Genetic hackers: How young biotechnologists want to make billions on life editing

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UNITED STATES (VOP TODAY NEWS) — The system called CRISPR has become one of the most relevant technologies in biology. Startups focused on its use have been the focus of investors.

When Rachel Haurwitz began to write her PhD thesis in biology at the University of California at Berkeley, biochemist Jennifer Doudna invited her to research part of the bacterial immune system. Haurwitz studied how microbes store virus gene fragments and recognize them to repel attacks in the future. Haurwitz herself calls this project esoteric.

Now – no esoteric. The system called CRISPR has become one of the most relevant technologies in biology. It is able to give scientists control over the “building blocks of life itself” and bring great profits to investors. When CRISPR was first described in 1987, it was not directly related to human health.

However, Dudna and her colleagues found a way to turn the system into a tool for editing genes. In 2011, Haurwitz and Dudna participated in the creation of Caribou Biosciences. Haurwitz, who was not even 30 at the time, became the company’s general director the following year.

Haurwitz is not the only young entrepreneur who is interested in gene editing. Dudna and her colleagues at Stanford founded Mammoth Biosciences, which raised $ 23 million from investors such 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 in transplanting pig organs to humans. Omar Abudayyeh and Jonathan Gutenberg, who are not even 30 years old, founded the company Sherlock Biosciences.

– Virus-criminals –

CRISPR is short for “short palindrome repeats, regularly arranged in groups.” Bacteria store viral DNA fragments in their genomes, as if they were photographs of criminals. These markers are used to identify the virus, just as the human immune system uses polio virus control fragments derived from a vaccine.

If the virus coincides with the saved “photo of the criminal”, the enzymes associated with CRISPR break the death-carrying DNA of the virus into harmless parts. Dudna, along with other scientists, has figured out how to use these enzymes to cut DNA at specific points to change genes.

Thus, CRISPR promises to simplify the process of DNA rewriting, which is expensive and troublesome, opening up new ways of treating diseases caused by genetic mutations, creating cheaper diagnostic tests and cells that kill cancer.

Eight years after the launch in Berkeley, Caribou raised $ 41 million and entered into licensing deals (which can bring hundreds of millions of dollars) with DuPont Pioneer, Novartis and others.

– Business from PhD –

Haurwitz grew up in Austin, Texas and received a bachelor’s degree in biology from Harvard. She didn’t have a clear plan when she went to the University of California at Berkeley, but she thought she might become a patent attorney once.

The more exciting her Ph.D. turned out to be, the more the plans changed. Haurwitz and Dudna had a long discussion about how they could use CRISPR to change genomes and cure diseases.

If you program the CRISPR system so that it cuts the gene that needs to be changed, then it is theoretically possible to use it to change the genetic program to either correct the “shortcomings” that cause the disease or disrupt the production of unwanted protein.

Caribou emerged from the idea of ​​adapting CRISPR technology for DNA editing (it can be used in drug development, agriculture, basic biological research). The founders of Caribou did not want to leave academic science and were “crazy enough to allow a 26-year-old girl who had never worked for the company to take on the role of president and CEO,” says Haurwitz.

Caribou then received an exclusive license for some CRISPR patents owned by the University of California and University of Vienna. Haurwitz offered to finance a startup for venture investors, but almost no one showed interest in the project.

However, as early as next year, scientific papers appeared that increased interest in CRISPR. Soon followed the money from investors. Editas Medicine, created by Feng Zhang from Sherlock Biosciences, raised $ 43 million to apply this technology in therapy.

The next was Intellia Therapeutics, founded by Caribou, which in 2014 raised $ 15 million. And CRISPR Therapeutics, created by one of the pioneers of the technology Emmanuel Charpentier, collected $ 89 million. All three companies became public in 2016, and now their total market value is $ 3,8 billion

Meanwhile, the Haurwitz began to call from breeding and pharmaceutical companies. In 2015, DuPont invested $ 11 million in the company, and next year Caribou raised another $ 30 million.

Genus (a company working in the field of animal genetics) paid Caribou for the exclusive right to use its patented technology to work with genes from pigs and other livestock. Jackson Laboratory pays Caribou for using CRISPR to create new populations of mice that simulate human diseases.

Soon, Haurwitz will again have to seek capital. The fact is that Caribou is developing drugs – a costly, but potentially more profitable business. The primary goal is to improve existing cancer treatments. CRISPR, according to Haurwitz, can be used to edit the DNA of immune cells from healthy donors so that these cells can be transferred to any cancer patient. The company plans to begin human trials next year.

In addition, Caribou is developing a program in another “hot” area: it is a microbiome, or a multitude of bacteria that inhabit all parts of the human body, especially the intestines. This time, investors know what CRISPR is, and Haurwitz has already attracted some of them to their side.

“She’s one of the few people I’ve met in my life, who can instantly switch from business to science,” says Ambar Bhattacharyya, an investor in Caribou from Maverick Ventures.

– Ethical issues –

The area in which Caribou operates is characterized not only by competition, but also by conflicts in the field of intellectual property. Overlapping patent applications from the University of California and the Broad Institute were filed for the underlying technology, which includes the Cas9 enzyme used to make a cut in the DNA.

The lawsuit between institutions was decided in favor of the Broad Institute, but the US Patent Office granted patents to both.

Those who own this technology will receive a huge profit. Caribou may conduct tests related to a particular gene, but if other companies want to conduct tests related to other genes, they may have to turn to Caribou, says Jacob Sherkow, a professor at New York Law School. “They will have to pay generously.”

In addition to legal battles, a new field of research can cause an ambiguous public reaction. In November, Chinese scientist He Jiankui announced that he had used CRISPR to edit the genomes of human embryos – twin girls – thereby prompting the public to put more pressure on scientists to develop rules for using the new technology.

According to Haurwitz, Caribou license agreements include a section prohibiting the use of development on human embryos.

You need to study the scientific side of editing the genes of embryos, and then discuss how to responsibly use such opportunities, says Dudna. “Are there real unresolved medical problems that require such changes, or not?” I think this is the question.”

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