The great promise and potential perils of CRISPR.
“Everything should be made as simple as possible, but not simpler,” said Albert Einstein, who managed to distill the relationship between mass, energy, and light into an equation so brief it could be text slang. The idea that nature, for all its complexity and variability, can be boiled down to simple rules, tools, and shortcuts has long been an appealing one—to scientists, especially.
And so it is no surprise that a four-year-old gene-editing tool called CRISPR-Cas-9 has been hailed as the biotech breakthrough of the century. CRISPR (Nature has a great overview here) builds ever-so-slightly off a strategy used by bacteria to fend off repeat attacks from viruses. In the human version, scientists use an RNA guide to direct an enzyme, Cas-9, to a specific point in any organism’s DNA—where, like an eagle-eyed copy editor, the enzyme snips out an errant letter or sequence as if it were expunging a typo. Just as effortlessly, a new genetic sequence can be inserted in its place. Researchers trumpet how quick, cheap, efficient, and easily learned the technique is.
Already, scientists have made progress—in animal models and human cells, at least—in correcting the genetic flaws that lead to diseases like cystic fibrosis and Duchenne muscular dystrophy, disabling viruses such as HIV, and rewriting the DNA of mosquitoes so that they can no longer transmit malaria. Yesterday, NIH director Dr. Francis Collins devoted a blog post to CRISPR’s potential to treat, and maybe even cure, sickle cell disease, a debilitating blood condition caused by a single genetic mistake. The procedure can do everything from make animal organs safe for transplant to genetically engineering larger fish or fowl to feed the planet to supercharge T cells against cancer.
Three rival companies, founded by scientific pioneers in this field—Editas Medicine, Intellia Therapeutics, and CRISPR Therapeutics—have gone public this year, and together command a market cap of nearly $1.7 billion. And that’s where the simple ends. Humans and an array of mighty institutions—University of California, MIT, and Harvard, among them—have managed to turn the straightforward defensive reflex of a prokaryote into a bitter dispute about who owns it. For the intrepid and diehard curious, the dispute at the U.S. Patent and Trademark Office is an extraordinary lesson in the complexity of academic science. For a shorter version, check out IPStudies’s remarkable portrait of the CRISPR patent landscape.
And at last returning to dear Einstein’s aphorism, there is some strong evidence that CRISPR may be too simple at that. The wonderful Sharon Begley at STAT has a nice piece on the possible danger of this technique—beyond, of course, the obvious risks and ethical dilemmas of bioterrorism, embryo engineering, and mucking with the human germline for an eternity. It turns out that this incredibly precise molecular scissors may occasionally snip at the wrong address.
More stories below.
CRISPR heralds hope for fighting HIV and sickle cell disease. In a breakthrough with potentially major ramifications, scientists from the University of California, San Francisco and the Gladstone Institutes have used CRISPR gene-editing technology to identify specific genes that could be targeted to combat HIV infection. The process involved modifying the genomes of hundreds of thousands of healthy immune T cells and then exposing them to HIV to see which edited genes led to protection against HIV (they identified a half dozen). Still, challenges remain considering previous studies have shown HIV recurrence in genome-edited cells, and creating drugs that mimic the researchers’ meticulous process is no easy feat. Separately, National Institutes of Health (NIH) director Dr. Francis Collins heralded CRISPR’s potential lead to an “ultimate cure” for sickle cell disease in a blog post. (STAT News, NIH)
Technology is good for your healthcare. Fortune editor Alan Murray hits on the myriad ways that technology can transform a juggernaut as inefficient as the American health system—and reminds readers of our upcoming Brainstorm Health conference in San Diego next week. Speakers and panelists at the inaugural event will include GSK CEO Andrew Witty, Fitbit CEO James Park, AthenaHealth CEO Jonathan Bush, IBM’s Deborah DiSanzo, Dr. David Agus, Deepak Chopra, Arianna Huffington, and blockchain startup Gem CEO Micah Winkelspecht. And you can bet that we’ll be covering all the action in Brainstorm Health Daily. (Fortune)
Can blockchain make medical claims tracking less of a nightmare? Capital One announced Monday that it’s teaming up with a compendium of tech partners, including the blockchain startup Gem, to tackle one of the most convoluted aspects of American healthcare: medical claims tracking and analytics. The banking giant hopes that the digital ledger technology can help whip a notoriously inefficient and stratified system into shape by harnessing the algorithms at the heart of BitCoin. (Fortune)
IBM Watson Health and Teva are significantly expanding their partnership. IBM Watson and Teva Pharmaceuticals made a pair of big announcements this morning about their ongoing health partnership—a double whammy of projects that aim to find new uses for old drugs and help doctors better treat their patients who suffer from chronic illnesses like asthma. The Watson supercomputer will be used to conduct the wide-ranging analytics, and both initiatives represent a significant expansion of IBM’s existing efforts with Teva. Stay tuned for more coverage of the partnership on Fortune today.
