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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
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Advancing Gene-Targeted
Therapies for Central
Nervous System Disorders

PROCEEDINGS OF A WORKSHOP

Lisa Bain and Clare Stroud, Rapporteurs

Forum on Neuroscience and Nervous System Disorders

Board on Health Sciences Policy

Health and Medicine Division

images

THE NATIONAL ACADEMIES PRESS
Washington, DC
www.nap.edu

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
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THE NATIONAL ACADEMIES PRESS 500 Fifth Street, NW Washington, DC 20001

This activity was supported by contracts between the National Academy of Sciences and Alzheimer’s Association; Cohen Veterans Bioscience; Department of Health and Human Services’ Food and Drug Administration (5R13FD005362-02) and National Institutes of Health (NIH) (HHSN26300089 [Under Master Base #DHHS-10002880]) through the National Center for Complementary and Integrative Health, National Eye Institute, National Institute of Mental Health, National Institute of Neurological Disorders and Stroke, National Institute on Aging, National Institute on Alcohol Abuse and Alcoholism, National Institute on Drug Abuse, and NIH Blueprint for Neuroscience Research; Department of Veterans Affairs (VA240-14-C-0057); Eli Lilly and Company; Foundation for the National Institutes of Health; Gatsby Charitable Foundation; Janssen Research & Development, LLC; The Kavli Foundation; Lundbeck Research USA; Merck Research Laboratories; The Michael J. Fox Foundation for Parkinson’s Research; National Multiple Sclerosis Society; National Science Foundation (BCS-1064270); One Mind; Sanofi; Society for Neuroscience; Takeda Pharmaceuticals International, Inc.; The University of Rhode Island; and Wellcome Trust. Any opinions, findings, conclusions, or recommendations expressed in this publication do not necessarily reflect the views of any organization or agency that provided support for the project.

International Standard Book Number-13: 978-0-309-49584-4
International Standard Book Number-10: 0-309-49584-9
Digital Object Identifier: https://doi.org/10.17226/25529

Additional copies of this publication are available from the National Academies Press, 500 Fifth Street, NW, Keck 360, Washington, DC 20001; (800) 624-6242 or (202) 334-3313; http://www.nap.edu.

Copyright 2019 by the National Academy of Sciences. All rights reserved.

Printed in the United States of America

Suggested citation: National Academies of Sciences, Engineering, and Medicine. 2019. Advancing gene-targeted therapies for central nervous system disorders: Proceedings of a workshop. Washington, DC: The National Academies Press. https://doi.org/10.17226/25529.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×

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The National Academy of Sciences was established in 1863 by an Act of Congress, signed by President Lincoln, as a private, nongovernmental institution to advise the nation on issues related to science and technology. Members are elected by their peers for outstanding contributions to research. Dr. Marcia McNutt is president.

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Learn more about the National Academies of Sciences, Engineering, and Medicine at www.nationalacademies.org.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
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Consensus Study Reports published by the National Academies of Sciences, Engineering, and Medicine document the evidence-based consensus on the study’s statement of task by an authoring committee of experts. Reports typically include findings, conclusions, and recommendations based on information gathered by the committee and the committee’s deliberations. Each report has been subjected to a rigorous and independent peer-review process and it represents the position of the National Academies on the statement of task.

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
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PLANNING COMMITTEE ON ADVANCING GENE THERAPY FOR NERVOUS SYSTEM DISORDERS1

STORY LANDIS (Co-Chair), Director Emeritus, National Institute of Neurological Disorders and Stroke

LAMYA SHIHABUDDIN (Co-Chair), Sanofi

ZESHAN AHMED, Eli Lilly and Company

DAVID BREDT, Janssen Research & Development, LLC

DANIEL BURCH, PPD Biotech

JOSEPH BUXBAUM, Icahn School of Medicine at Mount Sinai

BEVERLY DAVIDSON, University of Pennsylvania Perelman School of Medicine

JOSHUA GORDON, National Institute of Mental Health

FRANCES JENSEN, University of Pennsylvania

JOHN KRYSTAL, Yale University

MARYANN REDFORD, National Eye Institute

TODD SHERER, The Michael J. Fox Foundation for Parkinson’s Research

HAO WANG, Takeda Pharmaceuticals

CLINTON WRIGHT, National Institute of Neurological Disorders and Stroke

Health and Medicine Division Staff

CLARE STROUD, Director, Forum on Neuroscience and Nervous System Disorders

SHEENA M. POSEY NORRIS, Program Officer

PHOENIX WILSON, Senior Program Assistant

ANDREW M. POPE, Senior Director, Board on Health Sciences Policy

___________________

1 The National Academies of Sciences, Engineering, and Medicine’s planning committees are solely responsible for organizing the workshop, identifying topics, and choosing speakers. The responsibility for the published Proceedings of a Workshop rests with the workshop rapporteurs and the institution.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×

