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Gene Hackers: The Young Biotech Entrepreneurs Looking To Make Billions By Editing Life Itself

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hen Rachel Haurwitz started her biology Ph.D. at the University of California, Berkeley, the award-winning biochemist Jennifer Doudna suggested Haurwitz investigate part of a bacterial immune system. She studied how microbes store genetic mementos of attacking viruses and recognize them to fight off future assaults. “It was an esoteric project,” Haurwitz says. 

It’s esoteric no more. This system, called Crispr, has become one of the hottest technologies in biology, with the potential to give scientists control of the building blocks of life and give investors rich rewards. Crispr had no obvious relevance to human health when it was first described in 1987, but Doudna, who won the Breakthrough Prize in Life Sciences for her Crispr work, and other pioneers have discovered ways to turn it into a gene-editing tool. Haurwitz and Doudna helped found Caribou Bio­sciences in 2011 to get in on the action. Haurwitz, still in her 20s, became CEO the next year. 

Haurwitz is not the only young entrepreneur who sees opportunity in gene editing. Doudna cofounded Mammoth Bio­sciences with some of her other doctoral students and two Stanford Ph.D.s. Trevor Martin, the company’s 30-year-old CEO, has raised $23 million from such investors as Apple CEO Tim Cook. In 2015, in Cambridge, Massachusetts, 29-year-old Luhan Yang founded eGenesis with her mentor, Harvard geneticist George Church, to use Crispr to help transplant pig organs into people. Omar Abudayyeh and Jonathan Gootenberg, also in their 20s, cofounded Sherlock Biosciences with another Crispr pioneer, 37-year-old Feng Zhang of the Broad Institute of MIT and Harvard.

“They may be young, but in both cases these are people at the top of their game scientifically,” Doudna says of her cofounders. “They’re fearless in all the right ways and very aware of the ethical challenges.”   

Given that no one had built a Crispr company until a few years ago, “there’s maybe more of an opportunity for people with nontraditional backgrounds,” Haurwitz says.

Crispr is an acronym for “clustered regularly interspaced short palindromic repeats.” It refers to the way bacteria store, in their genomes, snippets of viral DNA, like mug shots. Those markers are used to identify invaders that return, much as a human immune system uses telltale elements of a polio virus remembered from a vaccine.

If an invading virus matches a stored mug shot, enzymes associated with Crispr break the virus’ lethal DNA into harmless pieces. Doudna and others figured out how to use those enzymes to snip DNA at precise points in order to insert or modify genes. Thus does Crispr promise to make the expensive and buggy process of rewriting DNA easier, opening up new ways to treat dis­eases caused by genetic mutations, create cheaper diagnostic tests and engineer cells that kill cancer.

Eight years after its start in Berkeley, Caribou has raised $41 million and cut licensing deals—potentially worth hundreds of millions of dollars—with DuPont Pioneer, Novartis and others. It’s starting to develop medical therapies.

Haurwitz grew up in Austin, Texas, and earned a bachelor’s degree in biology at Harvard. She didn’t have a clear plan when she went on to UC Berkeley, but she thought she might later become a patent attorney. 

That thinking changed as her Ph.D. work got more exciting. Haurwitz and Doudna spent a lot of time talking about how they could repurpose Crispr for modifying genomes to cure disease. Program the naturally occurring Crispr system to cut the gene you want to modify, and it’s theoretically possible to use it to change the genetic code to either fix “misspellings” that cause illness or disrupt the production of an unwanted protein.

Caribou started out with the notion of making Crispr technology available for DNA editing in applications such as drug development, agriculture and basic biological research. Haurwitz’s cofounders didn’t want to leave academia and were “crazy enough to let a 26-year-old who had never worked for a company in her life take on the role of president and CEO,” she says. 

Haurwitz took a few business classes before getting her Ph.D., then pitched venture capitalists on funding a technology they didn’t really understand. Caribou was securing an exclusive license to some Crispr patents held by the University of California system and the University of Vienna. Still, “pretty much every VC we ­talked to kind of said, ‘Meh,’ ” Haurwitz remembers. This was 2012, and they thought she was overestimating Crispr’s potential. 

The papers that propelled Crispr into the limelight came the next year, and investor dollars and a wave of new companies quickly followed. Editas Medicine, cofounded by Sherlock’s Feng Zhang, raised $43 million to apply the technology to medical therapies. Next was Intellia Therapeutics, cofounded by Caribou, which raised $15 million in its 2014 launch. And Crispr Therapeutics, founded by Crispr pioneer Emmanuelle Charpentier, raised $89 million. The three went public in 2016 and now have a combined market capitalization of $3.8 billion.

