Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial.

ABSTRACT: Autologous transplantation of gene-modified cells for treatment of sickle cell disease has involved myeloablative conditioning with associated cytopenias and toxicities. We report results of seven patients treated in a first-in-human phase 1/2 study for sickle cell disease using reduced-intensity conditioning transplant of autologous hematopoietic stem cells genetically modified with a lentiviral vector (GbG^M), with 2-7 yr of follow-up. GbG^M encodes a modified γ-globin gene that expresses a potent anti-sickling fetal hemoglobin, HbF^G16D. The primary study objectives were safety (occurrence of adverse events and duration of neutropenia and thrombocytopenia) and feasibility of treatment. Primary feasibility endpoints of collection of at least 8 × 10⁶

SUBMITTER: Grimley M 

PROVIDER: S-EPMC12702520 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature