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immunodeficiency

This page shows the latest immunodeficiency 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

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  • Bluebird Bio: on the cusp of a gene therapy revolution Bluebird Bio: on the cusp of a gene therapy revolution

    pancreatitis. Then GSK’s Strimvelis followed in 2016 for a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency).

  • ‘How is your day?’ ‘How is your day?’

    These are: Primary Immunodeficiency Diseases (PID – causes severe infections); Chronic Inflammatory Demyelinating Polyneuropathy (CIDP – causes of loss of limb function/disability); bleeding disorders (haemophilia and related conditions);

  • Health on instalment Health on instalment

    years. Initial disease targets include cancer, neuromuscular disease, haemophilia, immunodeficiencies and blindness.

  • Europe vs the US: New drug product approvals Europe vs the US: New drug product approvals

    Two Advanced Therapy Medicinal Products (ATMP), Strimvelis (for the treatment of severe combined immunodeficiency) and Zalmoxis (for the treatment of certain types of high-risk haematological malignancies), were approved in the

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