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beta thalassaemia

This page shows the latest beta thalassaemia news and features for those working in and with pharma, biotech and healthcare.

Vertex gains FDA fast track designation for diabetes stem cell therapy

This includes CTX001 – an investigational gene therapy – which has already demonstrated promising early data in transfusion-dependant beta thalassaemia (TDT) and severe sickle cell disease (SCD) patients.

Latest news

bluebird bio says AML case "very unlikely" to be related to gene therapy LentiGlobin

the FDA that we believe these results support lifting the clinical holds on our beta-thalassemia and sickle cell disease programmes,” he added. ... bluebird bio said that, based on the available data, it has initiated discussions with regulators to
bluebird bio posts modest Q4 revenue growth as it continues investigation into troubled LentiGlobin

In addition to the clinical holds, bluebird has also temporarily paused marketing of the gene therapy in Europe, where it is currently marketed as Zynteglo for the treatment of beta thalassaemia. ... Zynteglo was initially approved in the EU in 2019,
NICE turns down bluebird bio’s gene therapy Zynteglo

The UK’s National Institute for Health and Care Excellence (NICE) has rejected bluebird bio’s gene therapy Zynteglo for patients with transfusion-dependent beta-thalassaemia (TDT) in draft guidance. ... The one-off gene therapy was approved by the
bluebird bio to separate oncology and rare diseases units

bluebird bio will continue to focus on delivering gene therapies in its core areas – beta-thalassaemia, cerebral adrenoleukodystrophy and sickle cell disease. ... This includes its beta-thalassaemia gene therapy betibeglogene autotemcel, which is
ASH 2020: Gilead eyes new Yescarta indication, Vertex/CRISPR make headway in sickle cell and more

bluebird bio’s beta thalassaemia gene therapy Zynteglo scores promising long-term data. ... bluebird bio also posted long-term data for its own beta thalassaemia gene therapy Zynteglo (betibeglogene autotemcel) at ASH 2020.

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Latest Intelligence

Case-study for thalassaemia: Dimitri Azzopardi

An in-depth look at beta thalassaemia for Rare Disease Day. Beta thalassaemia major is a serious, lifelong genetic blood disorder. ... At least 75, 000 children are born. with thalassaemia worldwide every year, of which 10, 000 have the debilitating form
20 for 2020 – Five pharma companies to watch

treatment ozanimod and myelodysplastic syndromes/beta thalassaemia candidate luspatercept – to deliver on their potential.

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