Bay Area scientist launches new company with sights on gene-edited babies
Child born from 30-year-old embryo: 'It's a miracle'
Fox Dr. Mike Cirigliano discusses the "miracle" of a child being born from a 30-year-old frozen embryo.
BERKELEY, Calif. - A UC Berkeley biochemistry PhD and gene editing expert has launched a new effort in the field of genetic engineering on human embryos as a way to prevent disease before birth.
What they're saying:
"Our mission is to determine whether the newest generation of gene editing technologies can be used safely and responsibly to correct devastating genetic conditions for future children," Lucas Harrington stated in a recent blog post announcing his new company Preventive.
Harrington received his Ph.D. in the UC Berkeley lab of Nobel Laureate chemist Jennifer Doudna. Along with her and two other scientists, Harrington co-founded Brisbane-based biotech company, Mammoth Biosciences.
He said he’s now launching his new venture, Preventive, which he called a public benefit corporation, to further research the potential of embryonic gene editing.
What we know:
Last month, as he announced the launch, he said that Preventive has raised almost $30 million from private funding.
The funding is reportedly coming from some heavy hitters in the tech world, including OpenAI CEO Sam Altman and his husband Oliver Mulherin.
Harrington also said his team included leading experts in the fields of reproductive technology, reproductive medicine and genome-editing.
SEE ALSO: Groundbreaking blood test can detect more than 50 cancers, Bay Area biotech says
"Our goal is straightforward," he wrote, "to determine through rigorous preclinical work whether preventive gene editing can be developed safely to spare families from severe disease."
Harrington acknowledged the major ethical concerns around the science and the gray areas in the regulatory process, which he said, have opened the field to potentially detrimental outcomes.
"The combination of limited expert involvement and lack of a clear regulatory pathway has created conditions for fringe groups to take dangerous shortcuts that could harm patients and stifle responsible investigation," the researchers said, adding, "Given that this technology has the potential to save millions of lives, we do not want this to happen."
Gene editing can only be used in in vitro fertilization to allow for the first step of genetic testing on an embryo.
"It requires IVF because you have to have the embryo in a dish," explained Stanford law professor Henry (Hank) Greely, a leading expert on ethical, legal, and social implications in bioscience technologies.
Once a test determines an embryo has the DNA makeup of a genetic disease, for example, like Huntington's or cystic fibrosis, scientists would then use the DNA editing technique known as Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPR, to make alterations to the DNA.
"You would need to do a lot of research with embryos in dishes, which I think is what he's planning – to do human embryos, edit them, see how well the edits take, see how well they grow for the first 14 days or longer," Greely speculated. "And in order to do this you'd have to do some research with human embryos."
Greely said if the laboratory trials are promising, the next step in Harrington’s research could be to test on non-human primates.
"I would think before you were willing to try to make a baby this way, you'd want to be sure that a whole bunch of macaque monkeys, healthy monkeys, had been born through this," though the professor stressed testing on monkeys is not the same as testing on humans, and furthermore, there are a lot of challenges involved in lab work on primates.
"Research with non-human primates is expensive, difficult, and actually dangerous. They can carry a disease that kills people, simian herpes virus. And it's a public relations nightmare to do non-human primate work," he said. "Now, monkeys aren't humans. But they're closer to humans than anything other than the great apes, and nobody's gonna want to do this in a chimp or a gorilla."
Legal to test on human embryos
As far as the legality of testing on human embryos, Greely said, "There is no statute in the U.S. that says this is illegal, that editing human embryos is illegal."
But the Stanford law professor said there are laws in each state on human embryo testing, and while legal in most states, including California, states like North and South Dakota have strict laws in place prohibiting the practice.
Clinical trials not legal
Where the federal laws would come into play, was if Preventive wanted to move into clinical trials in women.
"The FDA only has jurisdiction when you put it in people," Greely explained. "It's when you put that gene-edited embryo into a woman, with a chance of becoming itself a person, that's when the FDA has jurisdiction… And the FDA right now can't even let you try it in humans because of Congress."
‘Significant responsibility’
Harrington himself stressed that rigorous research and regulatory oversight would be required on this path forward into a territory marked with deep ethical and legal considerations.
"Intervening at this delicate stage of development presents a significant responsibility," he said, adding, "Unfortunately, to date, most scientists and regulatory bodies best equipped to evaluate these technologies have avoided this controversial field."
The biochemist pointed to the advancements made in CRISPR to support his belief that the time is ripe for further research on embryonic gene editing.
Dig deeper:
The CRISPR technique, invented by Doudna and her French colleague Emmanuelle Charpentier, is sometimes described as acting like a "find and replace" editing tool for a document. A "guide RNA" molecule leads something like a pair of molecular scissors to the target location where the scissors are then used to precisely cut the DNA to disable a gene or to replace it with new DNA, in effect correcting genetic errors that cause a disease.
