Young biotechnology entrepreneurs looking to make billions by changing their lives

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By Ellie Kincaid and Michela Tindera

^When Rachel Haurwitz began her PhD in Biology Jennifer Doudna, an award-winning biochemist at the University of California at Berkeley, suggested Haurwitz to study part of a bacterial immune system. She studied how microbes store genetic memories of virus attacks and have recognized them to combat future aggression. "It was an esoteric project," says Haurwitz. & Nbsp;

It is no longer esoteric. This system, called Crispr, has become one of the most popular technologies in biology, with the potential to give scientists control over the basics of life and give investors rich rewards. Crispr had no obvious relevance to human health when it was described in 1987, but Doudna, winner of the Breakthrough Prize in Life Sciences for his work Crispr, and other pioneers have discovered ways to transform it into a gene editing tool. Haurwitz and Doudna helped found Caribou Biosciences in 2011 to participate in the action. Haurwitz, still in his twenties, became CEO the following year. & Nbsp;

Haurwitz is not the only young entrepreneur to consider gene editing opportunities. Doudna co-founded Mammoth Biosciences with some of her other doctoral students and two PhD students from Stanford. Trevor Martin, CEO of the company, 30 years old, raised $ 23 million from investors such as Tim Cook, Apple's CEO. In 2015, in Cambridge, Massachusetts, Luhan Yang, 29, founded eGenesis with his mentor, George Church, geneticist at Harvard, use Crispr to help transplant pig organs into humans. Omar Abudayyeh and Jonathan Gootenberg, also in his twenties, co-founded Sherlock Biosciences with another Crispr pioneer Feng Zhang, 37, from the Broad Institute of MIT and Harvard.

"They may be young, but in both cases they are top-notch scientists," says Doudna of his co-founders. "They do not fear all obstacles and are very aware of ethical challenges." & Nbsp; & nbsp;

Since no one had built a Crispr company until a few years ago, "there may be more opportunities for people with no formal training," says Haurwitz.

Crispr is the acronym for "short palindromic repetitions, regularly interspersed and grouped". It refers to the way in which bacteria store viral DNA fragments in their genome, such as cup clichés. These markers are used to identify returning invaders, in the same way that a human immune system uses polio virus signaling elements stored in a vaccine.

If an invading virus matches a stored identity photo, the Crispr-associated enzymes divide the deadly DNA from the virus into harmless fragments. Doudna and others have understood how to use these enzymes to cut DNA at specific points in order to insert or modify genes. Thus, Crispr promises to facilitate the expensive and tedious process of rewriting DNA, to open up new ways to treat diseases caused by genetic mutations, to create less expensive diagnostic tests and to create cells that kill cancer.

Eight years after its launch in Berkeley, Caribou has raised $ 41 million and signed licensing agreements – potentially worth hundreds of millions of dollars – with DuPont Pioneer, Novartis and others. He begins to develop medical therapies.

Haurwitz grew up in Austin, Texas, and earned a BSc in Biology at Harvard. She did not have a clear plan when she went to UC Berkeley, but she thought she could later become a patent attorney. & Nbsp;

This thought has changed while his PhD work has become more exciting. Haurwitz and Doudna spent a lot of time talking about how they could use Crispr to modify genomes to cure diseases. Program the natural Crispr system to cut the gene you want to modify. It is theoretically possible to use it to modify the genetic code in order to correct the "misspelling" that causes the disease or to disrupt the production of an unwanted protein.

Caribou began with the idea of making Crispr technology available for DNA publishing in applications such as drug development, agriculture, and basic biological research. The co-founders of Haurwitz did not want to leave the academic world and were "crazy enough to let a 26-year-old woman, who had never worked for a company in her life, take on the role of president and general manager," he says. -she.

Haurwitz took a few business courses before getting his doctorate, and then invited venture capitalists to finance a technology they did not really understand. Caribou was granted an exclusive license on certain Crispr patents held by the University of California system and the University of Vienna. Still, "pretty much every VC we talked to sort of said," Meh, "Haurwitz recalls. It was in 2012 and they thought she overestimated the potential of Crispr. & Nbsp;

Young scientists and entrepreneurs working at Crispr at the Forbes Under 30 Summit in Boston: From left to right, Omar Abudayyeh of Sherlock Biosciences; Catherine Freije, Ph.D. candidate at the Broad Institute of MIT and Harvard; Trevor Martin from Mammoth

Jamel Toppin for Forbes.com

The newspapers that propelled Crispr into the limelight appeared the following year. Investor money and a wave of new businesses quickly followed. Editas Medicine, co-founded by Sherlock's Feng Zhang, raised $ 43 million for the application of technology to medical therapies. Then, Intellia Therapeutics, co-founded by Caribou, raised $ 15 million when it was launched in 2014. And Crispr Therapeutics, founded by Crispr pioneer Emmanuelle Charpentier, raised $ 89 million. All three companies went public in 2016 and now have a combined market capitalization of $ 3.8 billion.

Meanwhile, Haurwitz was called cold by plant breeding and pharmaceutical companies. DuPont has made an investment of $ 11 million in 2015. Caribou has raised an additional $ 30 million next year and has been able to sustain itself in terms of funding and payments from licensing and partnerships .