FORUM ON NEUROSCIENCE AND NERVOUS SYSTEM DISORDERS1

JOHN KRYSTAL (Co-Chair), Yale University

STORY LANDIS (Co-Chair), Director Emeritus, National Institute of Neurological Disorders and Stroke

SUSAN AMARA, Society for Neuroscience

RITA BALICE-GORDON, Sanofi

KATJA BROSE, Chan Zuckerberg Initiative

EMERY BROWN, Harvard Medical School and Massachusetts Institute of Technology

DANIEL BURCH, PPD Biotech

JOSEPH BUXBAUM, Icahn School of Medicine at Mount Sinai

SARAH CADDICK, Gatsby Charitable Foundation

ROSA CANET-AVILES, Foundation for the National Institutes of Health

MARIA CARRILLO, Alzheimer’s Association

E. ANTONIO CHIOCCA, Harvard Medical School (until July 2019)

TIMOTHY COETZEE, National Multiple Sclerosis Society

JONATHAN COHEN, Princeton University

ROBERT CONLEY, Eli Lilly and Company

JAMES DESHLER, National Science Foundation

BILLY DUNN, Food and Drug Administration

MICHAEL EGAN, Merck Research Laboratories

JOSHUA GORDON, National Institute of Mental Health

HENRY T. GREELY, Stanford University (until July 2019)

RAQUEL GUR, University of Pennsylvania

MAGALI HAAS, Cohen Veterans Bioscience

RAMONA HICKS, One Mind

RICHARD HODES, National Institute on Aging

STUART HOFFMAN, Department of Veterans Affairs

STEVEN HYMAN, Broad Institute of Massachusetts Institute of Technology and Harvard University

FRANCES JENSEN, University of Pennsylvania

GEORGE KOOB, National Institute on Alcohol Abuse and Alcoholism

WALTER KOROSHETZ, National Institute of Neurological Disorders and Stroke

ALAN LESHNER, American Association for the Advancement of Science (Emeritus)

HUSSEINI MANJI, Janssen Research & Development, LLC

___________________

1 The National Academies of Sciences, Engineering, and Medicine’s forums and roundtables do not issue, review, or approve individual documents. The responsibility for the published Proceedings of a Workshop rests with the workshop rapporteurs and the institution.

Page viii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×

CAROLINE MONTOJO, The Kavli Foundation

ATUL PANDE, Tal Medical

STEVEN PAUL, Voyager Therapeutics

EMILIANGELO RATTI, Takeda Pharmaceuticals International, Inc.

DOUGLAS SHEELEY, National Institute of Dental and Craniofacial Research

TODD SHERER, The Michael J. Fox Foundation for Parkinson’s Research

DAVID SHURTLEFF, National Center for Complementary and Integrative Health

PAUL SIEVING, National Eye Institute

NORA VOLKOW, National Institute on Drug Abuse

ANDREW WELCHMAN, Wellcome Trust

DOUG WILLIAMSON, Lundbeck

STEVIN ZORN, MindImmune Therapeutics, Inc.

Health and Medicine Division Staff

CLARE STROUD, Forum Director

SHEENA M. POSEY NORRIS, Program Officer

PHOENIX WILSON, Senior Program Assistant

BARDIA MASSOUDKHAN, Financial Associate

ANDREW M. POPE, Senior Director, Board on Health Sciences Policy

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×

Reviewers

This Proceedings of a Workshop was reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise. The purpose of this independent review is to provide candid and critical comments that will assist the National Academies of Sciences, Engineering, and Medicine in making each published proceedings as sound as possible and to ensure that it meets the institutional standards for quality, objectivity, evidence, and responsiveness to the charge. The review comments and draft manuscript remain confidential to protect the integrity of the process.

We thank the following individuals for their review of this proceedings:

JEAN BENNETT, University of Pennsylvania Perelman School of Medicine

VIVIANA GRADINARU, California Institute of Technology

Although the reviewers listed above provided many constructive comments and suggestions, they were not asked to endorse the content of the proceedings nor did they see the final draft before its release. The review of this proceedings was overseen by ELI ADASHI, Brown University. He was responsible for making certain that an independent examination of this proceedings was carried out in accordance with standards of the National Academies and that all review comments were carefully considered. Responsibility for the final content rests entirely with the rapporteurs and the National Academies.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
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Page R7
Page viii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×
Page R8
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×
Page R9
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×
Page R10
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25529.
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Page R12
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On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.

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