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Gene Therapy: Bluebird’s Eli-Cel Efficacy Uncertain, Malignancy Risk Concerning, US FDA Says

Executive Summary

Interpretation of efficacy from single-arm studies in patients with cerebral adrenoleukodsytrophy complicated by comparability with external controls and limited follow-up, FDA says in advisory committee briefing documents which also cite three cases of myelodysplastic syndrome and raise broader questions about lentiviral vector safety.

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