Updated results from a Phase 2/3 clinical trial, Starbeam, evaluating bluebird bio's (BLUE -1.6%) gene therapy Lenti-D in patients with a rare severe inherited disorder called cerebral adrenoleukodystrophy (CALD) showed a sustained treatment effect. The data were presented at the European Pediatric Neurology Society Congress in Athens.

Treated patients remained free of major functional disabilities (MFDs) for as long as five years (and counting) with no reports of graft failure or treatment-related deaths. No new safety signals have been observed.

The study is assessing the safety and efficacy of autologous CD34+ hematopoietic stem cells transduced with Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. In other words, stem cells are extracted from the patient, modified with Lenti-D, then infused back into the patient after myeloablative conditioning (bone marrow activity is intentionally decreased to reduce the risk of complications).

88% (n=15/17) of treated patients who reached or would have reached 24 months' follow-up and completed the study are still alive and MFD-free.

Development is ongoing.

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Bluebird gene therapy shows sustained benefit in CALD study - Seeking Alpha