WASHINGTON DC – Francis Collins, MD, PhD, director of the National Institutes of Health (NIH), has reaffirmed his support for a moratorium on heritable genome editing.

Francis Collins
Francis S. Collins, MD, PhD

Speaking at a Policy Summit* hosted by the American Society of Gene and Cell Therapy (ASGCT), Collins said the future of heritable or germline genome editing was “one of the most important public debates in my 26 years at the NIH.”

Offering his full support for an international moratorium on the first anniversary of the birth of the CRISPR babies in China, Collins said, “We should proceed not at all at the moment and only with great caution in the future.”

Collins noted that the two major reports on germline editing—published by the National Academies of Sciences and Medicine in 2017 and the U.K.’s Nuffield Council on Bioethics in 2018—opened the door to the idea that germline editing could be ethically acceptable under certain circumstances, although legally, this is not permissible in the United States.

Collins also criticized the focus of the ongoing World Health Organization (WHO) commission into germline editing, co-chaired by former FDA commissioner Peggy Hamburg, which has expanded to include somatic genome editing, specifically a call to build a registry of somatic gene editing protocols. “I worry that’s a distraction,” Collins said, suggesting the WHO commission should stick to its original mandate.

Why a moratorium?

Collins gave three main reasons for a moratorium: safety, medical, and ethical. The safety concerns with current CRISPR-Cas9 technology are well known. “I wish we had more access to the two Chinese babies” to monitor their health, he said, acknowledging that was unlikely given the current political tensions between the countries.

On medical grounds, Collins asked rhetorically if there were medical needs that only germline editing could meet? In most cases, preimplantation genetic testing (PGT) would offer couples the chance of a healthy biological child, he said. While acknowledging that PGT is not an option for some rare cases, such as the hearing-loss couples identified by Russian scientist Denis Rebrikov, PhD, Collins said that was “one of the very rare circumstances I don’t think we should be using to make bad scientific decisions.”

On ethical and legal grounds, Collins highlighted consent issues, questions of justice and equal access, as well as broader philosophical and theological issues. “What does it mean to do a fully informed, societal consultation about a technology that could alter the very nature to be human? We must never allow our technology to eclipse our humanity.”

Following his talk, Collins was pressed by Neenar Nizar, president of the Jansen’s Foundation, who has a very rare genetic disorder called Jansen’s disease. Nizar is one of only two dozen patients in the world with the disease, including her two sons, and only two adults in the United States.

“Science and medicine have a responsibility to try to find answers” to rare diseases such as hers, Collins assured her. For instance, his own lab at NIH is studying potential somatic gene-editing treatments for progeria, a rare hereditary form of premature aging, which affects about 200 patients worldwide.

Addressing Nizar, he said, “The need to do germline editing for your condition, or a long list of other genetic conditions, is uncompelling to me, because it would require you to go through in vitro fertilization, at which point, for a dominant condition like yours, half of the embryos should be normal, without needing editing. So why not just implant those? We don’t need a gene editing solution.”

Collins acknowledged that this workaround was not applicable in rare scenarios, such as an individual homozygous for a dominant disorder like Huntington’s disease or the Russian hearing-loss couple being offered germline editing by Rebrikov. “OK, I’ll give you that that is a challenge,” Collins said. “But those are vanishingly rare, and again, I don’t know that we as an international society should be driven to go forward with something that has all of these profound consequences on the basis of those examples.”

Trials ongoing

By contrast, somatic gene therapy, Collins said, was one of the most exciting developments that has come along in a long time. Indeed, Keith Joung, MD, one of the pioneers of genome editing in the pre-CRISPR era and a co-founder of multiple companies including Editas Medicine and Beam Therapeutics, pointed out there were more than 30 clinical trials underway featuring genome editing. More than half (19) use CRISPR editing, the others feature zinc finger nucleases (9), TALE nucleases (5), or other platforms (2).

In terms of disease areas, almost half the trials are in oncology (16), with rare diseases (8), HIV/HPV (7), hemoglobinopathies (5), and Editas’ trial for a rare hereditary blindness (Leber’s congenital amaurosis) making up the rest.

Collins singled out the recent launch of the first genome editing trial for sickle-cell disease in the United States, a daunting ex vivo approach. “That is not for the faint-hearted, that does not scale if we’re talking about doing it on lots of people. We need to work to take early experiments and render them more accessible and more affordable,” Collins said.

In collaboration with the Bill and Melinda Gates Foundation, NIH is investing $200M over the next four years to develop an affordable gene-based cure for sickle-cell disease and HIV. “We should have as our goal getting [a treatment] to the people where the disease is the most frequent—and that’s not in the United States.”


*ASGCT Policy Summit, Washington DC; November 4-6, 2019.

Editor’s Note: The CRISPR Journal has published a special issue in October 2019 on The Ethics of Human Genome Editing.”

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