Bluebird Bio products
Lyfgenia®
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Generic: lovotibeglogene autotemcel (lovo-cel)
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Indication: gene therapy for sickle cell disease (patients ≥12 years with recurrent vaso-occlusive events)
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Usage: one-time autologous infusion of genetically modified hematopoietic stem cells following conditioning regimen; long-term follow-up required
Skysona®
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Generic: elivaldogene autotemcel (eli-cel)
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Indication: treatment of cerebral adrenoleukodystrophy (CALD) in pediatric patients
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Usage: single autologous stem cell infusion via IV after conditioning; includes long-term safety monitoring for potential risks
Zynteglo®
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Generic: betibeglogene autotemcel (beti-cel)
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Indication: transfusion-dependent β-thalassemia in patients aged ≥12 years without β0/β0 genotype
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Usage: one-time autologous IV infusion of modified stem cells following conditioning regimen; intended to produce lifelong transfusion independence
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