The goals of modern genetic engineering
The Russian government in the roadmap for the development of biotechnologies and
They are already trying to implement this idea abroad:in the US Academy of Sciences allow the possibility of editing germ cells and embryos under the strictest control and for medical purposes. For example, to combat diseases that cause disabilities. The United States understands that the development of technology is inevitable, as well as changes in the genome of sperm, stem cells and others in the foreseeable future. In China, this idea has already been resolved at the state level: in 2016, changes were made in the genome of an adult - they transplanted immune cells to cure lung cancer.
Modern genetic engineering is fighting for victoryon oncological diseases: most of the developed gene therapy drugs are focused specifically on the treatment of cancer. It is possible that the first applications of the genome editing technique will be precisely on patients with incurable stages of cancer. In addition, there are serious diseases of the hematopoietic system (hemophilia, thalassemia and others) - CRISPR Therapeutics and Vertex Pharmaceuticals are already working with them, altering bone marrow stem cells.
In addition, the problem is not only in complexity.development of treatment, but also in the cost of therapy. Ways to defeat cancer are emerging, but they are expensive: both for the manufacturing company (high scaling costs) and for the consumer. Therefore, it is necessary to look for alternative methods that will be more accessible.
Latest scientific advances
The biggest breakthrough of the decade was the systemCRISPR / Cas9, for which its creators Jennifer Doudna and Emmanuelle Charpentier received the Nobel Prize in Chemistry in 2020. CRISPR / Cas9 is a high-precision genome editing method that allows you to change the genes of living microorganisms, including humans. And with its help there are chances to create methods of combating HIV and other diseases, which today sound like a sentence.
Scientists around the world began to experiment withCRISPR / Cas9, but the loudest (and at the same time important for the world) story took place in China. In 2018, genetically modified children were born - girls Lulu and Nana. The zygote was obtained using IVF (in vitro fertilization), genetically altered with CRISPR / Cas9 and implanted into the uterus of the woman who gave birth to the girls. It is possible to argue for a long time about the ethics of the experiment and the modification of people (in many countries, editing the genes of the embryo is prohibited due to unforeseen consequences for the gene pool), but it cannot be denied that the experiment is of great importance for science.
Zygote DNA edited to tryexclude the occurrence of HIV infection (which the father had), as well as smallpox and cholera through excision of the CCR5 gene. It is noteworthy that the story took place on the eve of the Second International Summit dedicated to editing the human genome, where the audience discussed: is it time to allow people to edit themselves and how. If the method of using CRISPR / Cas9 in embryos still gets government approval (and this is not just about China), it will finally become possible to have healthy children through IVF, even if the parents (one or both) have mutations. Experiments in 2017 did not show any "side effects" from CRISPR / Cas9 in mice, monkeys and 300 human embryos, but it is still too early to assert their absolute safety.
Less resonant scientific achievement - in 2019year in the journal Optics Letters, an article appeared on the development of a new sensor for the detection of a biomarker of oncology (S100A4 protein) in urine. The peculiarity of this technology lies in the high sensitivity of the sensor: it can detect the presence of cancer even with a low concentration of the corresponding biomarkers. In addition, this technology can be scaled up for mass use at a lower cost than the sensors that existed until then - they were too large and expensive to integrate into devices for regular monitoring of the patient's condition. The technology is based on an optical microchip with probes that bind to the S100A4 protein biomarker in a urine sample. Next, the frequency shifts of the laser in the chip are monitored and the presence of cancer is determined.
Leading startups in genetics
In the USA in 2008, the invention of the year was the serviceDNA testing from startup 23andMe. The company turned people's minds around, as Apple did with the iPhone the year before: 23andMe began the personal genomics revolution. DNA typing became available to everyone who bought a special kit. This turned out to be important for planning pregnancies: the tests made it possible to see mutations in the unborn child (based on the genes of the parents) and to exclude the risk of having a child with diseases that could lead to his disability.
In Russia, after the success of 23andMe, they began to appearsimilar startups. For example, “Genoanalitika”, which was one of the first in our market to offer DNA testing. But the startup Genotek is better known, called the leader in commercial genomics. The company appeared in 2010 on the basis of the Moscow State University business incubator, began work with cooperation with the Russian Academy of Sciences and already in the first year became profitable.
Today Genotek works with 71 countries, fulfillingresearch for clinics and private clients. The most famous development of Genotek is a DNA test, with the help of which the genotyping procedure is carried out. The client transfers the biomaterial (saliva sample) to the laboratory and sees the result in his personal account: predisposition to specific diseases, dietary recommendations, possible adverse reactions to certain medications, and much more, including hereditary pathologies and problems of conception. The company has also developed high sensitivity PCR tests for COVID-19 for Russia and the CIS countries.
In addition to the classic DNA tests thatare offered by several Russian startups, there are also more narrowly focused ones. For example, JVS Diagnostics is developing a test that detects malignant tumors in the early stages with an accuracy of 90%. It works with the support of Skolkovo, and in 2018 raised $ 400,000 to bring the test to the masses. Another narrow niche is the study of genes that influence ophthalmic diseases. The startup Oftalmic has been doing this since 2008: it offers 60 types of DNA tests to diagnose eye problems. Nothing is known about the attracted investments.
There are also similar startups in Europe:for example, Blueprint Genetics (Helsinki) offers AI-powered genetic testing solutions. The startup is focused on diagnosing hereditary diseases using the CLINT AI platform and OS-Seq, a patented targeted sequencing technology (a set of methods for determining the nucleotide sequence of elements in a DNA molecule). For Europe, Blueprint is one of the leading providers of genetic tests covering over 2,200 diseases in 14 areas from cardiology to hematology.
British startup Freeline went elsewheredirection: he develops gene therapy methods for the treatment of chronic systemic diseases. In 2020, the project received € 106 million to develop solutions aimed at treating liver diseases that are considered incurable and poorly understood. In the future, the startup plans to expand its program to treat hemophilia, Fabry disease and Gaucher disease. All of them belong to orphan (rare) diseases, of which there are more than 6 thousand in the world, but only a few amenable to therapy.
Market forecasts
One of the emerging trends that analystspredict further development - this is the leadership of the Asia-Pacific region. The stimulus for active development should be given by developments for the management of diseases: both prevention and treatment (or slowing down the progression). In addition, personalized medicine will continue to gain momentum - something that has appeared thanks to companies that release DNA tests to the masses.
Leaders remain 23andMe, Oxford NanoporeTechnologies and Veritas Genetics, which commercialize their developments. Until 2017, only medical specialists had access to tests, and now similar studies are being carried out for the mass consumer. Also, Veritas Genetics began to conduct genomic sequencing of newborns in 2017 in China for the early detection of severe pathologies: the test can report almost a thousand diseases. True, it costs $ 1.5 thousand. And its results continue to be questioned three years later.
Domestic experts are sure that the nearestthe future of genetics is the active development of methods for the treatment of oncology and neurodegenerative diseases. Now the efforts of most companies in the MedTech and BioTech segment are focused on the study of the DNA sequence in cancer cells, their changes and the search for individual methods of therapy for a specific tumor. The first drugs began to appear on the market, and there will be more of them. It is possible that the technology of using genetically modified cells in medicine will be scaled up, and just as antibiotics made more than a dozen fatal diseases curable in the 20th century, cancer and neurodegenerative diseases will also cease to sound like a sentence.
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