American agency FDA (Food and Drug Administration) has recently approved a new breakthrough gene editing therapy called Casgevy, developed by CRISPR Therapeutics. This marks the second regulatory permit for the innovative CRISPR technology.
Transfusion-dependent beta-Thalassemia, a serious genetic disease that disrupts the normal production of hemoglobin in the blood, requires patients to regularly undergo blood transfusions. Casgevy utilizes advanced CRISPR gene editing technology to modify the blood cells of patients. After modification, these cells are transplanted back into the bone marrow, which helps increase the production of hemoglobin.
However, the FDA warns that patients should be prepared for potential side effects such as ulcers in the mouth, fever, and a decrease in appetite. Nevertheless, the prospects discovered by this therapy are seen as a significant step in the treatment of genetic diseases.
Vertex, the pharmaceutical company behind the development of Casgevy, plans to create a network of specialized medical centers throughout the United States that will directly introduce the drug to patients. Currently, there are already nine such centers in operation, with plans to launch more in the coming weeks. The company emphasizes that the treatment procedure of Casgevy requires a specialized experience in the field of stem cell transplantation.
In recognition of their groundbreaking work, Emmanuel Sharpe and Jennifer Dawd received the Nobel Prize in 2020 for their contributions to the development of the CRISPR tool. The Nobel Committee hailed CRISPR as a revolutionary technology that has opened new opportunities for the treatment of previously incurable diseases.
The success of CRISPR Therapeutics in obtaining regulatory approvals has significantly boosted its stock performance, with shares increasing by more than 50% last year.