Meanwhile, Haurwitz was being cold-called by plant-breeding and drug companies. DuPont led an $11 million investment in 2015. Caribou raised another $30 million the next year and has been able to sustain itself on that funding and payments from licensing and partnership deals.

Caribou licensed to Integrated DNA Technologies the right to sell biology researchers what they’d need for gene-editing experiments. Genus, an animal genetics firm, paid Caribou an undisclosed amount for the exclusive right to use its proprietary Crispr technology to engineer the genes of pigs and other livestock. Similarly, the Jackson Laboratory is paying Caribou to use Crispr to engineer new populations of research mice that model human diseases. 

Haurwitz will soon have to seek venture capital again, as Caribou has pivoted to drug development, which is expensive but potentially more lucrative. Her first focus: improve on existing cancer therapies that take patients’ immune cells and train them to attack cancer. Crispr, she says, could be used to edit the DNA of immune cells from healthy donors so that these cells could be given to any cancer patient. The company plans to start trials in humans next year. There’s competition, from Allogene Therapeutics and its partner Cellectis, which have a combined market cap of $3.9 billion.

Caribou is also developing a program in another buzzy area: the microbiome, or the many bacteria that inhabit all parts of the human body, particularly the gut. This time, investors know what Crispr is, and Haurwitz has already won some over. “She’s one of the few people that I’ve met in my life that is able to toggle between business talk and scientific talk in a heartbeat,” says Ambar Bhattacharyya, a Caribou investor at Maverick Ventures.

Beyond the competition, there is an intellectual property conflict. Overlapping patent claims from the University of California and the Broad Institute emerged for the foundational technology, which involves an enzyme called Cas9, used to cut DNA. A lawsuit between the institutions was decided in favor of the Broad, but the U.S. Patent Office has granted patents to both. UC’s patents claim broader rights than were demonstrated in its application, says Lisa Ouellette, a Stanford Law School professor, and could make them vulnerable to a legal challenge. (UC disagrees.)

Whoever owns the technology will command fat fees. Caribou might run trials related to a particular gene, but if other companies want to run trials related to other genes, they may have to approach Caribou, says Jacob Sherkow, a professor at New York Law School. “They’re going to have to pay handsomely.”

Legal battles aside, the new field risks public backlash. In November, Chinese scientist He Jiankui announced he’d used Crispr to tinker with the genomes of human embryos born as twin girls, thereby heightening pressure on ­Crispr scientists to consider the ethics of how they’re using the life-altering tech. Caribou’s license agreements include language to prevent its use on human embryos, Haurwitz says. 

Doudna says researchers need to vet the science of editing the genes of embryos, and then people need to discuss how to use it responsibly. “Are there real unmet medical needs that would require this kind of editing or not? I think that’s one question.”

Debate over the answer will shape Crispr’s path to commercialization, one that holds immense potential for its youthful founders—and the likelihood of yet more controversy and conflict.

-Michela Tindera;Forbes Staff

-Ellie Kincaid;Forbes Staff

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Apple Is Donating 9 Million Masks To Combat The Coronavirus

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Topline: Apple will donate 9 million N95 protective masks to combat the coronavirus, Vice President Mike Pence said on Tuesday, making Apple one of several California tech companies pitching in as hospitals across the country report a shortage of protective gear.

  • Pence thanked Apple for agreeing to donate 9 million N95 respirator masks to healthcare facilities across the country during a press briefing on Tuesday.
  • Pence’s remarks come after Apple CEO Tim Cook tweeted over the weekend the company was “working to help source supplies for healthcare providers fighting COVID-19” and “donating millions of masks for health professionals in the US and Europe,” but did not offer more specifics.
  • N95 respirators are masks that form a protective seal around a wearer’s mouth, filtering  out at least 95% of particles in the air, according to the Centers for Disease Control, which makes them necessary to protect healthcare workers from being exposed to the disease from patients.
  • Facebook has also said it is donating its stockpile of 720,000 masks purchased during the California wildfires last year, which degraded the air quality in the San Francisco Bay Area.
  • Apple did not immediately respond to a request for comment from Forbes asking if all of the donated masks were stockpiled because of the wildfires or if the company got them from somewhere else.

Chief critic: Teddy Schleifer, a reporter at Recode, wrote that health systems shouldn’t rely on the generosity of big tech companies to make up for the failures of the federal government. 

“But there is a risk in relying on corporate philanthropy—rather than the government—in solving this problem. For starters, it depends on the voluntary generosity of these companies to deal with an unprecedented emergency, an altruism that could vanish at any time,” he wrote.

Crucial quote: “And I spoke today, and the president spoke last week, with Tim Cook of Apple. And at this moment in time Apple went to their store houses and is donating 9 million N95 masks to healthcare facilities all across the country and to the national stockpile,” Pence said.

