Gene-editing human embryos to prevent disease

A Chinese scientist horrified the world in 2018 when he revealed he had secretly engineered the birth of the world’s first gene-edited babies.

His work was reviled as reckless and unethical because, among other reasons, gene-editing was so new and the technology’s full risks were unknown.

China imprisoned the scientist, He Jiankui, for three years for violating medical regulations.

Now, however, Silicon Valley venture capitalists, futurists, East Coast entrepreneurs, and pronatalists — who fear falling birth rates pose an existential threat to the human race — are eager to push the technology forward. And that’s kindling both great hopes and intense fears.

Fresh interest from private companies

“You’ve got a convergence of people who are thinking that they can improve their children — whether it’s their children’s health, or their children’s appearance, or their children’s intelligence, along with people who are comfortable using the newest technologies and people who have the money and the chutzpah — the daring — to try and do this,” said R. Alta Charo, a University of Wisconsin professor emerita, lawyer and bioethicist, who’s now consulting with government agencies and private companies.

U.S. regulations prohibit editing genes in embryos that could become babies. But that could change, given the Trump administration’s deregulatory stance and support for reproductive technologies like in vitro fertilization, some observers say.

And the first company to publicly announce plans to try to genetically modify human embryos to create gene-edited babies just unveiled its plans.

“I think the timing is right for having this conversation,” Tie said. “There’s a lot of promise in this technology.”

A small scientific team has already been assembled to conduct methodical experiments in a Manhattan lab. The team plans to start by studying mice before moving to primates and then human cells before ultimately working with human embryos.

The company hopes to produce enough evidence to persuade federal officials to fund the research and regulators to approve moving ahead, she said.

“Right now the goal is really just to inform regulators and the public what this technology is capable of — and what it’s not — and hopefully empower regulators in the future, when proven safe and efficient, to allow research in this space,” Tie said. “We hope to support that regulatory approval process.”

Safety is “first and foremost,” she said.

Her ultimate goal, she said, is to prevent serious genetic diseases.

“There are so many diseases that have no cures and there’s not going to be a cure for them for many more decades,” Tie said. “And I think that we have the responsibility to talk about this with patients that do have these terrible diseases and see if they want the option to not pass that on to future generations. Parents should have the choice.”

But the company would not go beyond preventing illnesses, such as the genetic lung disease cystic fibrosis and the inherited blood disorder beta thalassemia, she said.

“Our focus is on disease prevention,” she said. “We draw the line at disease prevention.”

She co-founded the firm with Eriona Hysolli, who headed biological sciences at Colossal Biosciences, which is working on a controversial project to use gene-editing to bring back extinct animals like the wooly mammoth.

The Manhattan Project did not reveal more details, including how much money had been raised, the investors or a timeline.

Investors see an opportunity

But the company is hardly alone.

“We are definitely evaluating whether it makes sense to actually incubate and help build a company that we think could do this safely and responsibly,” said Lucas Harrington, who co-founded SciFounders, a San Francisco venture capital firm. “I think there’s huge benefit if it can be done safely and responsibly.”

Harrington envisions using newer and hopefully safer gene-editing techniques, such as “base editing,” to modify embryos to make babies. He said his focus too would be on preventing diseases.

The Chinese scientist used the gene-editing technique known as CRISPR, which allows scientists to make very precise changes in DNA much more easily than ever before but can cause potentially dangerous random mutations.

“I think how we’ve been going about it until now has really been burying our head in the sand and not wanting to talk about it because it’s too controversial,” Harrington said. “The tools over the past decade have dramatically changed.”

Others, however, talk about using cutting-edge genetic research to go further than eliminating illnesses before they start.

At least some investors in cutting-edge reproductive technologies agree.

“People can say, ‘Well, you’re playing God by using this type of technology.’ And I say, ‘People would say that with any technology of the past,’ ” said Malcolm Collins, a self-described pronatalist. Collins and his wife, Simone, said they’re supporting a variety of experimental reproductive technologies, ranging from “artificial” wombs and laboratory-made embryos to gene-edited babies.