Greely, who authored the book, "CRISPR People: The Science and Ethics of Editing Humans," called the technique "the greatest discovery in biosciences of this century."
And while the method is being used in gene therapy for adults, primarily in clinical trials, creating a genetically edited baby has far greater implications, beyond the legal aspects.
"If you change the embryo, you're changing every cell in that baby or adult, including the cells that become eggs and sperm," Greely explained, altering the generations that come after.
Gene therapy in adults vs. embryos
Harrington said that adults have benefited from using gene therapy to address genetic diseases. But he said, the technology could be far more impactful if implemented in the early stages of development.
"Yet despite remarkable scientific advances, this promise remains largely unfulfilled," the scientist said, adding, "Applying this kind of medicine in adults after the disease has taken hold in hundreds of billions of cells is difficult and usually cannot reverse the damage that has already occurred."
He said that editing genes to address a disease before birth could be a game changer. "It is far easier to correct a smaller number of cells before disease progression occurs, such as in an embryo," Harrington explained. "The potential benefits of addressing disease in an embryo are significant, including the possibility of greater precision, efficiency, and lower cost than gene editing in adults."
Deep ethical divide
The ethical implications are significant, with a deep divide between those who are for progress in this technology and those who believe it’s just wrong.
"Some people, including obviously Harrington, say that if it's proven safe enough," Greely said, "then maybe there are times when it's worth trying. Other people say no, we should never do it, even if it's safe, because we're not wise enough to engineer our own future."
Safety as paramount
The issue of safety, Harrington said, would be at the center of his research.
"We will not advance this technology to clinical human use if safety cannot be established through extensive research. We will not compromise safety standards to accelerate timelines," he promised in his blog, also noting that if researchers concluded that the practice cannot be done safely in humans, "that conclusion is equally valuable to the scientific community and society."
Greely appreciated Harrington’s commitment to try and advance this field safely and responsibly, but he challenged him to secure those assurances of safety from expert sources outside his team of researchers.
"That is the right answer," the law professor said. "I am glad to hear him reaffirm... Now, what I would also like to hear is not until he thinks it's safe, but until some appropriate regulatory authority thinks it's safe, because people always have a tendency to overestimate how good what they do is."
Greely said, if proven to be safe, he didn’t see anything inherently wrong with advancing this field in this direction. But he also questioned the actual need for it.
"Rather than change one embryo so it doesn't have sickle cell disease, make ten embryos and pick one that doesn't have sickle cell disease. And this is a good thing for cystic fibrosis. Come up with a cure for cystic fibrosis, so even if the baby is born with cystic fibrosis, it won't suffer from it," Greely said. "I just don't think it's gonna be necessary very often."
Super babies?
When it comes to the ethical question of genetic intervention in embryos, the discussion has often focused on the controversial idea of creating so-called "super babies," from embryos that are engineered for enhanced traits like intelligence.
Greely said he’s not worried about the implications of using the technology for that end because there is still so much unknown.
"We don't know the genes for super babies. We know the genes that cause lots of diseases. We know a lot of genes that are heavily involved in intelligence. If the gene is broken, your intelligence is extremely low. We know almost nothing about genetic variations that give you above-average intelligence," he said.
The unknowns outweighed the knowns, said Greely, who predicted we’re decades away.
"And if it's not intelligence but you're interested in math ability or music ability or any kind of behavioral stuff? Yeah, genes are involved, but it looks like thousands of genes are involved. We're nowhere close to being able to know what combination of genes will lead to the kinds of behavioral changes that we like. And some of those might also lead to behavioral changes that we don't like," the professor said. "So super babies, maybe in 50 years we'll be able to do it, but that's my grandchildren's problem."
We are a lot closer to the reality of using embryonic gene editing to help prevent disease, according to Greely.
"I think if you invested the money and resources to do a thorough job and if it turned out to be safe, to look safe, then in about ten years you'd have enough evidence that a responsible regulator, in a country where it was legal, could say, ‘Yes, you can try this in people. Yes, you can try to make a baby this way,’" the professor said.
Bay Area biotech leader
Local perspective:
He also explained there are great advantages of Harrington advancing his initiative here in the Bay Area, which has long been one of the epicenters of the world in the field of technology and bioscience.
"California in general, and the Bay Area in particular, has always been one of the leaders," the Stanford expert said. "So things are likely to happen first here. In part because we have the infrastructure, we have the researchers, we have the labs, we have the machinery."
KTVU reached out to Harrington to request an interview. He politely declined and referred us to his blog. But he did make a point to say, "It's important to clarify that our focus is exclusively on laboratory research to evaluate the safety of these technologies, and we are not involved in any clinical applications."
In his blog, he expressed cautious hope and optimism for technology.
"If proven to be safe," the Preventive founder said, "we believe preventive gene editing could be one of the most important health technologies of the century."