A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

Clinical Publications

• Case study of betibeglogene autotemcel gene therapy in an adult Greek patient with transfusion-dependent β-thalassaemia of a severe genotype
• Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial
• Gene therapy in transfusion-dependent non-β0/β0 genotype β-thalassemia: first real-world experience of beti-cel
• Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia
• Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study
• Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
• Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for β-thalassemia

Protocol

• Statistical Analysis Plan
• FDA Approved ZYNTEGLO
• Clinical Trial Protocol