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immunodeficiency

This page shows the latest immunodeficiency news and features for those working in and with pharma, biotech and healthcare.

Daily Brief: Orchard Therapeutics raises 150m, Astellas swoops for early-stage gene company, Amicus gets FDA Fabry approval

Daily Brief: Orchard Therapeutics raises 150m, Astellas swoops for early-stage gene company, Amicus gets FDA Fabry approval

The proceeds will be used to fuel the progress of Orchard’s three most advanced clinical programmes: OTL-101 for adenosine deaminase severe combined immunodeficiency (ADA-SCID), OTL-200 for metachromatic

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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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