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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.

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.”.

Latest news

  • 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.

  • GSK hands rare disease assets over to Orchard GSK hands rare disease assets over to Orchard

    Along with Strimvelis, GSK is passing over two therapies in late-stage clinical development for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS), and one clinical programme for beta thalassaemia.

  • Novo Nordisk pays $400m for EpiDestiny’s sickle cell drug Novo Nordisk pays $400m for EpiDestiny’s sickle cell drug

    Novo Nordisk has bolstered its biopharma unit with a global licensing deal for a small-molecule drug for genetic blood disorders sickle cell disease and beta thalassaemia. ... And as an oral, once-daily drug EpiDestiny’s candidate could be easier to

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