The final report of the phase 1/2 lentiviral gene therapy trial in severe β-hemoglobinopathies, HGB-205, shows a long-term safety profile and sustained reduction of transfusion requirements in patients with β-thalassemia and of sickle cell crises in patients with sickle cell disease.
- Elisa Magrin
- Michaela Semeraro
- Marina Cavazzana