND COMMITTEE INTRODUCTIONS Harold T. Shapiro, Ph.D. OM Committee Chair 11:25 a.m. OVERVIEW OF CIRM: INITIAL PROCESSES, FUNDING MODEL, ORGANIZATION, AND MANAGEMENT SYSTEMS Robert Klein II, J.D. Chair Emeritus, CIRM Governing Board

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APPENDIX A 137 ONLINE QUESTIONNAIRES The committee used online questionnaires to obtain public input from a variety of stakeholders on topics related to the study charge. A total of seven questionnaires in different topic areas targeting different stakeholders were placed on the Institute of Medicine (IOM) website. These questionnaires were intended to help the committee understand perspectives of a variety of stakeholders on CIRM and its work. The questionnaires were constructed for CIRM Independent Citizens Oversight Committee (ICOC) members, leadership from CIRM-funded institutions, CIRM principal investigators, university technology transfer professionals, investigators who applied for but did not receive funding from CIRM, CIRM for-profit industry partners, CIRM international partners, and stakeholders and members of the general public. A summary of themes from the responses to the questionnaires can be found in Appendix B. 12:30 p.m. LUNCH 1:15 p.m. OVERVIEW OF THE SCIENCE AND CIRM’S PROGRAMMATIC AND SCIENTIFIC SCOPE Ellen G. Feigal, M.D. Senior Vice President, Research and Development, CIRM 2:00 p.m. INTELLECTUAL PROPERTY AND BUSINESS DEVELOPMENT Elona Baum, J.D. General Counsel and Vice President, Business Development, CIRM 2:20 p.m. CHARGE TO THE COMMITTEE Jonathan Thomas, J.D., D.Phil. Chair, CIRM Governing Board 2:35 p.m. QUESTIONS AND DISCUSSION OF STUDY CHARGE 3:30 p.m. ADJOURN OPEN SESSION

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138 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE BOX A-2 Committee on a Review of the California Institute for Regenerative Medicine (CIRM) South San Francisco Conference Center 255 South Airport Boulevard South San Francisco, CA 94080 AGENDA FOR PUBLIC SESSION January 24, 2012 9:00 a.m. WELCOME AND COMMITTEE INTRODUCTIONS Harold T. Shapiro, Ph.D. IOM Committee Chair 9:10 a.m. EVOLVING SCIENTIFIC PRIORITIES AT CIRM Alan Trounson, Ph.D. CIRM President 10:10 a.m. OVERVIEW OF CIRM SCIENTIFIC AND MEDICAL ACCOUNTABILITY STANDARDS WORKING GROUP Bernard Lo, M.D. Standards Working Group Co-Chair 10:50 a.m. BREAK 11:00 a.m. CIRM INTELLECTUAL PROPERTY POLICIES Duane J. Roth CIRM Governing Board Vice Chair and Intellectual Property/Industry Subcommittee Member 11:40 a.m. OVERVIEW OF CIRM TRANSITION PLAN Jonathan Thomas, J.D., D.Phil. Chair, CIRM Governing Board 12:20 p.m. LUNCH

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APPENDIX A 139 1:15 p.m. PERSPECTIVES ON CIRM FROM THE PATIENT ADVOCACY COMMUNITY Jeff Sheehy  Co-Vice Chair of the Scientific and Medical Research Funding Working Group and CIRM Patient Advocate Board Member Director of Communications AIDS Research Institute University of California, San Francisco 1:50 p.m. PERSPECTIVES ON CIRM FROM PRINCIPAL INVESTIGATORS Irina Conboy, Ph.D. Assistant Professor, Department of Bioengineering University of California, Berkeley John P. Murnane, Ph.D. Professor, Department of Radiation Oncology University of California, San Francisco Howard Y. Chang, M.D., Ph.D. Stanford University School of Medicine, Howard Hughes Medical Institute Helen M. Blau, Ph.D. Donald E. and Delia B. Baxter Professor Director, Baxter Laboratory for Stem Cell Biology Stanford University School of Medicine Frederick J. Meyers, M.D., M.A.C.P. (by phone—invited) Executive Associate Dean, University of California, Davis, School of Medicine Alice F. Tarantal, Ph.D. (by phone) Professor and Vice-Chair for Research, Department of Pediatrics University of California, Davis, School of Medicine 3:15 p.m. BREAK continued

