Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial.

ABSTRACT: β-Thalassemias are inherited anemias that are caused by the absent or insufficient production of the β chain of hemoglobin. Here we report 6-8-year follow-up of four adult patients with transfusion-dependent β-thalassemia who were infused with autologous CD34⁺ cells transduced with the TNS9.3.55 lentiviral globin vector after reduced-intensity conditioning (RIC) in a phase 1 clinical trial ( NCT01639690) . Patients were monitored for insertional mutagenesis and the generation of a replication-competent lentivirus (safety and tolerability of the infusion product after RIC-primary endpoint) and engraftment of genetically modified autologous CD34⁺ cells, expression of the transduced β-globin gene and post-transplant transfusion requirements (efficacy-secondary endpoint).

SUBMITTER: Boulad F 

PROVIDER: S-EPMC9380046 | biostudies-literature | 2022 Jan

REPOSITORIES: biostudies-literature