Gene therapy effective in beta-thalassemia, reduces need for transfusion: NEJM
USA: A phase III study showed betibeglogene autotemcel (beti-cel) gene therapy to be safe and effective in patients with non-β0/β0 genotype β-thalassemia.
The study, published in the New England Journal of Medicine, found that treatment with the gene therapy led to a sustained HbAT87Q level and a total hemoglobin level that was high enough to allow transfusion independence in the study population, including those younger than 12 years of age.
Beti-cel gene therapy contains autologous CD34+ hematopoietic stem cells and progenitor cells transduced with the BB305 lentiviral vector encoding the β-globin (βA-T87Q) gene. Franco Locatelli and the team aimed to evaluate its safety and efficacy in adult and pediatric patients with transfusion-dependent β-thalassemia and a non–β0/β0 genotype.
For this purpose, 23 patients underwent myeloablation with busulfan (with doses adjusted on the basis of pharmacokinetic analysis) and received beti-cel intravenously. They were followed for a median of 29.5 months.
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