A breakthrough treatment for sickle cell disease and beta-thalassemia, CasJV, has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom. This innovative drug, the first to use CRISPR technology, opens a new era in the treatment of these genetic diseases. With promising results in clinical trials and a unique genetic modification approach, CasGevi represents great promise for improving the quality of life of patients.
A new treatment for sickle cell disease and β-thalassemia was recently approved by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for patients aged 12 years and older. This authorization is the result of a thorough assessment of the safety, quality and effectiveness of the treatment.
CasGevi is the first drug to use the genetic editing tool Crispr, which was awarded the Nobel Prize in 2020. This represents a revolution in the management of these genetic diseases. Sickle cell anemia and β-thalassemia are caused by genetic errors in the production of hemoglobin. These diseases are particularly prevalent in individuals of African, Caribbean, Mediterranean, South Asian, Southeast Asian, and Middle Eastern descent. Sickle cell disease presents with severe pain, severe infections, and attacks of anemia, while β-thalassemia results in severe anemia that requires regular blood transfusions.
mode of action of the drug
Casgevi works by replacing faulty genes in the patient’s bone marrow stem cells. These are taken, genetically modified, then reintroduced into the patient. Until now, bone marrow transplantation was the only solution for these patients, despite the risk of rejection. During trials, Casgevi demonstrated the ability to restore normal, healthy hemoglobin production.
In the sickle cell trial, 97% of treated patients were free from severe pain attacks for at least 12 months. For β-thalassemia, 93% of patients did not require a blood transfusion for at least one year after treatment. Side effects were similar to those of autologous stem cell transplantation, including nausea and fatigue.