The National Academy of Sciences and the National Academy of Medicine Committee on Human Gene Editing: Scientific, Medical, and Ethical Considerations was tasked with studying the scientific underpinnings of human gene-editing technologies—including human germline editing—and the clinical, ethical, legal, and social implications of their use. The committee also explored fundamental, underlying principles that could be adapted by any nation considering the development of guidelines for human gene editing. To respond comprehensively to its charge, the committee examined data from a variety of sources, including a review of the literature, open-session meetings and conference calls, public testimony and input, and other publicly available resources.
The National Academies of Sciences, Engineering, and Medicine (the National Academies) appointed a committee of 22 experts to undertake the statement of task. The committee was composed of members with expertise in basic science, clinical research and medicine, law and regulation, ethics and religion, patient advocacy, science communication, public engagement, and the biomedical industry. Appendix D provides the biographical information for each committee member.
MEETINGS AND INFORMATION-GATHERING ACTIVITIES
The committee deliberated from December 2015 to January 2017 to conduct its assessment, and gathered information and data relevant to its statement of task by conducting a review of available literature, inviting stakeholders to share perspectives at public meetings, and soliciting public comments both online and in person.
Several strategies were used to identify literature relevant to the committee’s charge. A search of bibliographic databases, including PubMed, Scopus, Web of Science, ProQuest Research Library, Medline, Embase, and LexisNexis, was conducted to obtain articles from peer-reviewed journals that discussed basic research, clinical applications, patient safety, scientific standards, ethics, oversight, and social issues associated with human gene editing. Staff reviewed recent news and literature to identify articles relevant to the committee’s charge and created a database of references. In addition, committee members, speakers, sponsors, and other interested parties submitted articles, reports, and policy statements on these topics. The committee’s database included several hundred relevant articles and reports, and was updated continuously throughout the study process.
Four of the five meetings held over the course of the study included sessions in which committee members obtained input from a range of stakeholders and members of the public. Three meetings were held in Washington, DC (December 2015, February 2016, and July 2016), and one meeting was held in Paris, France, hosted by the French National Academy of Medicine (April 2016).
The committee’s first meeting in December 2015 was held in association with the 3-day International Summit on Human Gene Editing: A Global Discussion, co-hosted by the U.S. National Academy of Sciences, U.S. National Academy of Medicine, Chinese Academy of Sciences, and The Royal Society of the United Kingdom. Although a separate ad hoc committee planned the summit, it provided a critical opportunity for the study committee to gather information. The summit convened experts from around the world to discuss scientific, ethical, and governance issues associated with human gene-editing research.1
1 The list of speakers at the International Summit included Peter Braude, Annelien Bredenoord, Philip Campbell, Alta Charo, George Church, Ralph Cicerone, Chad Cowan, George Daley,
The committee’s second meeting in February 2016 included perspectives from potentially affected stakeholder communities such as patient groups and representatives from companies developing gene editing–based therapeutics. It also featured presentations from experts on models for public engagement as well as federal and institutional oversight bodies.
The study’s third meeting in April 2016, hosted by the French National Academy of Medicine in Paris, France, explored the principles underlying governance of gene editing. Speakers during the meeting provided international perspectives spanning permissive, neutral, precautionary, and preventive governance approaches. Meeting discussions also addressed potential therapeutic clinical applications for human germline gene editing. This meeting was held the day following a gene-editing workshop convened by the Federation of European Academies of Medicine, the U.K. Academy of Medical Sciences, and the French Academy of Medicine (FEAM, UKAMS, and ANM, 2017). A number of committee members were able to participate in the preceding FEAM workshop, which provided an important opportunity to learn further about gene-editing regulatory and governance concerns and strategies across the European community.2
Finally, the study’s fourth meeting in July 2016 provided input on several of the social issues associated with human gene editing, including race and genetics in U.S. history and the intersection of moral views and public policy. The list of speakers who provided input to the committee in these meeting sessions is below.
