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Suggested Citation:"Appendix C: Registered Attendees." 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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Appendix C

Registered Attendees

Asa Abeliovich

Prevail Therapeutics

Amy Adams

National Institute of Neurological Disorders and Stroke

Andrew Adams

Eli Lilly and Company

Julie Adams

PPD Biotech

Diaa Ahmed

Utrecht University

Zeshan Ahmed

Eli Lilly and Company

Effie Albanis

Neurogene, Inc.

Robert Alexander

Takeda Pharmaceuticals

Reem Aljuhani

Georgetown University

Kathleen Anderson

National Institute of Mental Health

Megan Anderson Brooks

CRD Associates

Patrick Antonellis

Eli Lilly and Company

Irina Antonijevic

Wave Life Sciences

Mona Ashiya

OrbiMed

Nazem Atassi

Sanofi Genzyme

Shelli Avenevoli

National Institute of Mental Health

Suggested Citation:"Appendix C: Registered Attendees." 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.
×

Jennifer Beierlein

National Science Foundation

Frank Bennett

Ionis Pharmaceuticals

Sangeeta Bhargava

National Eye Institute

Anuradha Bhattacharyya

PTC Therapeutics

Dominika Bielak

Queens College, The City University of New York

David Bleakman

Redpin Therapeutics

Antonello Bonci

National Institute on Drug Abuse

Carsten Bonnemann

National Institute of Neurological Disorders and Stroke

Lizbet Boroughs

Association of American Universities

Chris Boshoff

National Institute of Neurological Disorders and Stroke

Linda Brady

National Institute of Mental Health

David Bredt

Janssen R&D

Katja Brose

Chan Zuckerberg Initiative

Bob Brown

Dicerna Pharmaceuticals

Bettina Buhring

National Institute of Mental Health

Daniel Burch

PPD Biotech

Joseph Buxbaum

Icahn School of Medicine at Mount Sinai

Sarah Caddick

Thalamic

Rosa Canet-Aviles

Foundation for the National Institutes of Health

Mantej (Nimi) Chhina

BioMarin Pharmaceutical, Inc.

Lisa Chong

Science Magazine

Stephanie Ciarlone

PTC Therapeutics

Timothy Coetzee

National Multiple Sclerosis Society

Jonathan Cohen

Princeton University

Renee Corcoran

Axovant

Ronald Crystal

Weill Cornell Medicine

Suggested Citation:"Appendix C: Registered Attendees." 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.
×

Bernard Dardzinski

Uniformed Services University of the Health Sciences

Beverly Davidson

Children’s Hospital of Philadelphia

University of Pennsylvania Perelman School of Medicine

Paula Demassi

Ministry of Public Health of Uruguay

Sarah DeVos

Denali Therapeutics

Jamie Driscoll

National Institute of Mental Health

Hank Dudek

Dicerna Pharmaceuticals

Lee Dudka

Dudka & Associates

Richard Esposito

U.S. Bureau of Labor Statistics

Keith Fargo

Alzheimer’s Association

Boris Feldman

Rite Aid Pharmacy

Boris Feldman

Wilson Sonsini Goodrich & Rosati

E’Lissa Flores

Milken Institute

Allyson Gage

Cohen Veterans Bioscience

Marcos Geovanini

PrevCor

Shaoqin Sarah Gong

University of Wisconsin–Madison

Joshua Gordon

National Institute of Mental Health

Viviana Gradinaru

California Institute of Technology

Julie Hagan

Apic Bio, Inc.

John Hall

IXICO

Chris Henderson

Biogen

Ross Henderson

CAREM, LLC

William Herring

Merck

Kevin Hibbert

Neurogene, Inc.

Ramona Hicks

One Mind

Mi Hillefors

National Institute of Mental Health

Richard Hodes

National Institute on Aging

Stuart Hoffman

Department of Veterans Affairs

Suggested Citation:"Appendix C: Registered Attendees." 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.
×

Bruce Hope

National Institute on Drug Abuse

Nina Hsu

National Institutes of Health

Steven Hyman

The Broad Institute

Yoshimasa Ito

Eisai

Marcus Jackson

Queens College, The City University of New York

Anjana Jain

Food and Drug Administration

Frances Jensen

University of Pennsylvania Health System

Sophia Jeon

National Institute of Neurological Disorders and Stroke

Jeymohan Joseph

National Institute of Mental Health

Akiva Katz

University of Illinois College of Medicine Rockford

Petra Kaufmann

AveXis

Anastasia Khvorova

RNA Therapeutics Institute

University of Massachusetts Medical School

Sooja Kim

National Institutes of Health

Rune Kjeken

Norwegian Medicines Agency

Kelly Knopp

Eli Lilly and Company

Ying-Yee Kong

Department of Veterans Affairs

Jeffrey Kordower

Rush University Medical Center

Paul Korner

Axovant Gene Therapies

Walter Koroshetz

National Institute of Neurological Disorders and Stroke

Ana Kostic

Seaver Autism Center for Research & Treatment, Icahn School of Medicine at Mount Sinai

Robert Kotin

Generation Bio

University of Massachusetts Medical School

John Krystal

Yale University School of Medicine

Lina Kubli

Department of Veterans Affairs

Story Landis

National Institute of Neurological Disorders and Stroke

Suggested Citation:"Appendix C: Registered Attendees." 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.
×

