Category: CALD

  • Bluebird claims EU nod for rare disease therapy Skysona

    Bluebird bio has its first approval for Skysona (formerly Lenti-D), its gene therapy for children with the rare disease cerebral adrenoleukodystrophy (CALD), after getting the nod from the EU regulatory authority. The EMA has cleared Skysona (elivaldogene autotemcel) for use in CALD patients aged under 18 who have an ABCD1 genetic mutation and no matched […]

  • CHMP backs bluebird bio’s gene therapy for rare disease ALD

    US biotech bluebird bio has had a challenging time in the last few months, so a recommendation for EU approval of its gene therapy for adrenoleukodystrophy (ALD) will give it a lift. The EMA’s Committee for Medicinal Products for Human Use (CHMP) has given the go-ahead to bluebird’s therapy – known as Skysona (elivaldogene autotemcel) […]

  • bluebird bio announces long-term data from CALD gene therapy

    bluebird bio has announced new clinical trial data from its gene therapy for patients with cerebral adrenoleukodystrophy (CALD), including long-term follow-up data. CALD is the most severe form of adrenoleukodystrophy (ALD), a rare, X-linked metabolic disease affecting 1 in 21,000 newborn boys worldwide. Approximately 40% of boys with ALD will develop CALD1, typically between the […]