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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.

CHMP backs Bayer’s tumour-agnostic drug Vitrakvi

CHMP backs Bayer’s tumour-agnostic drug Vitrakvi

The committee also recommended a new generic from Mylan of Novartis’ Exjade (deferasirox), which is used to treat chronic iron overload due to blood transfusions in patients with beta thalassaemia, ... transfusion-dependent thalassaemia syndromes and

Latest news

  • Sangamo surges on data for haemophilia A gene therapy Sangamo surges on data for haemophilia A gene therapy

    There was more encouraging news for some of Sangamo’s other programmes as well, including data from one patient treated with ST-400, an autologous cell therapy being developed for ... thalassaemia in collaboration with Sanofi.

  • Bluebird plays down report of EU gene therapy approval Bluebird plays down report of EU gene therapy approval

    They say Zynteglo “is now approved for use in all European countries covered by the EMA”  for the treatment of adolescents and adults with transfusion-dependent beta-thalassaemia (TDT) who do ... of transfusion-dependent beta-thalassaemia.”.

  • Orchard surpasses IPO expectations, raising $200m Orchard surpasses IPO expectations, raising $200m

    Orchard gained ex-vivo gene therapy Strimvelis, a treatment for children with adenosine deaminase severe combined immunodeficiency (ADA-SCID), from the deal, along with a clinical programme for beta thalassaemia, which

  • Celgene to launch five new products through to 2020 Celgene to launch five new products through to 2020

    myelofibrosis, thalassaemia therapy luspatercept, and CAR-T candidates lisocabtagene maraleucel (liso-cel) for lymphoma and bb2121 for multiple myeloma. ... read-outs from the MEDALIST trial of luspatercept in myelodysplastic syndrome (MDS) and the

  • Orchard plans IPO as gene therapy pipeline advances Orchard plans IPO as gene therapy pipeline advances

    beta thalassaemia – which has just bagged a Priority Medicines (PRIME) designation from the EMA. ... It has been tested so far in nine patients with transfusion-dependent beta-thalassemia, the most severe form of the disease.

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