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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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Appendix D

Registered Attendees

Sean Adler

Johns Hopkins University

Rachael Anatol

Food and Drug Administration

Eric Anthony

International Society for Stem Cell Research

Naomi Aronson

Blue Cross Blue Shield Association

Jane Atkinson

National Center for Advancing Translational Sciences

Dylan Bechtle

Genentech

Catherine Bollard

The George Washington University

Vence Bonham

National Human Genome Research Institute

Imein Bousnina

Genentech

Eilse Boutcher

Facing Our Risk of Cancer Empowered

Luke Brewster

Emory University; Atlanta Veterans Affairs Medical Center

Ariadne Campble

The Washington Center

Cora Trelles Cartagena

National Institutes of Health Vaccine Research Center

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.
×

Maitreyi Chattopadhyay

Food and Drug Administration

Nimi Chhina

BioMarin Pharmaceutical Inc.

Elaine Collier

National Center for Advancing Translational Sciences

Kayla Cooper

National Institutes of Health

Michelle Cortes

National Institute of Dental and Craniofacial Research

Abla Creasey

California Institute for Regenerative Medicine

Sameera Daniels

Ramsey Decision Theoretics

Barto Diaz

The Consumer Goods Forum

Nancy Drakeford

Todd Durham

Foundation Fighting Blindness

Ray Ebert

National Heart, Lung, and Blood Institute

Sam Edland

Optum

Michael Ferenczy

GlaxoSmithKline

Tempora Fisher

National Institutes of Health

Mae Frances Frazier

U.S. Postal Service Headquarters

Dylan George

In-Q-Tel

Lawrence Goldstein

University of California, San Diego

Cynthia Golson

Department of Corrections

Mary Groesch

National Institutes of Health

Dawn Henke

Standards Coordinating Body

Brittany Hollister

National Human Genome Research Institute

Christopher Hug

Sanofi-Genzyme

Nina Hunter

Food and Drug Administration

Rosemarie Hunziker

Connexon Life Sciences Consulting

Jean Hu-Primmer

GlaxoSmithKline Vaccines

Ilan Irony

Food and Drug Administration

Lisa Jordan

DC/MD League for Nursing

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.
×

Samira Kiani

Arizona State University

Ioannis Koutroulis

Children’s National Hospital

Timothy LaVaute

National Institute of Neurological Disorders and Stroke, National Institutes of Health

Rachel Levinson

Arizona State University

Sheng Lin-Gibson

Biosystems and Biomaterials Division, National Institute of Standards and Technology

Nadya Lumelsky

National Institutes of Health

Glenn MacLean

Colorado Center for Reproductive Medicine

Terry Magnuson

University of North Carolina at Chapel Hill

Diane Maloney

Food and Drug Administration

Richard McFarland

Advanced Regenerative Manufacturing Institute

Paul Melmeyer

Muscular Dystrophy Association

Joseph Menetski

Foundation for the National Institutes of Health

Maria Millan

California Institute for Regenerative Medicine

Alexis Miller

Sanofi

Nancy Miller

National Cancer Institute

Jill Morris

National Institutes of Health

Jack Mosher

International Society for Stem Cell Research

Venkatesha Murthy

Takeda

Ramraj Singh Nayak

Guru Ghasidas University, Bilaspur C.G.

Lisa Neuhold

National Eye Institute

Phillip Nichols

Gwood LLC

Patrick Nosker

Affinity Asset Advisors

Kristina Obom

Johns Hopkins University

Catherine O’Riordan

Sanofi

Elizabeth Ottinger

Therapeutic Development Branch, National Center for Advancing Translational Sciences, National Institutes of Health

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.
×

Jai Pandey

Food and Drug Administration

Bryan Parker

International Healthcare Access Group

Amy Patterson

National Heart, Lung, and Blood Institute

Duanqing Pei

Chinese Academy of Sciences

Mary Perry

National Institutes of Health

Leland Pierce

Food and Drug Administration

Anne Plant

National Institute of Standards and Technology

Deanna Portero

Office of Rare Diseases Research

Kimberlee Potter

Department of Veterans Affairs

Ronald Przygodzki

Department of Veterans Affairs

Yesenia Quintanilla

Prince George’s County Public Schools

Nishadi Rajapakse

National Institutes of Health

Sesquile Ramon

Biotechnology Innovation Organization

Peter Reczek

Standards Coordinating Body for Regenerative Medicine

Barbara Redman

New York University

Derek Robertson

Maryland Sickle Cell Disease Association

Kelly Rose

Burroughs Wellcome Fund

Anne Rowzee

Food and Drug Administration

Krishnendu Roy

Georgia Tech; National Science Foundation Engineering Research Center for Cell Manufacturing Technologies

Daisy Rubio

Kaiser

Krishanu Saha

University of Wisconsin–Madison

Rachel Salzman

American Society for Gene & Cell Therapy

Sandhya Sanduja

Food and Drug Administration

Frank Sasinowski

Hyman, Phelps & McNamara, PC

Ivonne Schulman

National Institute of Diabetes and Digestive and Kidney Disorders

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.
×

Ben Shaberman

Foundation Fighting Blindness

John Sheridan

Cystic Fibrosis Foundation

David Shindell

Odeon

Mona Shing

Food and Drug Administration

Eric Sid

National Center for Advancing Translational Sciences

Beth-Anne Sieber

National Institute of Neurological Disorders and Stroke

Jay Siegel

Food and Drug Administration; Johnson & Johnson (Retired)

Arthur Simen

Takeda

Mitchel Sivilotti

Centre for Commercialization of Regenerative Medicine

William Skach

Cystic Fibrosis Foundation

Lana Skirboll

Sanofi

Vivian Smith

Robert Star

National Institute of Diabetes and Digestive and Kidney Disorders

Sohel Talib

California Institute for Regenerative Medicine

William Tente

Humacyte

Katherine Tsokas

Johnson & Johnson

Timothy Turnham

Voz Advisors

James Valentine

Hyman, Phelps & McNamara, PC

Dan (Kelly) Wang

Food and Drug Administration

Anthony Welch

National Cancer Institute

Lis Welniak

National Heart, Lung, and Blood Institute

Michael Werner

Alliance for Regenerative Medicine

Keith Westby

IVERIC bio

Celia Witten

Food and Drug Administration

Ling Wong

National Institute of Neurological Disorders and Stroke

Cris Woolston

Sanofi

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.
×

Nora Yang

Cura Ventures

Carolyn Yong

Food and Drug Administration

Lih Young

Katie Zander

Standards Coordinating Body

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 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.
×
Page 106
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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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