LentiGlobin BB305 gene therapy reduced or eliminated the need for monthly transfusions among patients with transfusion-dependent beta-thalassemia, according to results from two phase 1/phase 2 clinical trials. “These study results exceeded our expectations with clinical benefit for nearly all patients and suggest that gene therapy may be an effective treatment for thalassemia in the future,” head of hematology at Lurie Children’s Hospital of Chicago, said in a press release.
Patients with transfusion-dependent beta-thalassemia — the most severe form of the disease — require long-term red cell transfusions for survival; however, risk for transfusion-related iron toxicity and infections remains high. Allogeneic hematopoietic stem cell transplantation serves as a possible curative option for beta-thalassemia, but use is limited by high risk for graft rejection, graft-versus-host disease…