The committee’s data-gathering meetings also provided opportunities for the committee to engage and interact with a variety of stakeholders. Each public meeting included a public comment period, in which the committee invited input from any interested party. The committee also worked to make its activities as transparent and accessible as possible and to ac-
Marcy Darnovsky, Victor Dzau, Fola Esan, Barbara Evans, William Foster, Bärbel Friedrich, Hille Haker, John Harris, John Holdren, Rudolf Jaenisch, Weizhi Ji, Pierre Jouannet, J. Keith Joung, Daniel Kevles, Jonathan Kimmelman, Eric Lander, Ephrat Levy-Lahad, Jinsong Li, Robin Lovell-Badge, Gary Marchant, Jennifer Merchant, Keymanthri Moodley, Indira Nath, Staffan Normark, Kyle Orwig, Pilar Ossorio, Duanquing Pei, Matthew Porteus, K. Vijay Raghavan, Klaus Rajewsky, Thomas Reiss, Janet Rossant, Ismail Serageldin, Bill Skarnes, John Skehel, Azim Surani, Sharon Terry, Adrian Thrasher, Fyodor Urnov, Marco Weinberg, Ernst-Ludwig Winnacker, Zhihong Xu, and Feng Zhang. Presentations and other materials from the Summit are available at http://nationalacademies.org/gene-editing/Gene-Edit-Summit/index.htm (accessed January 7, 2017).
2 The formal report of the FEAM workshop is available at http://www.feam-site.eu/cms/docs/activities/humangenomeeditingworkshop2016report.pdf.
commodate those with special needs or those who may not have been able to attend in person.
The study website was updated regularly to reflect the recent and planned activities of the committee. Study outreach also included a study-specific email address for comments and questions and social media feeds and tags. A subscription to regular email updates was available to share further information and solicit additional comments and input to the committee.
Live video streams with closed captioning and links to an online public comment tool were provided throughout the course of the study to allow the opportunity for input from those unable to attend meetings in person. All online comments and submissions were catalogued in the study’s public access file. Any information provided to the committee from outside sources or through the online comment tool is available by request through the National Academies’ Public Access Records Office.
The following individuals were invited speakers at data-gathering sessions of the committee:
Roberto Andorno, University of Zurich
Mónica López Barahona, Centro de Estudios Biosanitarios
Pierre Bégué, Académie Nationale de Médecine, France
Nick Bostrom, University of Oxford
Abby Bronson, Parent Project Muscular Dystrophy
Dominique Brossard, University of Wisconsin–Madison
Jacqueline Chin, National University of Singapore
Hans Clevers, Hubrecht Institute
Ronald Cole-Turner, Pittsburgh Theological Seminary
Francis Collins, National Institutes of Health
George William Foster, Congressman, IL-11
Søren Holm, University of Manchester
Rahman Jamal, National University of Malaysia
Bartha Knoppers, McGill University
Fredrik Lanner, Karolinska Institute
James Lawford-Davies, Hempsons Law Firm, United Kingdom
John Leonard, Intellia Therapeutics
Bruce Lewenstein, Cornell University
Andrew May, Caribou Biosciences
Vic Myer, Editas Medicine
Alondra Nelson, Columbia University
Erik Parens, Hastings Center
Guido Pennings, Ghent University, Belgium
Pearl O’Rourke, Partners HealthCare
Jackie Leach Scully, Newcastle University
Oliver Semler, University of Cologne
Trevor Thompson, Sickle Cell Foundation of Tennessee
Anna Veiga, Center of Regenerative Medicine, Barcelona, Spain
Thomas Voit, University College London
Elizabeth Vroom, United Parent Projects Muscular Dystrophy
Keith Wailoo, Princeton University
Michael Werner, Alliance for Regenerative Medicine
Nancy Wexler, Hereditary Disease Foundation
Bethan Wolfenden, Bento Bioworks
Carrie Wolinetz, National Institutes of Health
Philip Yeske, United Mitochondrial Disease Foundation
Xiaomei Zhai, Peking Union Medical College, China
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