2023-11-24 17:05:00
The United Kingdom this week authorized the use of generic editing technology Crispr as a treatment for sickle cell anemia and chronic anemia – also called “thalassemia”. Regulatory approval is considered a new revolution for medicine.
The method was developed by Nobel Prize winners in Chemistry, Jennifer Doudna and Emmanuelle Charpentier, and is considered a “single cure” to get to the cause of these diseases, as highlighted by the first to Wall Street Journal.
Among the main challenges are those that include the safety of the procedure. This is because the patient needs to undergo chemotherapy and other more incisive procedures, which involve the collection and manipulation of genetic material, in addition to the reimplantation of cells in the body, within the “ex vivo” method, that is, outside the body.
For analysts, it would be more interesting to carry out the therapy in the “in vivo” model. In other words, administering gene editing directly to the patient, not extracting and reintroducing cells.
Expectations for the future
These impasses also had an effect on investors, who consider themselves more cautious in relation to the case. Even so, pharmaceutical companies Vertex Pharmaceuticals and Crispr Therapeutics are interested in the matter.
In the future, the tendency is for gene therapy to be expanded to other diseases. While in this case, it involves the liver, there are plans for other research to be carried out to combat diseases such as cancer, AIDS and Alzheimer’s disease. The technology is also awaiting approval from the FDA, the United States regulatory body, scheduled for December.
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