Precision-editing the code of life.
Adenine. Thymine. Guanine. Cytosine. These four tiny compounds provide the basis of life. They are the “letters” that make up DNA’s code, and their various permutations can determine everything from our physical appearances to our risk of being born with a devastating disease.So it’s not surprising that being able to manipulate these chemical building blocks is one of the most exciting prospects in medicine. And thanks to a gene-editing technology known as Crispr-Cas9, it’s become a whole lot easier to do just that.
Crispr has been widely celebrated as one of the most (if not the most) groundbreaking biotech discoveries of the 21st century. That’s not to say gene editing is new (it isn’t), but Crispr simplifies the process by using molecular scissors that can be precisely targeted to snip out aberrant regions of genetic code, which can then be replaced with the correct sequences.
The technology’s possibilities are staggering—in theory, allowing medical scientists to do everything from cure genetic disorders like sickle cell disease to identify gene targets for combating HIV. Silicon Valley billionaire and cancer immunotherapy patron Sean Parker is funding the first U.S. Crispr trials in humans, which are expected to begin this year at the University of Pennsylvania and allied institutions; in March, pharma giant Allergan (agn, -0.06%) struck a $90 million deal with Crispr specialist Editas Medicine (edit, +4.21%) for access to the biotech’s experimental therapies to treat rare and serious eye diseases.
And Crispr-Cas9 isn’t even the only type of Crispr out there: On April 12, researchers at the University of Texas Southwestern Medical Center announced they had successfully paired the gene-editing tool with a different kind of enzyme, called Cpf1, to correct mutations associated with the devastating muscle-wasting disorder Duchenne muscular dystrophy. Crispr-Cpf1 could potentially prove even more promising than the Cas9 variety because the Cpf1 enzyme is smaller and can target certain genomic regions that Cas9 can’t reach.
What makes Crispr so exciting is that, thanks to its precision, the tool has opened up a world of innovation to research facilities that previously wouldn’t have been able to handle the expenses or complexities of genome editing. The possibility that different Crispr-associated enzymes may be more effective than others is fueling the scientific competition. So is the fact that Chinese scientists at Sichuan University in Chengdu launched the first-ever human Crispr trial, in a lung cancer patient last October—a milestone that American scientist Dr. Carl June predicted would “trigger Sputnik 2.0” and a “biomedical duel” in the field between China and the U.S.
The genomic revolution isn’t just a scientific one, though. It’s also regulatory. Gene-related tech has a way of putting the fear of God—or the fear of man playing God—in people. And regulators have been cautious when it comes to the field.
That’s partly what makes 23andMe’s landmark FDA victory in early April so notable. The Alphabet-backed Silicon Valley startup, valued at $1.1 billion, became the first company allowed to sell genetic tests (and accompanying health-risk reports) for 10 different diseases directly to consumers without a prescription. That includes conditions like Parkinson’s, Alzheimer’s, and celiac disease.
Victory wasn’t always assured. In November 2013, the FDA sent 23andMe cofounder and CEO Anne Wojcicki a stern warning saying that the company’s tests and health reports, which it was already selling straight to customers, were unapproved medical devices that hadn’t been cleared by the agency. The firm had to shelve many of its services as it worked to convince regulators that its genetic tests were accurate and the accompanying medical risk reports clear enough that they wouldn’t confuse or harm customers.
Now 23andMe has pulled off the kind of comeback that’s rare to see in biopharma. “The FDA has embraced innovation and has empowered people by authorizing direct access to this information,” said Wojcicki in a statement following the clearance.
Genomic technology has evolved from the stuff of science fiction to a tangible reality, with massive medical and financial implications. Just how high are the stakes? Enough that the brilliant minds behind Crispr-Cas9—University of California at Berkeley’s Jennifer Doudna, her academic partner Emmanuelle Charpentier of the Max Planck Institute for Infection Biology in Germany, and rival Broad Institute of MIT and Harvard scientist Feng Zhang—and the various biotechs affiliated with them are embroiled in an ugly, global patent spat over the rights to the tech. (Zhang and the Broad won a key intellectual-property ruling in the U.S. earlier this year, but the matter is far from settled in markets like Europe and Asia.)
And ethical concerns will continue to dog this space. The technology isn’t quite advanced enough to birth a world of “designer babies.” But even in the case of 23andMe’s home DNA kits, some question the morality of telling a customer he is at high risk for Alzheimer’s when there’s little the person can currently do about it.
No one said revolution was easy.