Reality bites: allocating gene therapy for hemoglobinopathies
- PMID: 40924431
- PMCID: PMC12447432
- DOI: 10.1182/bloodadvances.2025016943
Reality bites: allocating gene therapy for hemoglobinopathies
Conflict of interest statement
Comment on
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An ethical allocation scheme for scarce gene therapies in sickle cell disease and transfusion-dependent β-thalassemia.Blood Adv. 2025 Sep 9;9(17):4502-4512. doi: 10.1182/bloodadvances.2025016053. Blood Adv. 2025. PMID: 40273328 Free PMC article.
References
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- Kelkar AH, Achebe MO, Hantel A. An ethical allocation scheme for scarce gene therapies in sickle cell disease and transfusion-dependent beta-thalassemia. Blood Adv. 2025;9(17):4502–4512. - PubMed
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- Frangoul H, Locatelli F, Sharma A, et al. CLIMB SCD-121 Study Group Exagamglogene autotemcel for severe sickle cell disease. N Engl J Med. 2024;390(18):1649–1662. - PubMed
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- Locatelli F, Thompson AA, Kwiatkowski JL, et al. Betibeglogene autotemcel gene therapy for non-β0/β0 genotype β-thalassemia. N Engl J Med. 2022;386(5):415–427. - PubMed
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- Kwiatkowski JL, Walters MC, Hongeng S, et al. Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype beta-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial. Lancet. 2024;404(10468):2175–2186. - PubMed
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