Sickle cell anemia: first therapy with gene scissors approved

In the UK, a Crispr/Cas gene therapy called "exa-cel" has been approved for the first time for the treatment of sickle cell anemia and beta-thalassemia. This advanced method makes it possible to correct genetic defects in the haemoglobin gene by specifically cutting and modifying the DNA. The therapy uses modified blood stem cells that are altered in the laboratory and then introduced back into the body to produce healthy cells. The approval in the UK marks a significant advance in the use of Crispr/Cas in clinical practice. Experts emphasize the promising approach, but point to safety concerns and the need for intensive monitoring. The European Medicines Agency is currently carrying out an assessment.