Man modified: what to expect from technology for genome editing

Edit the genome will allow us to treat many serious diseases, and facilitate organ transplantation. In the United States and China the developers of these technologies are already actively attract investors

Now in the edit area of the genome are the real competitions that resemble what was happening in the 50-70-ies of the last century’s space race between the USSR and the USA. Only replaced the first space flight and launching satellites has come from the genetic experiments, and the place of the Soviet Union occupied China. The Chinese made the first edit the human genome with the help of acclaimed technology CRISPR/Cas9.


Scientists from the University hospital of Sichuan province conducted a pilot treatment of the patient with cancer in the last stage — metasteziruuschem lung cancer. Patients with this diagnosis usually extremely poor prognosis, because the usual methods of treatment for them is ineffective. Therefore, they first offer and have not proven methods.

The approach used by the Chinese, in fact, have long been known. It is to disable PD1 protein that suppresses the immune system cells and accelerates tumor growth. In case of successful inactivation, the cells begin to behave very aggressive and attack the tumors.

To increase the effectiveness of this technique has been just due to the technology of CRISPR/Cas9. It allows to purposefully edit the DNA of an organism without affecting other sections of the genome. The Chinese took the cells that contain the PD1 protein, and “parachuted” in their system CRISPR/Cas9. She quickly dealt with the PD1-receptor, and then these cells were injected back to the patient.

The exact results will be known in six months, but most likely will succeed. Of course, one hundred percent recovery is not, but in such severe cases, the extension of life even for a few months is a great achievement. It’s still raw and needs to Refine to make as safe as possible, but in any case it is a medical tool of the present, not the future.

Smart investments

The world is beginning to realize that the genome editing becomes really important. Now the demand for these technologies is growing rapidly, so the initial investors ‘ expectations are very high. Of course, they are too high and probably not justified fully in the “life Sciences”, nothing happens instantly.

An exception can be made only for those who are in this business is first. As, for example, venture Fund, Third Rock Venture, which has invested in a research company Editas Medicine $50 million when the nothing was created. However, EM began to receive funds from other venture capital funds, not just venture capital. The project has absorbed a lot more than really need for development — in ten or even a hundred times. These investments likely will not be proportional to income, and some part of the money will be lost.

Needed to create technology is a factor of “collapse” and leads to the collapse of the primary expectations. No matter how much we wanted to rely on and plan with any developments on the case goes otherwise. In their investments people are motivated by conservative experience, build the calculations for the next day based on what is happening today. And those who actually work with the technology, too far from financial Affairs and don’t know when to make adjustments. That’s why we need to be able to combine business intelligence with research.

World leaders

The Chinese are not particularly concerned by the fact that they have few patents and they are inferior in the field to other “players” at this stage they are not focused on sales. For them it is crucial first to integrate CRISPR technology in a therapeutic platform. With a population of 1.5 billion people it is easy to imagine what a wide medical application can obtain this technology. In the first 20-30 years they can rely only on his own, self-sufficient market to develop in the country without any problems.

A similar situation exists in America, adjusted for other market conditions and rules in the field of intellectual property. The Americans at the expense of a more balanced and sophisticated technological approach are by far the real leaders in the field of genome editing. Now they are preparing to experiment, which is similar to the Chinese. The experiment is already registered and has allocated at least $250 million.

CRISPR-therapy is developing in Europe. For example, the company CRISPR therapeutics has received strong support from the so-called Big Pharma, the largest pharmaceutical companies in the world. But the EU is extremely conservative legislation, so complex technological issues to do anything quickly impossible. Even the first European gene therapy drug GLYBERA by the European medicines Agency endorsed just three years after the filing of the first application. On the other hand, Europeans who work in the areas of research, are true pioneers. They carry the whole organization of the process and define how it should be governed and regulated. As this is the first case, along with them, created a legislative framework.

Cell technologies in Russia

We have only a few months ago there was adopted the Federal law “On biomedical cell products.” Fully work it will start only in 2018, if possible, to take all necessary regulations. And handling he will be the basics cells. For Russia such a law — it is “gas pedal”. It is, of course, will be refined, and most importantly, this process continued and gained momentum.

Our brake is not in the laws. The engine of innovation is always money, in this case those companies that are invested in genetic engineering. In America, this branch of technology has become a business in China — business with government support and huge investment. In Russia, however at the moment there is no such money, no such companies. However, current events in China and America, forced to move us.

It is clear that we are already very late. The problem is that our state and entrepreneurs will not begin to Fund something that is not yet lit in the West. The process starts only when the same America about the new technology will know almost every second passer-by. The hype will begin to subside, and we will only proceed to funding.

As much as we wanted in Russia have not yet formed the market in the field of genomic editing. This is dictated by the history of our business the lack of transparency and clear rules of investment. To invest at all and always have very carefully. This conservatism in extremely small compared to USA and China investment to us is not so bad. But at the same time it does not allow venture funds to develop.


Edit the genome using CRISPR/Cas9 in addition to cancer, most likely, can be used in the treatment of autoimmune and chronic infectious diseases, and in the more distant future and in organ transplantation. The real limits of applicability are not yet fully understood, and so there is time to perform, whither the technological train.

The authors ‘ point of view, articles which are published in the section “Opinions” may not coincide with ideas of editorial.

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