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Suggested Citation:"Appendix D: Registered Attendees." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Page 101
Suggested Citation:"Appendix D: Registered Attendees." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×
Page 102
Suggested Citation:"Appendix D: Registered Attendees." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×
Page 103
Suggested Citation:"Appendix D: Registered Attendees." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×
Page 104
Suggested Citation:"Appendix D: Registered Attendees." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×
Page 105
Suggested Citation:"Appendix D: Registered Attendees." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Page 106

Below is the uncorrected machine-read text of this chapter, intended to provide our own search engines and external engines with highly rich, chapter-representative searchable text of each book. Because it is UNCORRECTED material, please consider the following text as a useful but insufficient proxy for the authoritative book pages.

Appendix D Registered Attendees Sean Adler Vence Bonham Johns Hopkins University National Human Genome Research Institute Rachael Anatol Food and Drug Administration Imein Bousnina Genentech Eric Anthony International Society for Stem Cell Eilse Boutcher Research Facing Our Risk of Cancer Empowered (FORCE) Naomi Aronson Blue Cross Blue Shield Association Luke Brewster Emory University; Atlanta Veterans Jane Atkinson Affairs Medical Center National Center for Advancing Translational Sciences Ariadne Campble The Washington Center Dylan Bechtle Genentech Cora Trelles Cartagena National Institutes of Health Catherine Bollard Vaccine Research Center The George Washington University 101 PREPUBLICATION COPY—Uncorrected Proofs

102 NOVEL CLINICAL TRIAL DESIGNS FOR GENE-BASED THERAPIES Maitreyi Chattopadhyay Tempora Fisher Food and Drug Administration National Institutes of Health Nimi Chhina Mae Frances Frazier BioMarin Pharmaceutical Inc. U.S. Postal Service Headquarters Elaine Collier Dylan George National Center for Advancing In-Q-Tel Translational Sciences Lawrence Goldstein Kayla Cooper University of California, San Diego National Institutes of Health Cynthia Golson Michelle Cortes Department of Corrections National Institute of Dental and Craniofacial Research Mary Groesch National Institutes of Health Abla Creasey California Institute for Dawn Henke Regenerative Medicine Standards Coordinating Body Sameera Daniels Brittany Hollister Ramsey Decision Theoretics National Human Genome Research Institute Barto Diaz The Consumer Goods Forum Christopher Hug Sanofi-Genzyme Nancy Drakeford Nina Hunter Todd Durham Food and Drug Administration Foundation Fighting Blindness Rosemarie Hunziker Ray Ebert Connexon Life Sciences Consulting National Heart, Lung, and Blood Institute Jean Hu-Primmer GlaxoSmithKline Vaccines Sam Edland Optum Ilan Irony Food and Drug Administration Michael Ferenczy GlaxoSmithKline Lisa Jordan DC/MD League for Nursing PREPUBLICATION COPY—Uncorrected Proofs

APPENDIX D 103 Samira Kiani Maria Millan Arizona State University California Institute for Regenerative Medicine Ioannis Koutroulis Children’s National Hospital Alexis Miller Sanofi Timothy LaVaute National Institute of Neurological Nancy Miller Disorders and Stroke, National National Cancer Institute Institutes of Health Jill Morris Rachel Levinson National Institutes of Health Arizona State University Jack Mosher Sheng Lin-Gibson International Society for Stem Cell Biosystems and Biomaterials Research Division, National Institute of Standards and Technology Venkatesha Murthy Takeda Nadya Lumelsky National Institutes of Health Ramraj Singh Nayak Guru Ghasidas University, Bilaspur Glenn MacLean C.G. Colorado Center for Reproductive Medicine Lisa Neuhold National Eye Institute Terry Magnuson University of North Carolina at Phillip Nichols Chapel Hill Gwood LLC Diane Maloney Patrick Nosker Food and Drug Administration Affinity Asset Advisors Richard McFarland Kristina Obom Advanced Regenerative Johns Hopkins University Manufacturing Institute Catherine O’Riordan Paul Melmeyer Sanofi Muscular Dystrophy Association Elizabeth Ottinger Joseph Menetski Therapeutic Development Branch, Foundation for the National National Center for Advancing Institutes of Health Translational Sciences, National Institutes of Health PREPUBLICATION COPY—Uncorrected Proofs

104 NOVEL CLINICAL TRIAL DESIGNS FOR GENE-BASED THERAPIES Jai Pandey Peter Reczek Food and Drug Administration Standards Coordinating Body for Regenerative Medicine Bryan Parker International Healthcare Access Barbara Redman Group New York University Amy Patterson Derek Robertson National Heart, Lung, and Blood Maryland Sickle Cell Disease Institute Association Duanqing Pei Kelly Rose Chinese Academy of Sciences Burroughs Wellcome Fund Mary Perry Anne Rowzee National Institutes of Health Food and Drug Administration Leland Pierce Krishnendu Roy Food and Drug Administration Georgia Tech; National Science Foundation Engineering Anne Plant Research Center for Cell National Institute of Standards Manufacturing Technologies and Technology Daisy Rubio Deanna Portero Kaiser Office of Rare Diseases Research Krishanu Saha Kimberlee Potter University of Wisconsin–Madison Department of Veterans Affairs Rachel Salzman Ronald Przygodzki American Society for Gene & Cell Department of Veterans Affairs Therapy Yesenia Quintanilla Sandhya Sanduja Prince George’s County Public Food and Drug Administration Schools Frank Sasinowski Nishadi Rajapakse Hyman, Phelps & McNamara, PC National Institutes of Health Ivonne Schulman Sesquile Ramon National Institute of Diabetes Biotechnology Innovation and Digestive and Kidney Organization Disorders PREPUBLICATION COPY—Uncorrected Proofs

APPENDIX D 105 Ben Shaberman Sohel Talib Foundation Fighting Blindness California Institute for Regenerative Medicine John Sheridan Cystic Fibrosis Foundation William Tente Humacyte David Shindell Odeon Katherine Tsokas Johnson & Johnsons Mona Shing Food and Drug Administration Timothy Turnham Voz Advisors Eric Sid National Center for Advancing James Valentine Translational Sciences Hyman, Phelps & McNamara, PC Beth-Anne Sieber Dan (Kelly) Wang National Institute of Neurological Food and Drug Administration Disorders and Stroke Anthony Welch Jay Siegel National Cancer Institute Food and Drug Administration; Johnson & Johnson (Retired) Lis Welniak National Heart, Lung, and Blood Arthur Simen Institute Takeda Michael Werner Mitchel Sivilotti Alliance for Regenerative Medicine Centre for Commercialization of Regenerative Medicine Keith Westby IVERIC bio William Skach Cystic Fibrosis Foundation Celia Witten Food and Drug Administration Lana Skirboll Sanofi Ling Wong National Institute of Neurological Vivian Smith Disorders and Stroke Robert Star Cris Woolston National Institute of Diabetes Sanofi and Digestive and Kidney Disorders PREPUBLICATION COPY—Uncorrected Proofs

106 NOVEL CLINICAL TRIAL DESIGNS FOR GENE-BASED THERAPIES Nora Yang Lih Young Cura Ventures Katie Zander Carolyn Yong Standards Coordinating Body Food and Drug Administration PREPUBLICATION COPY—Uncorrected Proofs

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Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.

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