Key background: Apple is one of several California tech companies to give away N95 masks. In addition to Facebook, Salesforce, Tesla and IBM have also announced mask donations.

News peg: Doctors and nurses are sounding the alarm that they don’t have enough masks to protect healthcare workers. Not only does inadequate protective gear put important frontline health workers at risk, public health experts say, any situation endangering medical personnel may only further depletes the U.S. health system which already doesn’t have enough capacity to handle a surge in cases. State officials in New York and Illinois have criticized President Donald Trump for not stepping in to force companies to manufacture masks or allocate masks from private companies to ensure that states don’t outbid each other for the same supplies.

Rachel Sandler, Forbes Staff, Breaking News

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Video Games Are Being Played At Record Levels As The Coronavirus Keeps People Indoors

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Topline: With school closures, mandatory work-from-home policies and lockdowns taking place in the U.S. as a result of the Covid-19 coronavirus pandemic, gaming has seen higher engagement, especially over this past weekend.

  • Steam, the most popular digital PC gaming marketplace, reached new heights Sunday, drawing a record 20,313,451 concurrent users to the 16-year-old service, according to third-party database SteamDB.
  • Counter-Strike: Global Offensive, released by Steam-owner Valve in 2012, seems to be the top beneficiary of the increased engagement, breaking it’s all-time peak on Sunday with 1,023,2290 concurrent players, topping its previous peak last month by a million, which itself beat the record set in April 2016.
  • Like other esports, CS:GO has had to cancel events due to the virus, particularly the Intel Extreme Masters in Katowice earlier this month, though its peak viewership reached over a million, making it one of the most watched tournaments in the esports’ history.
  • Activision Blizzard’s new free-to-play battle royale spinoff Call of Duty: Warzone, launched March 10 on PC, Xbox One and PlayStation 4, is also likely benefiting, drawing in a staggering 15 million in three days, besting the record 10 million in three days by last year’s battle royale sensation Apex Legends.
  • These new heights follows similar effects of the virus on China and Italy: Telecom Italia’s CEO told Bloomberg it saw a 70% increase in traffic over its landline network, with Fortnite playing a significant part, while Chinese live-streaming service Douyu experienced increased viewership of the country’s most popular games, according to market analyst Niko Partners.
  • While gaming was considered “recession proof” during the 2008 market crash, stocks aren’t immune to the current historic drops: software developers like Activision Blizzard are facing a 9% decrease in price year-to-date, while hardware companies that rely on Chinese manufacturing like Nintendo are seeing bigger drops of 24%.

What To Watch For: If these records keep rising as the closings and lockdowns continue. Arriving this week is Nintendo’s long-awaited Animal Crossing: New Horizons for the Switch console, a relaxing “life-simulator” that’s set to have a big day with many fans not-so-jokingly asking Nintendo to launch early.

Surprising Fact: Plague Inc., a game that tasks players in creating a virus that wipes out humanity, surged in popularity late January, becoming the top-paid game on the Chinese app store at one point, but the game has now been removed in China at the direction of the government.

Further Reading: So You’re Suddenly Working From Home And Want To Try Gaming? Here’s How To Get Started.

Matt Perez, Forbes Staff, Innovation


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Amazon Hoping To Hire 100,000 New Employees To Deal With Coronavirus Demand

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Topline: Amazon announced Monday that it would be opening 100,000 new full-time and part-time positions to deal with increased buying demand as people practice social distancing during the Covid-19 coronavirus pandemic.

  • The company will also increase pay by $2 in the U.S. from its current $15 an hour, £2 in the UK and €2 in Europe for those working in fulfillment centers, transportation services, stores or people making deliveries, amounting to a total of $350 million.
  • Amazon last Friday shared that the increase in online commerce has unsurprisingly resulted in shortages for household essentials and delays in shipment times.
  • Monday’s statement also noted that “We continue to consult with medical and health experts, and take all recommended precautions in our buildings and stores to keep people healthy. We’ve taken measures to promote social distancing in the workplace and taken on enhanced and frequent cleaning, to name just a few.”
  • Last week, Amazon told all of its employees to consider working from home if they could, according to CNBC; for its fulfillment centers and delivery services, it also launched a $25 million relief fund that lets workers diagnosed with the coronavirus apply for  grants equal to two weeks pay, as well as unlimited unpaid time off for all hourly employees until the end of March.
  • Amazon currently employs 250,000 people at 110 fulfillment centers.

News Peg: According to Johns Hopkins, 181,200 people have been infected with the coronavirus, with 7,115 deaths reported. School closures, lockdowns and curfews have been put in place to promote social distancing, with the White House today recommending to avoid groups of more than 10 people.

Matt Perez, Forbes Staff, Games

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