This study is testing if a new treatment, called CTX001, is safe and works for people with transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD). β-thalassemia is when the body makes less hemoglobin, which carries oxygen in the blood. Sickle cell disease is when red blood cells are shaped like a sickle and can block blood flow. The study uses CRISPR-Cas9, a tool that can change genes, to improve stem cells from the participant's own body.
Key Points:
- Participants will receive one dose of the treatment.
- Participants must have certain types of TDT or SCD and need frequent blood transfusions or sickle cell crises.
- Participants cannot have a healthy donor available or previous stem cell transplants.
The study is open-label, which means both the doctors and participants know what treatment is being given. It is important for participants to know that they will be monitored closely for any side effects or improvements. They should talk to their doctor to see if this study is a good option for them.