- In January 2024, the U.S. FDA granted approval for Casgevy, a CRISPR-based therapy, for its second indication: the treatment of transfusion-dependent beta thalassemia in patients aged 12 and older. This approval follows its previous authorization in December 2023 for the treatment of sickle cell disease. Casgevy's expanded approval marks a significant advancement in gene-editing therapies for blood disorders, offering new hope for patients with these conditions
- In August 2022, the U.S. FDA approved Zynteglo (beti-cel) as the first curative gene therapy for patients with beta thalassemia who require regular red blood cell transfusions. This approval represents a major breakthrough, offering the potential for long-term relief from transfusion dependence. Zynteglo uses gene editing to address the root cause of beta thalassemia, providing a transformative treatment option for patients
- In June 2021, the U.S. FDA granted breakthrough therapy designation to Rusfertide (PTG-300) as a potential treatment for polycythemia Vera. This designation recognizes its ability to reduce erythrocytosis in patients who do not require additional treatments for thrombocytosis or leucocytosis. The approval highlights Rusfertide's promising role in managing this rare blood disorder by targeting the underlying causes of excessive red blood cell production
- In April 2022, Himalaya Wellness Company partnered with the Sankalp India Foundation to provide support for thalassemia patients, focusing on offering a permanent curative option. This collaboration aims to facilitate bone marrow transplants, which can potentially cure the disease. By joining forces, the two organizations seek to improve access to life-changing treatments for thalassemia patients in need
