bluebird reports additional Phase II/III data for Lenti-D gene therapy in childhood CALD

In October, bluebird bio Inc. (NASDAQ:BLUE) reported additional data from 17 boys with cerebral adrenoleukodystrophy (CALD) in the Phase II/III Starbeam (ALD-102) trial showing that Lenti-D led to no major functional disabilities at 24 months post-infusion -- the primary endpoint -- in 88% of patients. On secondary endpoints, Lenti-D led to a Neurological Function Score (NFS) of ≤1 point in 15 patients, and stabilization of the Loes score, defined as maintaining a score of ≤9 points or not increasing a score by ≥6 points, in 12 patients.

The therapy led to resolution of gadolinium positivity on MRI - another secondary endpoint -- in 16 patients by six months. Diffuse gadolinium enhancement re-emerged in six patients at month 12. At the last follow-up, 13 patients were negative for gadolinium enhancement...