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.<\/p>\n
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.<\/p>\n
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).<\/p>\n
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.<\/p>\n
Development is ongoing.<\/p>\n
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Bluebird gene therapy shows sustained benefit in CALD study - Seeking Alpha<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" 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. Continue reading