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Bluebird bio's gene therapy candidate for rare X chromosome-linked disorder shows disease-stabilizing effects in Phase 2/3 study; investors unimpressed, shares down 5%

Investors appear to lack enthusiasm over interim data released today by bluebird bio (BLUE -4.8%) from a Phase 2/3 clinical trial assessing gene therapy candidate, Lenti-D, in pediatric patients with cerebral adrenoleukodystrophy (CALD), a rare inherited disorder linked to the X chromosome characterized by the buildup of fats in the brain. The results are being presented today at the 68th Annual Meeting of the American Academy of Neurology in Vancouver, BC.

The trial, called a>