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Bluebird Bio

Bluebird bio's Lenti-D gene therapy shows promise in cerebral adrenoleukodystrophy

Bluebird bio has revealed updated data for its investigational Lenti-D gene therapy in the treatment of cerebral adrenoleukodystrophy (CALD), a rare genetic and rapidly progressive disease that carries the threat of severe loss of neurologic function and death, a the 13th European Pediatric Neurology Society (EPNS) Congress in Athens.

The Massachusetts-based biotech unveiled findings from a Phase 2/3 study involving 32 CALD patients who are 17 years old and younger with a median follow-up of 21.2 months.

EMA approves Bluebird Bio's gene therapy Zynteglo

The European Commission has granted a conditional marketing authorisation to bluebird bio’s gene therapy Zynteglo.

The Massachusetts-based firm’s one time treatment has been approved for patients 12 years and older with transfusion-dependent β-thalassaemia (TDT).

In order to survive, patients with TDT must maintain their haemoglobin levels through regular blood transfusions.  These transfusions carry the risk of progressive, multi-organ damage which is caused by iron overload.

CHMP recommendation for bluebird bio's gene therapy in β-thalassaemia

Massachusetts-based bluebird bio has taken a significant leap forward in its pursuit of regulatory authorisation for its first gene therapy, Zynteglo (autologous CD34+ cells encoding β-globin AT87Q gene), securing recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) ) in the treatment of transfusion-dependent β-thalassaemia (TDT).

Specifically, the recommendation referred to the treatment of the blood disorder in patients over 12 years of age who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC

Working Life: Nicola Redfern, UK General Manager, bluebird bio

Published on 28/02/19 at 12:26pm

How did you find your way into the pharma industry? Was it always an aspiration of yours?

My mum worked for the industry when I was a baby and my grandma and I used to drive around Devon and Cornwall with her whilst she gave lectures as a nurse on female health and sexual health. So, in some ways, you could argue it’s in my blood.

bluebird bio reveals new UK General Manager

Published on: 09/01/19

bluebird bio, a US-based gene therapy biotech with a focus on cancer and severe genetic conditions,  has named Nicola Redfern as the new General Manager of its UK business.

Medigene and bluebird bio expand immunotherapy partnership to $1.5bn

Medigene has announced that it has bolstered the existing partnership it has in place with US firm bluebird bio, originally signed in 2016, bringing the total potential value of the deal to $1.5 billion.

The expansion of the deal will mean that the total number of projects between the two firms will rise to six, meaning Medigene will receive an additional one-off payment of $8 million. These projects will focus on the identification of T-cell receptor-modified T cell (TCR-T) immunotherapies to fight tumours.

bluebird backs own CAR-T prospect, takes option to co-develop with Celgene

Biotech companies showing faith in their own prospect drug candidates is not an unusual occurrence, some do it to extremes. However, when it happens to be a CAR-T candidate and one that is targeting anti-B-cell maturation antigen (BCMA), a few more people sit up to take notice.

Bluebird Bio gene therapy posts encouraging results in treatment of rare brain disease

Bluebird bio (NASDAQ: BLUE) has announced positive interim data from a Phase II/III trial evaluating their Lenti-D gene therapy in paediatric patients with rare brain disorder, cerebral adrenoleukodystrophy (CALD).

In the ongoing STARBEAM study, 17 young boys were treated with Lenti-D and none of them developed the major functional disabilities which are typical of the disease. Furthermore the therapy helped to stabilise neurological function in all of the participants, with one exception.

Gene therapy advance hailed for thalassemia

A new gene therapy for one of the most frequently inherited genetic disorders has produced promising results.

The trial by biotech company Bluebird Bio showed that for the first time a patient with severe beta-thalassemia has been living without the need for transfusions over a sustained period of time.

The study involved a single patient who had been dependent on transfusions since early childhood, but has now been free of transfusions for 21 months thanks to the gene therapy.

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