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The International Commission on the Clinical Use of Human Germline Genome Editing was tasked with developing a framework for scientists, clinicians, and regulatory authorities to consider when assessing potential clinical uses of human germline genome editing, should society conclude that heritable human genome editing (HHGE) applications are acceptable.
COMMISSION COMPOSITION
The U.S. National Academy of Medicine, the U.S. National Academy of Sciences, and the U.K.’s Royal Society appointed a Commission of 18 experts to undertake the Statement of Task. The Commission’s membership spans 10 nations and 4 continents and includes experts in science, medicine, genetics, ethics, psychology, regulation, and law. Appendix B provides biographical information for each Commissioner.
In addition, an International Oversight Board (IOB) of leaders from national academies of sciences and international institutions was charged with ensuring that the Commission followed due processes, including approving the Statement of Task and membership of the Commission and ensuring that the Commission’s report underwent rigorous external review prior to publication.
MEETINGS AND INFORMATION-GATHERING ACTIVITIES
The Commission deliberated from approximately June 2019 through March 2020 to conduct its assessment and prepare its final report. To
address its task, the Commission analyzed information obtained from current literature and other publicly available resources and undertook information-gathering activities such as inviting stakeholders to share perspectives at public meetings, holding webinars, and soliciting public input online and in person.
Public Meetings and Webinars
Sessions at meetings and webinars held over the course of the study enabled Commissioners to obtain input from a range of stakeholders and members of the public.
The Commission’s first meeting was held in August 2019 in Washington, DC. Public sessions provided an opportunity for the Commission to discuss its Statement of Task with the co-chairs of IOB and sponsoring organizations and to hear presentations on the state of understanding of genetics and genetic manipulation; on somatic genome editing translational pathways from scientists, developers, and regulatory bodies; and on the views of genetic disease patient communities.
In November 2019, the Commission held a second meeting and workshop in London, United Kingdom. The Commission heard from invited experts on topics such as the medical ethics of HHGE, how clinical use of HHGE would intersect with the use of assisted reproductive technologies, and technologies that might enable HHGE, including making and validating edits in embryos and germ cells, and what we can learn from animal models. In addition, the Commission hosted a session on governance developed in consultation with two members of the WHO Advisory Committee.
At a third meeting in January 2020, Commission members developed the conclusions and recommendations presented in this report.
In October 2019, the Commission also held a series of four public webinars on the state of research in relevant areas. These webinars covered (1) informed consent in the context of HHGE, (2) the impact of genome editing on embryo viability and the state of the science on editing spermatogonial stem cells, (3) homology directed repair and single cell genomics, and (4) validating on-target and off-target edits.
The list of speakers who provided input to the Commission in these meeting and webinar sessions is below.
Public Comments
The Commission’s data-gathering meetings provided opportunities for the Commission to interact with a variety of stakeholders. Each public meeting included a public comment period, in which the Commission
invited input from any interested party. The Commission also worked to make its activities as transparent and accessible as possible.
The study websites hosted by the U.S. National Academies and the U.K.’s Royal Society were updated regularly to reflect recent and planned Commission activities. Study outreach included a study-specific email address for comments and questions. A subscription to email updates was available to share further information and solicit additional comments and input to the Commission.
Live video streams with closed captioning were provided throughout the course of the study to allow the opportunity for input from those unable to attend public meetings in person. Information provided to the Commission from outside sources or through online comment is available by request through the National Academies’ Public Access Records Office.
Call for Evidence
To inform its deliberations, the Commission invited responses to a public call for evidence during fall 2019. Several of the questions invited broad input on considerations associated with HHGE, while others asked for technical input in areas such as preclinical safety and efficacy and the use of genome editing in human embryos. Still other questions asked about considerations for informed consent, long-term monitoring, and oversight of HHGE.
There were 83 responses received. Respondents came from every continent and included academic leaders, lawyers, social scientists, philosophers, and representatives from disability advocacy groups, journals, national ethics councils, industry, and scientific societies.
Consulted Experts
The following individuals were invited speakers at data-gathering sessions of the Commission or provided other expert input.
Sonia Abdelhak
Institut Pasteur de Tunis, Tunisia
Britt Adamson
Princeton University, USA
Fabiana Arzuaga
Ministry of Science, Technology and Productive Innovation, Argentina
Richard Ashcroft
City University of London, U.K.
Christina Bergh
University of Gothenburg, Sweden
Peter Braude
King’s College London, U.K.
Annelien Bredenoord
University Medical Centre Utrecht, Netherlands
Aravinda Chakravarti
New York University School of Medicine
Sarah Chan
University of Edinburgh, U.K.
Ellen Clayton
Vanderbilt University, USA
Chad Cowan
Harvard Stem Cell Institute, USA
James Lawford Davies
Hill Dickinson LLP, U.K.
Tarek El-Toukhy
Guy’s and St Thomas’ Hospital NHS Foundation Trust, U.K.
Frances Flinter
King’s College London, U.K.
Denise Gavin
Food and Drug Administration, USA
Melissa Goldstein
George Washington University, USA
Margaret Hamburg
U.S. National Academy of Medicine and Co-chair of WHO Expert Advisory Committee, USA
Muntaser Ibrahim
University of Khartoum, Sudan
Pierre Jouannet
Paris Descartes University, France
Jin-Soo Kim
Seoul National University, South Korea
Robert Klitzman
Columbia University, USA
Bruce Levine
University of Pennsylvania, USA
Robin Lovell-Badge
Francis Crick Institute, U.K.
Sandy Macrae
Sangamo Therapeutics, USA
Julie Makani
Muhimbili University of Health and Allied Sciences, Tanzania
Nick Meade
Genetic Alliance U.K.
Shoukhrat Mitalipov
Oregon Health & Science University, USA
Vic Myer
Editas Medicine, USA
Kathy Niakan
Francis Crick Institute, U.K.
Sarah Norcross
Progress Educational Trust, U.K.
Helen O’Neill
University College London, U.K.
Kyle Orwig
Magee-Womens Research Institute, USA
Matthew Porteus
Stanford University, USA
Adam Pearson
Actor, Presenter, and Campaigner, U.K.
Catherine Racowsky
Brigham and Women’s Hospital, USA
Jackie Leach Scully
University of New South Wales, Australia and Newcastle University, U.K.
Azim Surani
University of Cambridge, U.K.
Sarah Teichmann
Wellcome Sanger Institute, U.K.
Sharon Terry
Genetic Alliance, USA
Peter Thompson
Human Fertilisation and Embryology Authority, U.K.
Carrie Wolinetz
National Institutes of Health, USA
Xiaoliang Sunney Xie
Peking University, China
Hui Yang
Institute of Neuroscience, Chinese Academy of Sciences, China
Mohammed Zahir
Muhimbili University of Health and Allied Sciences, Tanzania