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CALD gene therapy

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

EU approves bluebird bio’s CALD gene therapy Skysona

EU approves bluebird bio’s CALD gene therapy Skysona

The European Commission (EC) has approved bluebird bio’s gene therapy Skysona for the rare inherited neurological disease cerebral adrenoleukodystrophy (CALD). ... The EC has cleared Skysona (elivaldogene autotemcel) for the treatment of early CALD in

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  • Bluebird: on a mission to ‘recode’ the DNA of healthcare Bluebird: on a mission to ‘recode’ the DNA of healthcare

    Europe is the test ground for the gene therapy pioneer’s outcomes-based model, which could set the standard for the emerging sector. ... pipeline. This incudes Lenti-D, a gene therapy for patients with cerebral adrenoleukodystrophy (CALD), a rare,

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