A task force of the Alliance for Regenerative Medicine (ARM) consisting of gene-editing therapy developers today sided with the emerging scientific consensus against germline gene editing in human clinical research without coming to terms with legal, ethical, and other implications—part of a statement intended to articulate a bioethical framework for use of the technology in therapeutic applications.

ARM’s Gene Editing Task Force offered its endorsement of research into therapeutic applications of somatic cell gene editing, which does not pass genetic modifications to subsequent generations—as well as its opposition to both gene editing of the germline (sperm, eggs, and fertilized embryos) for human implantation purposes, as well as implantation of a human embryo carrying gene-modified cells. The Task Force defines somatic cells to include pluripotent stem cells.

“Gene-editing technologies have not matured to the point where human trials of edited germline cells are appropriate. Many important safety, ethical, legal, and societal issues involved with this type of gene editing remain unresolved,” the Task Force stated. “Unless and until ethical and potential safety questions with respect to germline gene editing are adequately addressed, we do not support or condone germline gene editing in human clinical trials or for human implantation.”

Addressing those questions, the Task Force added, should be done by the FDA and European Medicines Agency through evolving national and regional regulatory frameworks which the panel praised as being “important to support appropriate development of these technologies and should act as the primary regulatory and enforcement mechanism.”

The panel coupled that recommendation with a warning against resolving legal and ethical issues through “arbitrary and ancillary oversight bodies or processes,” which according to the Task Force “may carry the risk of delaying research and development efforts, which in turn would adversely impact afflicted patient populations.”

In its statement, the Task Force also voiced support for the development of standards to govern gene-editing research by the National Institute of Standards and Technology’s NIST Genome Editing Consortium, U.S. Pharmacopeia, International Organization for Standardization (ISO), and other “recognized” standards development organizations. Standards should address key concepts that include off-target effects and their impact on tumor suppressors and oncogenes as well as the measurement and monitoring of genetic mosaicism, the Task Force added.

The Task Force’s full letter can be read here.

“Safe and ethical manner”

“As therapeutic developers utilizing gene-editing technologies, we must ensure that our efforts, above all else, are carried out in a safe and ethical matter,” Sandy Macrae, CEO of Sangamo Therapeutics and co-chair of ARM’s Gene Editing Task Force, said in a press release accompanying the Statement of Principles. “With our current base of knowledge this means focusing solely on approaches that do not alter the germline unless and until ethical and potential safety questions with respect to germline gene editing are adequately addressed.”

The Alliance statement was signed by Sangamo and 12 other developers of therapies based on gene editing, including: Audentes Therapeutics, bluebird bio, BlueRock Therapeutics (which Bayer earlier this month agreed to acquire for up to $600 million), Caribou Biosciences, Casebia Therapeutics, and CRISPR Therapeutics.

Also among the 12 were Editas Medicine, Homology Medicines, Intellia Therapeutics, LogicBio Therapeutics, Precision Biosciences, and Tmunity Therapeutics.

The Task Force released its statement in Geneva, where the World Health Organization (WHO)’s expert advisory committee on governance and oversight of human genome editing is meeting through tomorrow to evaluate effective measures to deter and prevent initiation of human pregnancy via genome-edited embryos. The committee has recommended against clinical applications of human germline genome editing as “irresponsible at this time”—a recommendation that the WHO accepted last month.

“Gene editing is a rapidly developing technology that represents one of the most exciting developments in medicine. These techniques will be integral to the next generation of advanced therapeutics and we welcome their potential to provide important, and potentially life-saving, treatments for patients,” ARM CEO Janet Lambert stated. “As with all breakthrough biotechnologies, we need to exercise caution and good stewardship in our research and development practices and ensure that work involving the genetic modification of cells takes place within the bioethical framework outlined in these principles.”

Calls for moratorium

The ARM Gene Editing Task Force echoes the view of the more than 60 genome editing and gene therapy luminaries who urged a “binding global moratorium” on germline editing in an April 24 letter to Alex Azar, the Secretary of the Department of Health and Human Services (HHS). That moratorium should stand, they argued, “until serious scientific, societal, and ethical concerns are fully addressed.”

In March, a group of 18 key researchers and ethicists from seven countries called for a temporary moratorium on all clinical uses of human germline editing pending the creation of an international governance framework for carrying out the research under certain conditions, in a commentary published March 13 in Nature.

The statements followed the international outrage and condemnation that emerged within the scientific community after reports emerged that Chinese researcher He Jiankui, PhD, had supervised the birth of twin girls following germline editing.

He defended his research at the second Human Genome Editing Summit, whose organizing committee issued a closing statement criticizing his research. He was fired in January by his university, the Southern University of Science and Technology (SUSTech) in Shenzhen, China, following the release of an official Chinese investigation into He’s actions, which was announced by the state-run Xinhua News Agency.

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