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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.
Novartis and Precision BioSciences announce in vivo gene editing collaboration
The companies will work together to develop potentially curative treatments for disorders including sickle cell disease and beta thalassaemia. ... one-time transformative treatment option for diseases including certain haemoglobinopathies such as SCD and
Latest news
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Celebrating the power of knowledge on International Thalassaemia Day 2022
In order to be born with the main form of thalassaemia – beta thalassaemia – a child has to inherit a copy of the faulty beta thalassaemia gene from both parents. ... also a carrier – eg if both parents have the faulty gene that causes beta
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bluebird bio splits to create new oncology firm 2seventy bio
progress with our product pipeline, including filing the US biologics licensing application for Beti-cel for beta-thalassaemia,” he added. ... Beti-cel (betibeglogene autotemcel) is a gene therapy for adult, adolescent and paediatric patients with
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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.
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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 In relation to its beta-thalassaemia gene therapy Zynteglo, bluebird bio said this would be withdrawn from the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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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.
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