This therapy is recommended for patients with transfusion-dependent beta-thalassemia (TDT), a form of thalassemia where regular blood transfusions are necessary. X003 addresses the root cause of the disease by adding a working copy of the beta-globin gene to the patient’s blood stem cells, allowing them to produce normal hemoglobin.

The treatment involves 7 core steps: screening, consent, stem cell collection (apheresis), manufacturing, myeloablative conditioning, cell infusion, and follow-up care. The entire process takes place over a few months, with most patients recovering within 1 to 3 months after treatment.

For patients with transfusion-dependent beta-thalassemia, this treatment is medically necessary as it offers the potential to cure the disease, reduce iron overload, and eliminate the need for lifelong transfusions.

The main benefits of X003 include achieving transfusion independence (94.1% success rate), faster recovery times, and long-term, stable disease control. It is effective for all known beta-thalassemia mutations.