Some futurists call these “Gattaca Stack” technologies, referring to the 1997 film about genetically engineered people, that could transform human reproduction. Pronatalists hope these developments will help counter declining births.

His wife agrees.

Room for painstaking science

Many scientists favor carefully exploring the editing of DNA in human sperm, eggs and embryos to learn more about human reproduction and possibly someday prevent diseases. And some U.S. scientists working in this field are glad to see private players helping what they consider underfunded research.

The National Institutes of Health “doesn’t typically support embryo research. So if the technology bros are interested, that would be welcome in the field,” said Dr. Paula Amato, a professor of obstetrics and gynecology at the Oregon Health & Science University in Portland. She has been working on embryo editing with her colleague Shoukhrat Mitalipov.

Amato and others stress, however, that whoever is working on this has to first make sure it can be done safely and should focus, at least initially, on preventing disease.

The moment could be ripe for another look at gene-editing embryos that could be taken to term.

“There’s a president who has some advisers and some political forces whispering in his ear that have a decidedly pronatalist bent that are interested in these technologies,” said L. Glenn Cohen, director of the Harvard Law School’s Center for Health Law Policy, Biotechnology and Bioethics. “All of that is opening up a moment where some of what would have been unthinkable may now become possible.”

There’s also talk about trying this technology in places like Prospera, a city on an island off the coast of Honduras. Prospera has looser regulations for business involved in fields ranging from cryptocurrency to biotechnology.

Bioethicists warn the risks are concerning

The emphasis on charging ahead worries many observers.

“Move fast and break things has not worked very well for Silicon Valley in health care,” said Hank Greely, a Stanford University bioethicist. “When you talk about reproduction, the things you are breaking are babies. So I think that makes it even more dangerous and even more sinister.”

This new push comes as He Jiankui, the CRISPR babies scientist, has shifted from repentant to defiant since being released from prison.

“AI is threatening humanity, we must fight back by gene editing,” he recently wrote on X.

“The nature of my relationship with him was personal, not professional and I’m also no longer married to him. He is not involved,” Tie said. “I wish him all the best.”

Nevertheless, all the renewed interest has contributed to anxiety among opponents of gene-edited babies.

“I do think this is a dangerous moment,” said Ben Hurlbut, a bioethicist at Arizona State University who recently helped organize an international meeting on inheritable human gene-editing.

“Just because you can do it doesn’t mean you should do it,” he said. “Do we need to tell us ourselves again that we shouldn’t go there?”

Others agree.

“Human heritable gene editing is clearly a terrible solution in search of a problem,” said Tim Hunt, chief executive officer at the Alliance for Regenerative Medicine, which along with the International Society for Cell & Gene Therapy and the American Society of Gene & Cell Therapy recently called for a 10-year moratorium on inheritable gene-editing. “If you make a mistake, the mistake passes onto all future generations. So that’s a pretty big ethical roll of the dice.”

Many critics argue that this movement is today’s version of eugenics, the long-discredited pursuit of supposedly genetically superior people.

“I think we should be deeply worried about this,” said Francoise Baylis, a bioethicist and professor emerita at Dalhousie University in Canada. “This is a continuation of the eugenic project that has been sort of in vogue at different times throughout civilization. This is just the modern incarnation of that idea.”

Others fear turning human reproduction into just another consumer product.

“We’re going to mass-produce genetically engineered human beings. And I think that’s a very dangerous way to approach these technologies,” said Katie Hasson, the associate director of the Center for Genetics and Society, a genetics technology watchdog group in Berkeley, Calif. “I’m very worried that all of this together means we’re headed straight into a new era of high-tech, market-based eugenics.”

But the Manhattan Project’s Hysolli argues it would be unethical not to use the technology if it’s safe.

“If we have the tools to prevent a disease that will be passed down for generations, is it more ethical to do it or not do it?” Hysolli said. “I would argue it would be more ethical to stop the mutation.”