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Bluebird’s Gene Therapy for Brain Disease Receives Accelerated Approval

  • Company sets $3 million price and won’t offer any refunds
  • Safety concerns created uncertainty around gene therapy
Updated on

Bluebird Bio Inc.’s shares fell Monday after US regulators approved its gene therapy for a brain-wasting disease with a warning about cancer risks and under the condition that the drugmaker can show that it works long term.

Bluebird will charge $3 million for the treatment, Chief Executive Officer Andrew Obenshain said in an interview, the highest-ever US price for a drug. It’s a one-time treatment for children living with cerebral adrenoleukodystrophy and will be sold under the brand name Skysona.