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

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Key points are not available for this paper at this time.

Abstract

Treatment with beti-cel resulted in a sustained HbAT87Q 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.).

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

Key Points

Abstract

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