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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 to lead development of CRISPR Therapeutics-partnered gene therapy
According to the companies, seven patients with transfusion-dependent beta thalassaemia (TDT), including three who have either a severe or b0/b0 genotype, were transfusion independent at the last follow-up.
Latest news
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bluebird bio anticipates lift of clinical holds on LentiGlobin in mid-2021
210 clinical studies of LentiGlobin for SCD, as well as the holds on the HGB-207 and HGB-212 studies of beti-cel for beta-thalassaemia. ... In relation to its beta-thalassaemia gene therapy Zynteglo, bluebird bio said this would be withdrawn from the
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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.
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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
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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,
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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has rejected bluebird bio’s gene therapy Zynteglo for patients with transfusion-dependent beta-thalassaemia (TDT) in draft guidance.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 anticipates lift of clinical holds on LentiGlobin in mid-2021
Vertex gains FDA fast track designation for diabetes stem cell therapy
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Latest Intelligence
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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
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20 for 2020 – Five pharma companies to watch
treatment ozanimod and myelodysplastic syndromes/beta thalassaemia candidate luspatercept – to deliver on their potential.
Case-study for thalassaemia: Dimitri Azzopardi
20 for 2020 – Five pharma companies to watch
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