The betibeglogene autotemcel (beti-cel) gene therapy caused paediatric and adult beta thalassaemia patients to achieve transfusion independence lasting over two years.
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Data from nearly seven years of follow up suggests one-time treatment with eli-cel may durably stabilise cerebral adrenoleukodystrophy disease progression.
BMS and bluebird bio have applied to the FDA for approval of ide-cel as a standard therapy for relapsed or refractory multiple myeloma based on positive Phase II trial results.
bluebird bio has chosen to suspend sales of Zynteglo while they investigate whether safety concerns identified with a related investigational gene therapy may apply to the licenced medicine.