For the first time in the world in the treatment of diseases CRISPR The use of gene editing technology was permitted. The first technology to receive this permission casgevy It became known as Exa-cel.

on November 16 UK Medicines and Healthcare Products Regulatory Agency (MHRA) The permission granted will be used in the treatment of sickle cell anemia and transfusion-dependent Mediterranean anemia. Previously, the US Food and Drug Administration had declared the drug safe for clinical use but did not allow it to be used in treatments. These permits were expected to be approved in December 2023.

A new era may dawn in the field of gene therapy

The MHRA’s decision is historic. After this decision gene therapy As a much more popular and accepted method, it will be useful in the fight against many hereditary diseases. However, the decision raises many questions that need to be answered regarding sustainability, costs and long-term effects.

casgevy It will first be used in the treatment of sickle cell anemia and transfusion-dependent Mediterranean anemia. In the first of these diseases, the red blood cells are not round and they are sickle-like It happens in a structure. These cells have problems such as sticking together and blocking blood vessels, causing pain and sensation of pain, and a short lifespan. If care is not taken, this disease can have serious consequences.

In Mediterranean anemia, the number of red blood cells in the blood is very low. In transfusion-dependent Mediterranean anemia This production is so low that patients regularly require red blood cell transplants to survive. Gene editing appears to be an important alternative for the treatment of these genetically occurring diseases.

How does CRISPR work?

First appeared in 2012 CRISPR The technology actually has a very simple working logic. In essence, CRISPR is used as ultra-precise genetic scissors. DNA a suitable copy for the part to be replaced. RNA It is synthesized and combined with an enzyme called Cas9. The part of the double-stranded DNA containing the incorrect code is cut out and the edited version is added in its place. In other words, if we think of people as digital products, some of the codes in our source software can be freed from bugs in this way.

Casgevy also targets a gene called BCL11A. This gene is responsible for the production of hemoglobin under normal conditions. of fetal version It synthesizes a protein that allows it to transition to the adult version. However, in sickle cell anemia and Mediterranean anemia, adults homoglobins It becomes problematic. Stopping BCL11A allows the hemoglobins to be used as they were at birth.

On the other hand, this process will not be easy at all. People may need to be hospitalized for about a month before all hemoglobin levels gradually change and the formation of red blood cells in the bones reaches the desired level. Also some medications immunosuppressants will be taken together.

Yet 28 of the 29 patients with sickle cell anemia who participated in clinical trials for more than a year He didn’t have an attack of pain. 39 of the 42 patients with Mediterranean anemia did not require red blood cell supplementation during the same period. The need for blood supplements in the remaining three patients was reduced by 70%.

Genetic engineering poses safety concerns.

In the eyes of many, genetic engineering is an important weapon in the fight against diseases. However, there are some general concerns about this topic.. Imperial College LondonDavid Rueda, head of the Molecular and Cellular Biophysics department atIt is known that CRISPR can have unknown consequences on cells undergoing treatment. “It may be necessary to see sequence data for the entire gene to reach a conclusion.” He uses the expressions. this too CRISPR It requires examining all DNA for unexpected effects of the treatment.

Casgevys It is not yet known when it will be available to patients. The ability to use the treatment largely depends on knowing the costs. Let’s say gene therapy could cost millions of dollars. Developing the drug Vertexhas not yet announced a price, but the company continues to negotiate with authorities to ensure the drug reaches patients and is covered by insurance.