Key clinical point: The gene therapy LentiGlobin reduced transfusion dependence in children and young adults with non-beta0/beta0 thalassemia in two clinical trials.
Major finding: In a phase 1/2 trial, 8 of 10 of patients achieved transfusion independence at a median follow-up of 36 months. In a phase 3 trial, transfusion independence was achieved by 2 of 3 patients with follow-up of at least 12 months.
Study details: A phase 1/2 trial of 10 patients and a phase 3 trial of 16 patients with non-beta0/beta0 thalassemia.
Disclosures: The presenter reported advisory board engagement with bluebird bio, which sponsored both trials.
Olson TS et al. ASPHO 2019, Abstract 2002.