poudre et solvant pour solution injectable / perfusion clinical trials

A single arm, open label, multicenter, single-dose, phase 2b clinical study evaluating efficacy and safety of gene therapy using autologous CD34+ hematopoietic stem cells transduced with the GLOBE lentiviral vector using an improved transduction protocol in subjects with transfusion-dependent beta-thalassemia.

This study is testing a new gene therapy for adults with beta-thalassemia who need regular blood transfusions. The main goal is to see if this treatment can help people stop needing transfusions for at least a year.