newsFDA approves first cell-based gene therapies for sickle cell disease12 December 2023 | By Caroline Peachey (European Pharmaceutical Review)The US FDA has approved Vertex’s Casgevy and bluebird bio’s Lyfgenia, for one-time treatment of sickle cell disease in patients 12 years and older.
newsFDA approves Zynteglo gene therapy for beta-thalassemia patients18 August 2022 | By Hannah Balfour (European Pharmaceutical Review)bluebird bio’s Zynteglo (betibeglogene autotemcel), a one-time treatment for transfusion-dependent beta-thalassemia, will roll out with a $2.8mn price tag per dose.
newsβ-thalassemia patients achieve transfusion independence with gene therapy23 February 2022 | By Hannah Balfour (European Pharmaceutical Review)Over 90 percent of β-thalassemia patients treated with Betibeglogene autotemcel (beti-cel) achieved transfusion independence lasting over a year.
newsbeti-cel gene therapy demonstrates durable efficacy in beta thalassaemia patients16 June 2021 | By Hannah Balfour (European Pharmaceutical Review)The betibeglogene autotemcel (beti-cel) gene therapy caused paediatric and adult beta thalassaemia patients to achieve transfusion independence lasting over two years.
newsbluebird bio releases new data on its gene therapy for cerebral adrenoleukodystrophy16 March 2021 | By Hannah Balfour (European Pharmaceutical Review)Data from nearly seven years of follow up suggests one-time treatment with eli-cel may durably stabilise cerebral adrenoleukodystrophy disease progression.
newsIde-cel provides lasting remissions in multiply-relapsed myeloma25 February 2021 | By Hannah Balfour (European Pharmaceutical Review)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.
newsMarketing of Zynteglo suspended as precaution due to potential cancer risk19 February 2021 | By Hannah Balfour (European Pharmaceutical Review)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.
newsBluebird bio joins the ABPI as a full member14 September 2017 | By Dr Zara Kassam (European Pharmaceutical Review)The Association of the British Pharmaceutical Industry (ABPI) has admitted the American biotech bluebird bio, Inc. as a full member...