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.
China imprisoned the scientist, He Jiankui, for three years for violating medical regulations.
Fast forward to today: Mainstream scientific organizations are encouraging very careful basic research to explore gene-editing and human reproduction. But they still warn any attempts to create more genetically modified children anytime soon should remain strictly off limits.
And the first company to publicly announce plans to try to genetically modify human embryos to create gene-edited babies just unveiled its plans.
“We want to be the company that does this in the light, with transparency and with good intentions,” Cathy Tie, a biotech entrepreneur, told NPR in an interview about her new company, dubbed: Manhattan Project.
As for the company’s name, Tie told NPR, “We chose our name deliberately. We believe the scale of our mission, to end genetic disease, is just as significant as the original science behind Manhattan Project.” Tie said she plans to move slowly and carefully, with stringent bioethical oversight, to explore a variety of gene-editing technologies.
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.
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.
“I’m absolutely very excited about this project,” Hysolli, who worked in George Church‘s Harvard genetics lab before Colossal, told NPR in an interview. “I truly believe that these tools are very powerful and can lead to benefits to human health.”
The Manhattan Project did not reveal more details, including how much money had been raised, the investors or a timeline.
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.
His wife agrees.
“We fundamentally believe in reproductive choice and we also very much support parents’ rights to give their children every privilege they can,” Simone Collins told NPR. “And for some people, that means, obviously, eliminating risks of very dangerous diseases. But for other people that means investing in education and tutoring to make them smarter or athletically better. And if people would like to start to do that at a genetic level they should have every right to do so.”
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.
“What I think is positive is: The discussion that will be stimulated through this activity. There is clearly a need for that,” said Dietrich Egli, a Columbia University professor of developmental cell biology. He has raised questions about the safety of CRISPR embryo editing through his experiments.
Egli said he’s talked about this with Brian Armstrong, a billionaire cryptocurrency entrepreneur who recently announced interest in starting an embryo-editing company. Armstrong initially agreed to an interview with NPR through a spokeswoman but then indefinitely postponed.
The moment could be ripe for another look at gene-editing embryos that could be taken to term.
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.
Tie was briefly married to He, but Tie told NPR they recently divorced. He will have nothing to do with her new company, she said.
“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.”
“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.
“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.”
Source: www.npr.org