Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia.
Franco LocatelliAlexis A ThompsonJanet L KwiatkowskiJohn B PorterAdrian J ThrasherSuradej HongengMartin G SauerIsabelle ThuretAshutosh LalMattia AlgeriJennifer SchneidermanTimothy S OlsonBen CarpenterPersis J AmroliaUsanarat AnurathapanAxel SchambachChristian ChabannonManfred SchmidtIvan LabikHeidi ElliotRuiting GuoMohammed AsmalRichard A ColvinMark C WaltersPublished in: The New England journal of medicine (2021)
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.).