The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) announced its approval for a treatment called Casgevy, which is set to address sickle cell disease and beta thalassemia, both of which are genetic disorders resulting from errors in the genes responsible for hemoglobin production.
Sickle cell disease, characterized by severe pain attacks, is more prevalent among individuals with African or Caribbean ancestry, while beta thalassemia primarily affects people of Mediterranean, South Asian, Southeast Asian, and Middle Eastern origin. To date, no universally effective treatment has been available for either of these conditions.
Traditionally, bone marrow transplants, requiring closely matched donors and carrying a risk of rejection, have been the sole permanent treatment option. However, Casgevy, the CRISPR-based gene-editing treatment, has shown promise in clinical trials by restoring healthy hemoglobin production in a majority of participants with sickle-cell disease and transfusion-dependent beta-thalassemia, thereby alleviating disease symptoms.
Julian Beach, the interim executive director of healthcare quality and access at MHRA, expressed his satisfaction with the regulatory approval, emphasizing the innovative and pioneering nature of the Casgevy gene-editing treatment. The CRISPR-Cas9 gene-editing technique enables precise modifications to DNA and has been hailed as a groundbreaking advancement in the field of genetic medicine, leading to the awarding of the Nobel Prize in chemistry to its inventors, Emmanuelle Charpentier and Jennifer A. Doudna, in 2020.