UK Clears Historic Gene Therapy for Sickle Cell Disease Treatment

The UK has approved the use of a gene-editing therapy called Casgev for patients with sickle cell disease and beta thalassemia. Using CRISPR-based gene editing technology, the therapy has been found to effectively relieve severe pain associated with the blood disorders for at least 12 months. This treatment has the potential to improve the current standard of care as it targets the genes in the bone marrow stem cells that produce faulty blood cells. However, the cost of CRISPR-based therapies may limit their availability to the general public. The average cost of such therapies is estimated to be between . Despite the costliness, CRISPR-based therapies show huge promise for rare conditions and may open the door for further applications of this technology in the future. Casgevy is still being reviewed for safety standards in other countries, including the United States and Saudi Arabia.