Current therapeutic landscape of β-thalassemia: focus on gene therapy - PubMed
18 hours ago
- #β-thalassemia
- #transfusion-dependent thalassemia
- #gene therapy
- β-thalassemia is an inherited blood disorder causing chronic anemia and transfusion dependence.
- Standard treatments include regular blood transfusions and iron chelation therapy, with curative allogeneic stem cell transplant limited by donor availability and risks.
- Two FDA-approved autologous gene therapies, betibeglogene autotemcel (beti-cel) and exagamglogene autotemcel (exa-cel), now offer high rates of sustained transfusion independence.
- Gene therapy involves myeloablative conditioning chemotherapy, with risks of short- and long-term toxicities.
- Challenges include high costs, centralized manufacturing, and limited access to gene therapy, despite its transformative potential.