Bioethics Issues in Somatic and Germline Gene Editing: Key Content

Theme 1: Trials Transparency & Data Sharing
:: Selected Bibliography – 12 Sep 2019 –
Selected Bibliography on Trials Transparency and Data Sharing in Gene Therapy_NYAS Workshop_10 Oct 2019_Michael Young

Theme 2: Long-term Follow-up/RWE

:: Selected Bibliography – 12 Sep 2019 –
Selected Bib on LTFU-RWE_NYAS Workshop of Bioethics of Gene Therapies_10 Oct 2019
:: Selected articles

NEJM-Real-World Evidence-What Is It and What Can It Tell Us
Intergenerational Monitoring Gene Editing

Theme 3: Consent [for Life]

:: Selected Bibliography – 12 Sep 2019 –
Selected Bibliography on Informed Consent_NYAS Workshop_Informed Consent in Gene Therapies_10 Oct 2019_Paige Fitzsimmons

Theme 4: Equity/Access/Affordability

:: Selected Bibliography – 12 Sep 2019 –
Selected Bibliography on Equity Access Affordability in Gene Therapy_NYAS Workshop_10 Oct 2019_Joanna Smolenski

:: Selected articles

Core Readings/General

.New England Journal of Medicine
August 1, 2019   Vol. 381 No. 5
Review Article
Frontiers in Medicine: Gene Therapy
Katherine A. High, M.D., and Maria G. Roncarolo, M.D.
Those who have followed the gene-therapy field over the decades may be weary of forward-looking positive statements. However, over the past 3 years, six gene-therapy products have been approved for clinical use. This article describes challenges, risks, and advances in gene-therapy clinical research.
AUDIO: Interview with Dr. Katherine A. High on gene therapy for genetic disease. (15:22) Download
PDF: NEJM_Gene Therapy_High and Roncarolo_Aug 1 2019_nejmra1706910
The Lancet
Vol 391 March 3, 2018
Lancet Commission_Stem Cells – Regenerative Medicine_March 2018
Lancet Commission: Stem cells and regenerative medicine
Giulio Cossu, Martin Birchall, Tracey Brown, Paolo De Coppi, Emily Culme-Seymour, Sahra Gibbon, Julian Hitchcock, Chris Mason, Jonathan Montgomery, Steve Morris, Francesco Muntoni, David Napier, Nazanin Owji, Aarathi Prasad, Jeff Round, Prince Saprai, Jack Stilgoe, Adrian Thrasher, James Wilson
Executive summary
In this Commission, we argue that a combination of poor quality science, unclear funding models, unrealistic hopes, and unscrupulous private clinics threatens regenerative medicine’s social licence to operate. If regenerative medicine is to shift from mostly small-scale bespoke experimental interventions into routine clinical practice, substantial rethinking of the social contract that supports such research and clinical practice in the public arena will be required.
NAS_Human Genome Editing_Science-Ethics-Governance _Feb 2017_24623
Genome editing is a powerful new tool for making precise alterations to an organism’s genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions.
NAS_Gene Editing Summit Report-HK_Jan 2019_25343
Second International Summit on Human Genome Editing: Continuing the Global Discussion: Proceedings of a Workshop in Brief (2019)
On November 27-29, 2018, the U.S. National Academy of Sciences and U.S. National Academy of Medicine, the Royal Society of the United Kingdom, and the Academy of Sciences of Hong Kong convened the Second International Summit on Human Genome Editing at the University of Hong Kong. The summit brought together more than 500 researchers, ethicists, policymakers, representatives from scientific and medical academies, patient group representatives, and others from around the world. Over the two-and-a-half-day event, topics including the potential benefits and risks of human genome editing, ethical and cultural perspectives, regulatory and policy considerations, and public outreach and engagement efforts were explored. This publication summarizes the presentations and discussions from the event.


Regulatory Guidance/Commentary

Collins-Gottlieb_Next Phase Gene Therapy Oversight_NEJM_Aug 15 2018_nejmp1810628

Statement from FDA Commissioner Scott Gottlieb, M.D. on agency’s efforts to advance development of gene therapies July 11, 2018

Gene Therapy_Remarks by Commissioner Gottlieb to the Alliance for Regenerative Medicine_22 May 2018



FDA Draft Guidance for Industry – Issued July 2018