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140 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE BOX A-2 Continued 3:30 p.m. RECAP/DISCUSSION OF MORNING AND AFTERNOON PRESENTATIONS 4:00 p.m. PUBLIC COMMENT Individuals register for a slot and have 5 minutes for comments on any topic related to the study charge. 5:00 p.m. ADJOURN OPEN SESSION

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APPENDIX A 141 BOX A-3 Committee on a Review of the California Institute for Regenerative Medicine (CIRM) Arnold and Mabel Beckman Center of the National Academies of Sciences and Engineering Huntington Room 100 Academy Irvine, CA 92617 AGENDA FOR PUBLIC SESSION April 10, 2012 9:00 a.m. WELCOME AND COMMITTEE INTRODUCTIONS Harold T. Shapiro, Ph.D. IOM Committee Chair 9:10 a.m. OVERVIEW OF LITTLE HOOVER COMMISSION REPORT Stuart Drown (by phone) Executive Director, Little Hoover Commission 10:00 a.m. PERSPECTIVES ON CIRM FROM STAKEHOLDERS Ruth Holton-Hodson California Deputy State Controller Ken Taymor, J.D. (by phone) Executive Director, Berkeley Center for Law, Business and Economy John Simpson (by phone) Director, Stem Cell Oversight and Accountability Project Consumer Watchdog Marcy Darnovsky, Ph.D. (by phone) Associate Executive Director, Center for Genetics and Society David Jensen Publisher-Editor, California Stem Cell Report continued

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142 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE BOX A-3 Continued 11:30 a.m. LUNCH 12:00 p.m. PERSPECTIVES FROM CIRM APPLICANTS Leonard H. Rome, Ph.D. (by phone) Senior Associate Dean for Research and Professor of Biological Chemistry, David Geffen School of Medicine University of California, Los Angeles Xuejun H. Parsons, Ph.D. Associate Professor of Regenerative Medicine and Scientific Director in Cardiovascular and Neural Regeneration San Diego Regenerative Medicine Institute 12:45 p.m. PERSPECTIVES ON CIRM FROM DISEASE TEAM INVESTIGATORS Donald Kohn, M.D. Professor, Department of Microbiology, Immunology, and Molecular Genetics and Department of Pediatrics University of California, Los Angeles Larry Goldstein, Ph.D. Professor, Department of Cellular and Molecular Medicine and Department of Neurosciences University of California, San Diego School of Medicine Dennis Clegg, Ph.D. Professor, Molecular, Cellular, and Developmental Biology University of California, Santa Barbara Catriona Jamieson, M.D., Ph.D. Associate Professor, Division of Hematology-Oncology University of California, San Diego Moores Cancer Center

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APPENDIX A 143 2:00 p.m. PERSPECTIVES ON CIRM FROM INDUSTRY Gregory A. Bonfiglio, J.D., Panel Moderator Managing Partner, Proteus Venture Partners Michael D. West, Ph.D. Chief Executive Officer, BioTime, Inc. Allan Robins, Ph.D. Acting Chief Executive Officer, Vice President and Chief Technical Officer, ViaCyte, Inc. Gail K. Naughton, Ph.D. CEO, Chairman of the Board, Histogen, Inc. 3:30 p.m. BREAK 3:45 p.m. PERSPECTIVES ON CIRM FROM TECHNOLOGY TRANSFER OFFICERS Casie Kelly, Ph.D. Technology Transfer Officer, Life Sciences, Office of Intellectual Property & Industry Sponsored Research University of California, Los Angeles 4:30 p.m. PUBLIC COMMENT Individuals register for a slot and have 5 minutes for comments on any topic related to the study charge. 5:30 p.m. ADJOURN OPEN SESSION

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