Uber delivers free flu shots. Ride sharing titan Uber is known for its special one-off services, such as having kittens or ice cream (or, in a first, an autonomous truck loaded with beer) delivered to your office or home. On Tuesday, it delved into the realm of public health, offering flu vaccines under the UberHEALTH option for the third year running. This time, though, the service was free, and offered five vaccinations per order. (Fortune)
Pharma lobby raises dues in preparation for an epic battle over drug prices. The Pharmaceutical Research and Manufacturers Association (PhRMA), the industry’s main trade organization and lobbying arm, has hiked its member companies’ dues by 50%, which will bring the powerful group an extra $100 million per year, according to Politico. The reason isn’t hard to guess—pharma has been one of the main villains of the 2016 election cycle in the wake of massive backlash to drug makers’ price hikes on life-saving products like Mylan’s EpiPen. As I’ve previously argued, the vote that the industry will really be watching on election night is California’s Proposition 61, which would cap how much public programs in the state pay for medicines. (Politico, Fortune)
Regulators are trying to give drug makers guidance on female sexual desire drugs. The controversial female libido drug Addyi, which was snapped up from Sprout Pharmaceuticals by Valeant for $1 billion but has floundered in sales, and drug maker interest in creating alternatives has spurred the Food and Drug Administration to release new draft guidance on the proper metrics for assessing female sexual function. (Bloomberg)
The EpiPen is losing some of its market share. In an interesting twist, the number of EpiPen prescriptions are actually rising despite controversy over its skyrocketing price over the last decade—however, Mylan is beginning to lose market share in the space that it dominates. It’s not a massive drop (the share of prescriptions went from 96% to 92%), but it does indicate that more providers and patients are opting for alternatives like Adrenaclick. More competition could also be on the horizon: the maker of epinephrine injector Auvi-Q, Kaleo Pharmaceuticals, says that it plans to bring its product back onto the market in 2017. (STAT News)
Vertex moves next-gen cystic fibrosis drugs into new trials. Vertex’s Orkambi became the first approved combination therapy to treat cystic fibrosis and has even helped the biotech achieve profitability after years of losses. But the pricey therapy has a fair share of side effects which is likely dragging down its overall market potential. Now, Vertex is planning to move two experimental therapies that it hopes will mitigate those side effects while boosting Orkambi’s efficacy into mid-stage clinical trials by the end of the year. (TheStreet)
THE BIG PICTURE
Donald Trump’s latest hit on Obamacare doesn’t make any sense. Republican presidential contender Donald Trump is no fan of the Affordable Care Act, aka Obamacare. But his latest criticism of the health law is baffling. Trump claimed that “all” of his employees were being crushed under the financial weight of Obamacare due to soaring premiums. The trouble is, the Trump Organization is a massive employer that Trump has previously said provides generous health benefits (fact check: true). Large employers don’t even have access to Obamacare’s marketplaces, which sell individual insurance plans, raising the question: Why would Trump’s workers even be on these plans in the first place? (Fortune)
Novartis CEO Joe Jimenez on putting people before profits. In an interview with Fortune, the pharma giant chief executive, who has been a vocal proponent of new drug pricing models and performance-driven pricing, discusses why he thinks it’s so important to take consumers’ needs and struggles into account. (Fortune)
The AARP is suing the government over wellness program discrimination. The lobbying powerhouse for elderly Americans is suing the Equal Employment Opportunity Commission (EEOC) over new rules it issued in May which would allow employers to set massive incentives for joining wellness programs. Workers who do participate in such programs can have their health insurance premiums slashed by as much as 30%; but they must also divulge significant medical information to their employers. The AARP argues that rules discriminate against older Americans who would either have to divulge sensitive health information or, essentially, incur a significant financial penalty for non-participation. (New York Times)
SAP’s CEO is on a Mission to Reform Healthcare, Interview by Susie Gharib
Why Pediatricians Now Approve of Video Chat for Infants, by David Z. Morris
|Produced by Sy Mukherjee|
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