DNA as medicine
Cell and gene therapy are changing pharmaceuticals
Daria Shipacheva, Forbes, 29.05.2018
Companies that conduct clinical trials of innovative drugs are already valued at billions of dollars.
Drugs obtained with the help of genetic engineering and cellular technologies are unlikely to become so widespread in the coming years that they will overtake antibiotics and antipyretics in sales. But these modern drugs are indispensable in the treatment of certain types of cancer, rare hereditary diseases, HIV therapy and other viral infections. And this is a large market with a potential turnover of tens of billions of dollars.
Stem cells and genetic engineering
Gene therapy is a common name for technologies (as well as related drugs) that use gene modification to achieve the desired medical effects. For example, a gene encoding a certain protein can be delivered to the patient's body. When the necessary DNA gets into the cells, they will be able to start producing this protein, which will have a therapeutic effect.
Another option is to edit the "defective" gene variants so that they start working correctly. If a person has only one mutant version of the gene that causes the disease, you can simply cut it off – and the DNA will repair itself by copying the genetic information from its second "half". In the case when both DNA chains are damaged, a donor sequence can be delivered to the cells.
Finally, it is possible to control gene expression with the help of special drugs. If the activity of a certain gene leads to a disease, it is suppressed. And vice versa: in a situation where the disease occurs due to a "defect" of some gene, the medicine helps to turn it on.
As for cell therapy, in most cases it is a stem cell treatment. They can develop into cells of any type, of any organ, if their growth is properly managed. And scientists have mastered this science, but it was also important to get a sufficient supply of stem cells. The problem was solved by Japanese scientist Shinya Yamanaka, who in 2006 for the first time was able to obtain induced pluripotent stem cells, that is, to return adult cells to a state where anything can grow out of them. Exciting prospects have opened up for science: it is possible to grow new organs, repair damaged areas of the nervous system, treat blood diseases – all this becomes possible with the use of stem cells.
At the junction of cell and gene therapy is the technology of cell modification. Today, scientists are experimenting mainly with immune cells – T-lymphocytes. With the help of genetic editing, they are "taught" to better recognize pests (for example, cancer cells) and effectively destroy them.
Gene Market
In 2016, the market for gene therapy drugs was estimated at $584 million. And by 2023, according to analysts, global revenue from the sale of such drugs will exceed $4.4 billion, which is more than 30% growth annually.
Among the market leaders are both traditional pharmaceutical giants (such as Novartis and GlaxoSmithKline) and more specialized companies (Spark Therapeutics, Bluebird bio, Amgen, Celgene).
As follows from the report of Allied Market Research, the vast majority of gene therapy drugs are produced for patients with oncological pathologies. And in the near future – at least until 2023 – this niche will retain its primacy in the market. Next to cancer drugs are gene therapies for rare diseases, cardiovascular diseases, neurological disorders, infections.
Market leaders follow trends and develop drugs for cancer patients. For example, at Amgen, which led the top 25 biotech companies in 2017 with a capitalization of $129.1 billion, 20 of the 37 drugs in clinical trials relate to the treatment of tumors and blood diseases.
And Novartis became the first company to be allowed by the American Food and Drug Administration (FDA) to launch mass production of cancer cell therapy based on genetic modification. This is the case when a pharmaceutical company produces a drug from T-lymphocytes, turning them into a means of combating oncology, namely acute lymphoblastic leukemia.
Novartis has already started to receive the first money from the sale of its drug called Kymriah. And considerable – each course of gene therapy for cancer costs about $ 475,000. Following this, the FDA approved another drug for the treatment of oncology with a similar mechanism of action – Yescarta from Gilead Sciences (capitalization in 2017 – $ 103 billion); it will be sold at a price of $373,000 per course and help patients with non-Hodgkin's lymphoma.
Novartis pharmaceutical giant will be able to earn about $300 million a year from Kymriah sales. And Gilead Science analysts predict revenue from the drug Yescarta at $ 250 million for 2018.
Novartis wants to expand the range of use of its medicine so that it can also be prescribed to patients with non–Hodgkin's lymphoma - this will allow the company to compete for the market, which is estimated at about $ 1 billion. But the pharmaceutical giant will have to compete not only with Gilead Sciences, which has already offered its own – and cheaper – solution, but also with Bluebird Bio and Juno Therapeutics, which are developing similar products.
Analysts still find it difficult to assess the profitability of cellular gene therapy. But according to their data, the cost of producing such cancer drugs should be at least $200,000 per course.
The truly revolutionary discoveries that are taking place in the gene therapy market and threaten to turn the pharmaceutical market upside down (at least in the field of oncology treatment) attract investment in the industry: over the past five years, manufacturing companies have received about $ 600 million in venture capital for their developments.
Market prospects for stem cells
According to the forecast of analysts from Grand View Research, the market of drugs based on stem cells will reach $15.6 billion by 2025. Today, the most promising applications of stem cells are the treatment of Parkinson's disease and Alzheimer's disease, as well as spinal cord injuries, type I diabetes mellitus, blood diseases and oncology.
But so far, even top stem cell companies cannot boast of the same scale as their colleagues in gene therapy. For example, the largest company that is traded on the NASDAQ exchange – Sangamo Therapeutics – has a capitalization of only $ 2.1 billion.
At the moment, Sangamo Therapeutics is developing two types of therapy based on its own – autologous – stem cells for the treatment of blood diseases. If the manufacturer succeeds, then with the help of these drugs it will be possible to treat such genetic blood diseases as beta-thalassemia and sickle cell anemia.
The development of the stem cell market is hindered by the fact that it is unprofitable to make drugs based on autologous cells, since such production cannot be scaled: a drug created for one patient cannot be used for another. At the same time, it may be profitable to produce drugs based on foreign – allogeneic – stem cells. But the development of such tools takes a lot of time: it is necessary to make them "invisible" to the immunity of patients in order to reduce the risk of rejection.
What's next?
According to all estimates, the market of cell and gene therapy will be actively developing in the coming years, and the average annual sales growth will be at least 30%. New discoveries in the field of gene therapy promise 2,200 clinical trials that are being conducted around the world: scientists are looking for remedies for various types of cancer, rare genetic disorders, Parkinson's disease, HIV and other diseases.
Ahead of the whole planet, as always, is the USA – about 55% of research takes place there. Also, developments in the field of gene therapy are being carried out in Europe, Canada and China. One drug created with the participation of genetic engineering was also launched in Russia – this is Neovasculgen, a drug designed to help with lower limb ischemia.
As for stem cells, market growth is expected as a result of the emergence of new regenerative medicine drugs – now many of them are under development. Most of the research takes place in the USA, but new stem cell solutions are also emerging in Singapore and Japan.
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