bluebird bio ‘baffled’ after NICE rejects beta thalassaemia gene therapy

Pharmaphorum

16 February 2021 - It’s back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance.

NICE is assessing Zynteglo (betibeglogene autotemcel), a one-off gene therapy for the condition, which can have life-threatening consequences and is associated with a curtailed life expectancy.

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Michael Wonder

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Michael Wonder