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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.

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

Latest news

  • 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

  • Third time lucky? Sanofi cuts an $11.6bn deal to buy Bioverativ Third time lucky? Sanofi cuts an $11.6bn deal to buy Bioverativ

    Buying Bioverativ does give it a stake in advanced approaches for haematological diseases however, as the biotech has an established collaboration with Sangamo Therapeutics looking at genome-editing approaches to beta ... thalassaemia and sickle cell

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