The party continues into the wee hours with cognac and a live jazz band, but the Doudnas—having already enjoyed audiences on the prize circuit with the emperor of Japan and the king of Spain—have an early wake-up call. Doudna’s son has to get back to California for school. For the Kavli laureates, there are more events as they head north to Trondheim to give talks at the country’s leading scientific university. Speculation is mounting that, as the scene shifts a few hundred miles to Stockholm, CRISPR will soon be crowned with a Nobel Prize. It’s only a matter of time, but it is not to be this year.
Two weeks after her excursion to Norway, Doudna was back home on the big island of Hawaii. She receives a rapturous welcome as she walks on stage wearing a traditional Hawaiian garland, or lei, featuring the red ohia lehua of the island. It’s a much more down-to-earth affair, followed by a celebratory dinner at Ken’s House of Pancakes.8 It was just another whistle-stop engagement for the scientist who, perhaps more than anyone, embodies CRISPR—the woman who literally has DNA in her name.
In the summer of 2017, U.S. senator Lamar Alexander was enjoying a fishing vacation in Canada, off the grid and only listening to the radio for the weather forecast. One day however, he happened to catch a story about CRISPR. We should have a hearing on this, he thought to himself. Luckily, as the chair of the Senate Health, Education, Labor and Pensions (HELP) committee, that was his prerogative.9
After running between U.S. Senate buildings trying to obtain my press credentials on a crisp November day, I made it to the hearing room just in time to hear Senator Alexander hailing CRISPR as “just one of the amazing discoveries that has come from basic research funded in part by the federal government.”10 But he was alarmed by “designer baby” headlines and a report from James Clapper, the former U.S. head of national security, that genome editing had been classified as a potential weapon of mass destruction.
Testifying before the HELP committee were three CRISPR experts—a CEO, a physician, and a bioethicist. “There are a few times in our lives when science astonishes us—this is one of those moments,” said Katrine Bosley, the CEO at the time of Editas Medicine, the first CRISPR biotech company to go public. There are some 6,000 known genetic diseases, Bosley testified. “What if we could repair those broken genes?” she asked. “We owe a responsibility to patients and their families.” But Bosley cautioned it would be a long road ahead. “Cures” was a big word, and Bosley, speaking on behalf of the biotech industry, didn’t want to overpromise.
Most of the Senators stayed only long enough to ask their individual questions before ducking out for more pressing business. Susan Collins, senator from Maine, came out swinging: “It would be possible for genes to be edited that could affect intelligence or athletic ability. We live in a global world; it seems that the scientific advancements have outpaced the policy in this area. How do we ensure this exciting breakthrough in gene editing is used for good by scientists in countries like China or Russia, as well as in our own country?”
I rolled my eyes and winked at science writer Emily Mullin. We’d barely started the questions and already the predictable “designer baby” trope had come up. But Jeffrey Kahn, a bioethicist at Johns Hopkins University, agreed that science advancement usually outpaces policy. “We do have robust structures for oversight that these technologies are used for purposes we intend. International dialogue is happening,” he said reassuringly. “A much smarter approach is to restrict control to allow careful responsible science to go forward, within our borders, not to push them out.”
African American senator Tim Scott, from South Carolina, wanted to know about the prospects of curing sickle-cell disease (SCD). The third witness, Stanford University physician-scientist Matthew Porteus, said he was preparing to enroll some “very brave” volunteers in a groundbreaking clinical trial. His team would harvest the patient’s stem cells, use CRISPR to edit the gene that carries the sickle-cell mutation—correcting a single letter in the three billion letters of the human genome. They would next return about a billion edited stem cells via IV to the patient. These fixed cells would find their way back to the bone marrow and reconstitute the patient’s blood. With any luck, the patient will no longer have their disease. “That’s pretty cool,” said Scott. And then he left.
Virginia senator Tim Kaine asked if CRISPR had any relevance for Alzheimer’s disease, but Bosley wasn’t so optimistic. New therapies were further out, she said, because the genetics was more complicated than say SCD. Kaine also asked about regulatory oversight. Kahn pointed to the UK, which has strict control processes that allows the country to license emerging biotechnologies. “People don’t want to go to jail for ten years” for violations, Kahn said. “We lose in multiple ways when we drive science underground.” Nor did the U.S. want to cede its leadership and competitiveness in gene editing to anyone else.
No one could have predicted that, one year later, Kahn’s hypothetical warnings would come kicking and screaming to life.
Six months later, in April 2018, the CRISPR road show landed in a most unlikely location—the Vatican. The “Unite to Cure” conference is the brainchild of Robin Smith, president of the Cura Foundation, in conjunction with the Pontifical Council for Culture. Smith’s rolodex is ridiculous: among the guests were TV personalities Dr. Mehmet Oz and CNN’s Sanjay Gupta, with cameo appearances from A-list celebrities including Katy Perry (the power of meditation) and Jack Nicklaus (stem cells). At times, the meeting felt uncomfortably like a late-night infomercial. Billionaire Ed Bosarge bought a clinic in the Bahamas and was receiving experimental injections that would cross the blood-brain barrier to combat memory loss and aging. “My goal is to be healthy and fit and playing tennis at 120,” he said with deadly seriousness.
Then there was Peter Gabriel, the first rock vocalist I ever heard (on Genesis’s Selling England by the Pound). Gabriel treated us to an unplugged concert, although he was a bit rusty as evidenced by a false start on “Solsbury Hill.” He emotionally dedicated the set to his wife Meabh, who had recovered from an aggressive form of non-Hodgkin’s lymphoma following CAR-T therapy. Later he said: “Rich people will live forever and poor people will die in their billions. The trickle-down model isn’t going to change that.”
With so many celebrities in attendance, a panel discussion between the CEOs of the three public CRISPR biotech companies was almost an afterthought. All three companies were launched by scientists at the heart of the CRISPR drama: Zhang and Doudna co-founded Editas Medicine, but Doudna quit over a patent dispute and later joined the founders of Intellia Therapeutics. Meanwhile, Charpentier launched her own company, CRISPR Therapeutics. Katrine Bosley said CRISPR was “the biggest thing to happen in biology in a generation,” moving beyond science fiction to treat genetic diseases. The CEO of CRISPR Therapeutics, Sam Kulkarni, said CRISPR had captured the imagination of millions of people because it was so easy to use. “It has completely democratized [gene-editing] technology.” Intellia’s CEO, John Leonard, said CRISPR clinical trials were imminent, and he predicted CRISPR would be the “standard of care for sickle-cell in short order.”
Bosley conceded there was a dark side to CRISPR. Technology is neither good nor bad, “it’s what we do with it,” she said. That was also the theme of a speech by Pope Francis, delivered in a private audience with a few hundred conference attendees in the stunning Audience Hall, the interior of which resembles a snake’s visage. The Pontiff spoke of the need to protect the environment and exercise caution in the application of gene editing. He acknowledged “the great strides made by scientific research in discovering and making available new cures,” especially in rare autoimmune and neurodegenerative diseases. But science, he said, has opened up new methods