Caribou has granted Integrated DNA Technologies the right to sell biology researchers what they need for gene editing experiments. Genus, an animal genetics company, has paid Caribou an undisclosed amount for the exclusive right to use its patented Crispr technology for the production of pork genes and other animals. Similarly, the Jackson lab pays Caribou the use of Crispr to create new populations of research mice modeling human diseases. & Nbsp;

Haurwitz will soon have to seek venture capital again, as Caribou has started the development of expensive but potentially more lucrative medicines. Its primary goal is to improve existing cancer therapies that take patients' immune cells and lead them to attack cancer. Crispr, she says, could be used to alter the DNA of immune cells from healthy donors so that these cells can be administered to all cancer patients. The company plans to begin testing with humans next year. Allogene Therapeutics and its partner Cellectis compete for a combined market capitalization of $ 3.9 billion.

Caribou is also developing a program in another very lively region: the microbiome, or the many bacteria that populate all parts of the human body, especially the intestines. This time investors know what is Crispr and Haurwitz has already won. "This is one of the few people I've met in my life to be able to switch between business and scientific discussions," says Ambar Bhattacharyya, a Caribou investor at Maverick Ventures.

Beyond the competition, there is a conflict of intellectual property. Overlapping patent applications from the University of California and the Broad Institute have emerged for the core technology, which involves an enzyme called Cas9, used to cut DNA. A lawsuit between the institutions was decided in favor of Broad, but the US Patent Office granted patents to both. UC's patents claim broader rights than those demonstrated in its application, says Lisa Ouellette, a professor at Stanford Law School, and could make them vulnerable to legal recourse. (UC does not agree.)

Whoever owns the technology will require significant costs. Caribou may be conducting tests on a particular gene, but if other companies want to experiment with other genes, they may have to go to Caribou, says Jacob Sherkow, a professor at New York Law School. "They will have to pay handsomely."

Aside from legal battles, the new terrain risks negative public reactions. In November, the Chinese scientist He Jiankui ad He had used Crispr to tinker with the genomes of human embryos born from twins, putting additional pressure on Crispr scientists to consider ethics the way they use the technology that transforms their lives. Caribou's licensing agreements include wording to prevent its use on human embryos, says Haurwitz. & Nbsp;

According to Doudna, researchers need to look closely at the science of gene modification of embryos, and then discuss how to use them responsibly. "Are there any real unmet medical needs that would require this type of publishing or not? I think that's a question. "

The response debate will shape the path of commercialization of Crispr, a path that offers tremendous potential for its young founders – and the likelihood of even greater controversy and conflict.

By Ellie Kincaid and Michela Tindera

^WRachel Haurwitz began her PhD in Biology Jennifer Doudna, an award-winning biochemist at the University of California at Berkeley, suggested Haurwitz to study part of a bacterial immune system. She studied how microbes store genetic memories of virus attacks and have recognized them to combat future aggression. "It was an esoteric project," says Haurwitz.

Haurwitz is not the only young entrepreneur to consider gene editing opportunities. Doudna co-founded Mammoth Biosciences with some of her other doctoral students and two PhD students from Stanford. Trevor Martin, the company's 30-year-old CEO, raised $ 23 million from investors such as Apple CEO Tim Cook. In 2015, in Cambridge, Massachusetts, Luhan Yang, 29, founded eGenesis with his mentor, Harvard geneticist, George Church, with the goal of using Crispr to assist in organ transplantation. pork in humans. Omar Abudayyeh and Jonathan Gootenberg, also in their twenties, co-founded Sherlock Biosciences with another Crispr pioneer, Feng Zhang, 37, from the Broad Institute of MIT and Harvard.

"They may be young, but in both cases they are top-notch scientists," says Doudna of his co-founders. "They are fearless of all good manners and very aware of ethical challenges."

Since no one had built a Crispr company until a few years ago, "there may be more opportunities for people with no formal training," says Haurwitz.

Crispr is the acronym for "short palindromic repetitions, regularly interspersed and grouped". It refers to the way in which bacteria store viral DNA fragments in their genome, such as cup clichés. These markers are used to identify returning invaders, just as a human immune system uses evidence of a polio virus stored in a vaccine.

Haurwitz grew up in Austin, Texas, and earned a BSc in Biology at Harvard. She did not have a clear plan when she went to UC Berkeley, but she thought she could later become a patent attorney.

Caribou began with the idea of making Crispr technology available for DNA publishing in applications such as drug development, agriculture, and basic biological research. The co-founders of Haurwitz did not want to leave the university world and were "crazy enough to let a 26-year-old woman who had never worked for a company in her life take on the role of president and general manager," she says.

Aside from legal battles, the new terrain risks negative public reactions. In November, Chinese scientist He Jiankui announced that he had used Crispr to tinker with the genome of human embryos born in the form of binoculars, thus increasing pressure on Crispr scientists to think about the ethics of the way they use the technology that will transform their lives. Caribou's licensing agreements include wording to prevent its use on human embryos, says Haurwitz.

The response debate will shape the path of commercialization of Crispr, a path that offers tremendous potential for its young founders – and the likelihood of even greater controversy and conflict.