Gene therapy for serious genetic disorder might be approved after all

10 June 2022

In a surprise unanimous vote, the US Food and Drug Administration’s scientific advisors have endorsed a rare disease therapy from bluebird bio (Nasdaq: BLUE), despite a perceived cancer risk.

Elivaldogene autotemcel (eli-cel) now looks likely to be approved for the treatment of early active cerebral adrenoleukodystrophy (CALD) in children who do not have a suitable stem cell donor.

More than 70% of people diagnosed with CALD do not have a matched sibling donor or an alternative option.

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