A world first. A treatment designed using the Crispr genome editing system has just been approved by the European Medicines Agency, according to information collected by The world. This technology makes it possible to make changes to genetic sequences directly in cells.
The health authority asked the European Commission on Friday to officially authorize this treatment, called “Casgevy”. It will concern any patient over the age of 12 suffering from serious forms of sickle cell anemia or beta thalassemia, two very disabling blood pathologies of genetic origin. The member states of the European Union now have complete freedom to define the conditions of use of this new treatment and its cost.
A clinical trial with impressive results
The results of the clinical trial of this new treatment are impressive. Among the 29 participating patients aged 12 to 35 suffering from severe forms of sickle cell disease, 28 no longer experienced a crisis for at least a year.
Worldwide, 300,000 to 400,000 children are born with sickle cell disease each year, making it the most common monogenic disease. In infected people, blood cells no longer carry oxygen as well, tend to clump together and create clots. This disease not only causes chronic anemia requiring transfusions, but also attacks of pain which can lead to the deterioration of several organs and strokes or even early death.