Betibeglogene Autotemcel Gene Therapy for Non–β ⁰ /β ⁰ Genotype β-Thalassemia

Abstract

Treatment with beti-cel resulted in a sustained HbA^T87Q level and a total hemoglobin level that was high enough to enable transfusion independence in most patients with a non-β⁰/β⁰ genotype, including those younger than 12 years of age. (Funded by Bluebird Bio; HGB-207 ClinicalTrials.gov number, NCT02906202.).