Larissa Lapteva

Food and Drug Administration

Timothy LaVaute

National Institute of Neurological Disorders and Stroke

Ernestine Lenteu

National Institute of Neurological Disorders and Stroke

Alan Leshner

American Association for the Advancement of Science

Liza Litvina

National Institute of Neurological Disorders and Stroke

Darya Litvinchuk

Apic Bio

David Litwack

Prevail Therapeutics

Laura Mamounas

National Institute of Neurological Disorders and Stroke

Glenn Mannheim

Food and Drug Administration

Janet Marchibroda

Bockorny Group

Peter Marks

Food and Drug Administration

Christine Marx

Duke University School of Medicine

Langston McKee

U.S. Agency for International Development

David McMullen

National Institutes of Health

Douglas Meinecke

National Institute of Mental Health

Fany Messanvi

National Institutes of Health

Mike Michaelides

National Institute on Drug Abuse

Enrique Michelotti

National Institute of Mental Health

Timothy Miller

PPD, Inc.

Yi Mo

Axovant Sciences

Jill Morris

National Institute of Neurological Disorders and Stroke

Jennifer Moser

Department of Veterans Affairs

Mattia Mroso

American Association for the Advancement of Science

Avindra Nath

National Institute of Neurological Disorders and Stroke

Rosie Stovell O’Donnell

Encoded Therapeutics

Suggested Citation:"Appendix C: Registered Attendees." 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.
×

Steven Oh

Food and Drug Administration

Eileen O’Reilly

Axios

Michael Ortiz

Roivant Sciences

Maire Osborn

Dicerna Pharmaceuticals

Michael Panzara

Wave Life Sciences

Bryan Parker

International Healthcare

Tom Parry

Ovid Therapeutics

Creighton Phelps

National Institute on Aging

Keith Phillips

Eli Lilly and Company

Richard Podell

Unaffiliated

Nicole Polinski

The Michael J. Fox Foundation for Parkinson’s Research

Jenn Poole

Takeda Pharmaceuticals

Gabriele Proetzel

Takeda Pharmaceuticals

Ronald Przygodzki

Department of Veterans Affairs

Raj Puri

Food and Drug Administration

Sesquile Ramon

Biotechnology Innovation Organization

Elizabeth Ramsburg

Janssen Pharmaceuticals

Kathleen Reape

Spark Therapeutics

Barbara Redman

New York University

Lorenzo Refolo

National Institute on Aging

Lauren Reoma

National Institute of Neurological Disorders and Stroke

Lawrence Richards

American Psychological Association

Christopher Ricupero

Columbia University Medical Center

Becky Roof

National Institute of Neurological Disorders and Stroke

Erik Runko

National Science Foundation

Cristina Sampaio

CHDI Foundation

R. Jude Samulski

University of North Carolina at Chapel Hill

Suggested Citation:"Appendix C: Registered Attendees." 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.
×

Eric Schaeffer

Janssen R&D

Tamera Schneider

National Science Foundation

Nina Schor

National Institute of Neurological Disorders and Stroke

Kelly Servick

Science Magazine

Douglas Sheeley

National Institute of Dental and Craniofacial Research

Chun-Pyn Shen

EMD Serono, Inc.

Lamya Shihabuddin

Sanofi

John Sims

Eli Lilly and Company

Anirudh Srirangam

Roivant Sciences

Michael Steinmetz

National Eye Institute

Junghae Suh

Rice University

James Sweet

Wilson Sonsini Goodrich & Rosati

Holly Tabor

Stanford University School of Medicine

Edmund Talley

National Institute of Neurological Disorders and Stroke

Amir Tamiz

National Institute of Neurological Disorders and Stroke

Hiroaki Tani

Eli Lilly and Company

Anderson Umah

Neolife Health International

Akshay Vaishnaw

Alnylam

Rita Valentino

National Institute on Drug Abuse

Rosaire Verna

Georgetown University

Christina Vert

National Institute of Neurological Disorders and Stroke

Jeannie Visootsak

Neurogene

Bhavya Voleti

Merck

Nora Volkow

National Institute on Drug Abuse

Hao Wang

Takeda Pharmaceuticals

Shiyu Wang

Dicerna Pharmaceuticals

Suggested Citation:"Appendix C: Registered Attendees." 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.
×

Jackie Ward

National Institute of Neurological Disorders and Stroke

Andrew Welchman

Wellcome Trust

Pamella Williams

Health Services Authority

Doug Williamson

Lundbeck

Rachel Witten

Food and Drug Administration

Clinton Wright

National Institute of Neurological Disorders and Stroke

Rui Wu

MeiraGTx

Winifred Wu

Strategic Regulatory Partners, LLC

Yining Xie

National Institutes of Health

Li-chiung Yang

Unaffiliated

Suggested Citation:"Appendix C: Registered Attendees." 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 73
Suggested Citation:"Appendix C: Registered Attendees." 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 74
Suggested Citation:"Appendix C: Registered Attendees." 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 75
Suggested Citation:"Appendix C: Registered Attendees." 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 76
Suggested Citation:"Appendix C: Registered Attendees." 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 77
Suggested Citation:"Appendix C: Registered Attendees." 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 78
Suggested Citation:"Appendix C: Registered Attendees." 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 79
Suggested Citation:"Appendix C: Registered Attendees." 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 80
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 Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop
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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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