EU green light for gene therapy on sickle cell anemia and thalassemia. Exagamglogene autotemcel [exa-cel] is the first gene editing therapy based on the CRISPR/Cas9 system.
This technology allows the patient's hematopoietic stem and progenitor cells to be genetically modified, leading to the production of high levels of fetal hemoglobin in red blood cells. In trials, the treatment reduced or eliminated vaso-occlusive crises and reduced the need for transfusions.