Bluebird’s gene therapy shows promise in keeping lethal Lorenzo’s Oil disease in check
After an average of 29 months following a one-time gene therapy infusion, a clear majority of boys suffering from lethal cases of cerebral adrenoleukodystrophy were alive and free of major complications from the disease — the primary endpoint in the Phase II/III study sponsored by bluebird bio $BLUE.
Publishing in the New England Journal of Medicine, investigators at Dana-Farber, Boston Children’s Hospital and Mass General recruited 17 patients with this disease, making it one of the largest gene therapy studies now underway. And they concluded that at this interim stage they have good evidence of disease stability for this very vulnerable group, with 88% still alive and complication free.
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