The European Commission has approved a new gene therapy for the treatment of beta-thalassemia and sickle cell anemia.
The treatment "has the potential to transform the lives of patients suffering from these two pathologies; now it is important that this therapy is quickly made available to eligible patients", said Franco Locatelli, director of the Department of Pediatric Hematology and Oncology at the hospital. Pediatric Bambino Gesù and principal investigator of two of the studies that led to the approval.
Sickle cell anemia and beta thalassemia are both hereditary blood diseases that affect red blood cells, affecting their ability to transport oxygen to the body's organs and tissues.
Both require lifelong treatment and cause a decrease in the quality and expectancy of life.
Currently, one of the possible potential treatments is stem cell transplantation from a compatible donor, but this option is only available to a small percentage of patients.
The new therapy whose name is 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.
The product can be used in patients aged at least 12 years suffering from transfusion-dependent beta-thalassemia or severe sickle cell anemia characterized by recurrent vaso-occlusive crises, for whom hematopoietic stem cell transplantation is appropriate but a related donor is not available compatible.
It is estimated that around 8 thousand patients in Europe are potentially eligible for treatment.
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