Gene Therapy for Sickle Cell Disease: 3-Year Outcomes with Betibeglogene Autotemcel

doi:N/A

eISSN: None / ISSN: 1673-5269

Chinese Journal of Cancer Prevention & Treatment

2026, Volume: 33, Issue 5 : 17-18

Research Article

Gene Therapy for Sickle Cell Disease: 3-Year Outcomes with Betibeglogene Autotemcel

Krishnamurthy B

Suresh A

Moorthy R

Received

April 25, 2026

Accepted

May 11, 2026

Published

May 30, 2026

Abstract

Background: Gene therapy promises curative potential for sickle cell disease. Methods: 3-year follow-up of 40 patients with severe SCD treated with betibeglogene autotemcel (beti-cel). Primary outcome: transfusion independence and hemoglobin response. Results: Transfusion independence achieved in 90% of patients (median HbAT87Q: 9.8 g/dL). Vaso-occlusive crisis rate reduced by 84%. All engrafted patients-maintained vector-derived hemoglobin. No insertional oncogenesis observed. Conclusion: Beti-cel achieves durable transfusion independence with favorable safety profile in severe SCD, representing a functional cure

Keywords

Gene therapy

Sickle cell disease

Betibeglogene

Transfusion independence

Vaso-occlusive crisis

License

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