Cathy Tie, founder and CEO of Origin Genomics, replenishes cell-sustaining nutrients that the New York startup uses to test its technology for repairing faulty genes.
Mackenzie Calle for NPR
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Cathy Tie puts on a white coat bearing her name and the logo of her startup: Origin Genomics. “To welcome!” she says as she leads an NPR reporter, photographer and videographer on an exclusive tour of Origin’s New York City labs. Tie, Origin’s CEO, asked NPR not to disclose the exact location for security reasons.
“We’re going to the lab,” she said.
Once inside, Tie puts on a pair of blue rubber gloves and points to a gray metal tank on the floor that looks like a giant thermos.
“It’s a cryogenic storage unit containing liquid nitrogen, where our embryos are stored,” she explains.
Attach the black lid to open the black lid, letting wisps of white mist from the liquid nitrogen swirl out of the tank. Tie glides smoothly on a long metal rod cradling frosted glass tubes at the end.
“As you can see, there are four tubes here,” Tie says. “Each tube can hold up to 10 embryos.”
Tie removes frozen human embryos carrying disease-causing mutations from a cryogenic storage unit at Origin Genomics’ new labs.
Mackenzie Calle for NPR
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These embryos were donated by couples whose families have suffered from generations of life-threatening genetic diseases. Later, a wife and husband who donated some of these embryos tell us about their dream of having healthy children through gene editing.
But first, Tie explains how Origin scientists extract stem cells from embryos to test the company’s experimental gene editors to see which of the molecular tools could safely correct genetic defects, preventing future generations from suffering the same fate.
Using genetically modified embryos to create babies is against US law
Tie wants to convince the world that the benefits of using his company’s advanced gene-editing technology to fight hereditary diseases outweigh the risks.
Editing genes in human embryos that would be passed down from generation to generation has long been taboo due to ethical and safety concerns. In the United States, it’s illegal.
She says it might be time to reconsider.
“Hundreds of millions of people around the world suffer from these diseases: Huntington’s disease, cystic fibrosis, hereditary cancers, Tay-Sachs,” Tie explains.
“What we are proposing is something completely revolutionary: applying this astonishingly precise genome editing to embryos that have a mutation before they are born,” says Tie. “This allows the next generation to not be born with these diseases. It changes the entire paradigm of medicine as we know it.”
Origin is also testing gene editors designed with the help of artificial intelligence on other cell types carrying disease-causing mutations. These cells include induced pluripotent stem cells, which scientists create in the laboratory from adult skin or blood cells, and other cells called fibroblasts.
Tie leads us into another room to show us this work, including cells carrying a breast cancer mutation that Origin is trying to repair.
She carefully removes a clear plastic tray from an incubator and places it under a microscope that projects images of the cells onto a monitor.
“What you see here is the gene editor actually getting into the cells,” she says.
Tie examines cells that carry a genetic mutation that increases the risk of breast cancer and that have been injected with one of Origin’s experimental gene editors.
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The cells look like fluorescent green spots. Those that glow a lighter green indicate that the gene editor has successfully entered the cell, she says.
“This means that we now have a very good platform to introduce our proprietary gene editors into these cells to then correct the mutation,” says Tie. “You’re looking at the very first step.”
Concerns about risks and ethics weigh on the field
Indeed, Origin is only just getting started, and at least one other American startup is doing this sort of thing, Preventive in San Francisco. But the very existence of these companies is striking, given the scale of the problems raised by the prospect of creating genetically modified babies and the controversial history of the field.
He Jiankui, a Chinese scientist, was jailed for three years after stunning the world by announcing in 2018 that he had used CRISPR gene editing to create the first genetically modified babies.
At that time, the technology was considered far too new for anyone to know whether it was safe for babies – or for future generations. Genetic modification could have inadvertently created dangerous mutations that could then be passed down from generation to generation.
Most scientists believe it is still premature to try to use genetically modified human embryos to produce babies – and it may never be safe. But newer, more precise gene editing techniques, such as master editing and base editing, have improved the accuracy of the technology.
Some scientists and bioethicists say these advances raise hope that it might one day be ethical to use gene editing to prevent certain devastating genetic diseases.
But already, Silicon Valley venture capitalists, East Coast entrepreneurs and others have emerged to try to advance this field.
Yet even among those who believe that edited human embryos might one day be safe enough to be used in very limited circumstances to prevent rare diseases, some worry about Origin Genomics.
“It gives people false hope,” says Robin Lovell-Badge, a biologist at the Francis Crick Institute in London. “And I worry that they will rush too fast and make mistakes, which will then cause the pack to fall back – and the hope that people can have these techniques and possibly help them will be lost.”
Could gene editing be used for designer babies?
Even if editing human embryos could prove safe, some worry that allowing it could open the door to designer babies and a dystopian future of genetically enhanced people.
“I’m very concerned about where this might go,” says Katie Hasson, who directs the Center for Genetics and Society, a watchdog group. “Some of the Silicon Valley investors behind these companies are very open about their vision of the future. It’s a vision of mass-produced, genetically engineered humans that is horrifying.”
Other companies already test embryos for traits like IQ and height, in addition to disease risk.
Working in the private sector worries some university researchers. “I just don’t think they’re being honest with us about why they’re actually doing this,” says Fyodor Urnov, who studies gene editing at the University of California, Berkeley. “They are really about improving the embryos – and therefore, ultimately, the babies.”
Tie says that’s not the case for Origin, emphasizing that it’s not interested in anything other than helping people have healthy children.
Some also fear that the advent of these companies could lead to the emergence of unregulated clinics abroad to offer embryo editing where regulations are more relaxed.
“I’m very concerned about genome editing tourism,” says R. Alta Charo, professor emeritus of law and bioethics at the University of Wisconsin-Madison, who now works as a consultant to government agencies and private companies. “That’s my fear for the future.”
Tie’s work is attracting greater scrutiny because she was personally involved with the famous Chinese scientist after his release from prison.
But for his part, Tie claims that the rogue scientist is in no way involved in Origin. She promises that she is moving very slowly and methodically, with independent oversight and transparency to ensure that embryo editing is safe.
“This is a very powerful technology. Safety must come first,” she says.
Tie hopes that Origin will produce enough evidence to prove that it would be safe to use genetically modified human embryos for reproduction.
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“I think if you have a technology that can be proven to be safe and can help hundreds of millions of people who suffer from these diseases, it’s…
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