Human Gene Editing, CRISPR and FDA: How Will They Mix?

Regulatory NewsRegulatory News | 02 December 2015 |  By 

A number of the world’s top scientists, policy experts and bio-ethicists met this week in Washington, DC to discuss the implications of human gene editing, and though the focus of the summit was centered more on ethical considerations than regulations, opinions flew on Wednesday over how some new technologies will likely be regulated.


As Jennifer Doudna, a molecular and cell biologist at University of California, Berkeley, and a leader in the field notes in a Nature outlook published Wednesday, the latest evolution of DNA modification technology can be seen in CRISPR-Cas9 technology, which because of its simplicity and specificity, allows researchers to make precise changes to genomes and enable experiments that were previously difficult or impossible to conduct.

But, as Doudna notes, the advancement of gene editing technology is progressing more quickly than ever.

“At least one thing is clear at this stage — we do not yet know enough about the capabilities and limits of the new technologies, especially when it comes to creating heritable mutations,” she writes.

Coupled with that lack of knowledge is an urgency from patients, particularly those with genetic or inheritable diseases who envision the possibility of this technology enabling them to literally cut out defects and potentially ensure problematic genes aren’t passed down to future generations.

A lot of the discussion around human gene editing is now focusing on ethical considerations, and other big questions, such as whether there should be a brief moratorium on gene editing research, though how the world’s regulators interact and address these new technologies will also be key to ensuring the risks and benefits are appropriately addressed.


On Tuesday, four academics from the US, France and China spoke of some of the ways that countries are currently regulating human gene editing and some of the potential ways that future technologies will need to be regulated by the US Food and Drug Administration (FDA) and others.

Barbara Evans, director of the Center for Biotechnology and Law at the University of Houston Law Center, spoke of how FDA may need to make some changes to its framework in order to best regulate these gene-editing technologies.

“I think we all have doubts whether a simple ethics framework that requires informed consent and an ethics review is going to be sufficient,” Evans said, noting that researchers should take note that the agency can regulate research that isn’t commercial if there’s a significant risk for patients.

She also explained that regulators need to investigate the idea of “not just regulating the technology” but regulating specific uses of the products “because we are all concerned about off-label use.”

As far as how FDA would regulate gene editing, Evans noted that the agency could regulate it as either a drug or a device, though “there's some real advantages to framing it as a device,” particularly if the gene editing is conducted on embryos or gametes.

And if FDA classifies certain technologies as devices, the agency can “put meaningful restrictions in place on what can be done after approval,” she noted.

“The problem with all this is, I’m not sure we can agree what a gene editing catastrophe would look like,” Evans added, noting that there might be a higher risk from plant gene editing than human gene editing. “The science of regulation is more precarious than the science of gene editing.”

International Harmonization

Evans also stressed that regulatory harmonization might not be the best path forward, as countries might be able to learn from one another if they try implementing different regulations in parallel.

However, Gary Marchant, a law professor at Arizona State University, noted that uniform national regulations and requirements would assure more equal protections for citizens and discourage medical tourism.

Though Pilar Ossorio, professor of law and bioethics at the University of Wisconsin, seems to believe international harmonization might be really difficult at this point. And as Jennifer Merchant of the Universite Paris II pointed out, public engagement is going to be key (as it has been for the UK and France in the past).

“Given the diversity of regulatory frameworks and legal frameworks, relating to gene editing into stem cells and so forth, I think it is unlikely that anybody imagines there would be actual harmonization,” Ossorio noted. “So it is probably more about degrees of harmonization and it seems to me that for a lot of scientist the concern is when you are doing international scientific projects, multi-site projects that you run into conflicts of law and regulatory frameworks that make it difficult to work across jurisdictions.”

International Summit on Human Gene Editing: A Global Discussion


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