There are a number of diseases caused by “breaking” in a particular gene. Since our genes are given to us from conception, they cannot be replaced and therefore cured of the disease. Now a completely new class of drugs has appeared: they literally deliver “working” copies of the gene to the relevant cells of the human body, where these genes produce the “proper” protein. This is called in vivo gene therapy, i. e. in a living organism.
Luxturna is the first drug having such an effect, approved by the FDA in 2017. It is intended for the treatment of one of the varieties of Leber’s amaurosis – a rare inherited retinal disease. Zolgensma was approved in the United States in 2019. It has a similar effect, but is intended for the treatment of some types of spinal muscular atrophy.
According to the WHO, more than 70 million people worldwide suffer from chronic hepatitis C. Many of them may not know about their diagnosis because for years the disease is asymptomatic. But most patients later develop liver cancer or cirrhosis.
In 2013, Sofosbuvir appeared on the global market, followed by several other drugs that can cure hepatitis C of various genotypes in most patients in just a few months and with minimal side effects. At a conservative estimate, this is the revolution in the treatment of hepatitis C, against which, incidentally, there is still no vaccine.
Bronchial asthma affects 235 million various aged people worldwide and it is the most common chronic disease among children.
According to the WHO, in 2015, 383 thousand people died from it. There are the drugs that help control asthma and improve patients’ quality of life. But in some cases they are not effective. Omalizumab, approved by the FDA in 2003, can then be used. The principle of its action is binding to immunoglobulin E in the body, which plays an important role in the development of an allergic reaction, thus blocking it. The drug is also used to treat chronic urticaria.
