Money from Genes

Ilya Khel, Hi-News.ru based on the materials of Scientific American

Just three years after discovering its potential in gene editing, the new CRISPR technique has become the hottest and most controversial in the field of genomics. And now it's more than science: it's big business. First discovered in bacteria, the CRISPR scheme (short palindromic repeats, regularly arranged in groups) is a gene editing tool that can be used to identify and accurately cut out the target gene in any organism on an RNA-DNA basis, thanks to the activity of the enzyme, CRISPR-associated protein 9, or Cas9. This technique can be used to remove, repair or replace genes; it is cheap, fast, reliable and – in principle – more accurate than other gene editing methods. Given the possible benefits to human health, the food industry and industrial biotechnology, it is not surprising that CRISPR quickly entered the biologists' Hall of Fame.

First it was pharmaceutical heavyweight Novartis, which signed two separate contracts with gene editing startups Intellia Therapeutics and Caribou Biosciences. The giant plans to use CRISPR to engineer immune cells and blood stem cells, as well as to search for drugs.

Just a few weeks after Novartis, AstraZeneca, another drug manufacturer, signed four deals with the Wellcome Trust Sanger Institute, Innovative Genomics Initiative, Broad and Whitehead Institutes in Massachusetts and Thermo Fisher Scientific. In addition to the already established CRISPR program, this technology will define and approve new goals in preclinical models for a number of disease areas.

Then the immunotherapy firm Juno Therapeutics shook hands with the gene editing startup Editas and started creating anti-cancer immune cell therapy; Vertex Pharmaceuticals and Crispr Therapeutics, another startup, entered into a $2.9 billion agreement; Regeneron Pharmaceuticals formed a licensing agreement with ERS Genomics, which owns the intellectual property rights to CRISPR by Emmanuel Charpentier, one of the creators of CRISPR. Not only pharmacists were noted in this case, the DuPont scientific company formed alliances with the University of Vilnius and Caribou Biosciences, having a certain interest in plant breeding and agricultural applications.

"Interest in gene editing by CRISPR-Cas9 from pharmaceutical companies literally exploded last year," says Bill Lundberg, senior researcher at Crispr Therapeutics. "I spent a significant amount of time talking to companies and organizations that were interested in working with us."

In addition to these transactions, there has been an increase in investment flows in startups that aim to commercialize CRISPR technology and related products, both in pharmaceuticals and in other areas. Caribou, founded by CRISPR pioneer Jennifer Dudna, has raised $15 million; Crispr Therapeutics, created by Charpentier, has raised $89 million since April 2014, plus $105 million through a deal with Vertex; Editas, founded by current Crispr patent holder Feng Zhang, has raised over $160 million.

Although this is just the beginning, analysts believe that this technology will be revolutionary for interested companies. "The fact that the deal alone was valued at $2.6 billion speaks volumes about the size that the technology can achieve," says Annette Breindl, senior science editor at Thomson Reuters BioWorld.

AstraZeneca is not only interested in the commercial potential of CRISPR. The company believes that this technology will offer a powerful solution for research and experimental problems that are slowing down the pharmaceutical industry, says company spokeswoman Karen Birmingham. From accelerating the identification and validation of new therapeutic targets, to creating improved animal models of human diseases in the shortest possible time and reducing the number of failed products, CRIPR will be able to reduce millions of R&D costs and accelerate the search for drugs. "Ultimately, through the use of CRISPR, we hope to increase the productivity of the pharmaceutical R&D process."

Similarly, in the agricultural space, CRISPR promises to be a solution for many manufacturers who want more precision in a tight time frame, says Neil Gutterson, vice president of R&D at DuPont Pioneer. Currently, the development period of agricultural products varies from 10 to 20 years, which complicates crop forecasting and imposes certain restrictions on the reaction. "Editing the Crispr-Cas9 genome will allow us to respond faster to farmers' needs and increase our ability to grow high-quality crops with the same or fewer resources."

In 2012, Dudna and Charpentier filed a patent application after working together on CRISPR, and a separate patent application from Zhang followed seven months later. Despite the fact that Zhang was the second, he received a patent based on the claimed date of invention. Now they are suing, trying to determine who invented the technique.

It is unclear what this will result in, says Marie Serebrov, editor of Thomson Reuters BioWorld. "If the court rules that this technology is not patented, it can cool the investment. On the other hand, if one group gets hold of the patent, it will result in a monopoly and make the license more expensive or will hinder research in this area." There will be a lull until the lawsuit is over.

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26.01.2015