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Bluebird bio's Lenti-D gene therapy shows promise in cerebral adrenoleukodystrophy

Published on 23/09/19 at 10:41am

Bluebird bio has revealed updated data for its investigational Lenti-D gene therapy in the treatment of cerebral adrenoleukodystrophy (CALD), a rare genetic and rapidly progressive disease that carries the threat of severe loss of neurologic function and death, a the 13th European Pediatric Neurology Society (EPNS) Congress in Athens.

The Massachusetts-based biotech unveiled findings from a Phase 2/3 study involving 32 CALD patients who are 17 years old and younger with a median follow-up of 21.2 months. Of this total, 15 have completed the study and moved on to a longer-term follow up study, while 14 patients are still taking part in the first stage. Three patients are no longer a part of the study.

It was revealed that, of the patients who have completed the study with 24 months of follow-up, 88% were alive and free of major functional disabilities (MFDs). These disabilities associated with CALD include wheelchair dependence, inability to communicate, incontinence, cortical blindness, the need to be fed via tube, and loss of voluntary movement.

Of those patients who are yet to finish the trial, none have shown evidence of MFDs.

Of the three patients who are no longer taking part in the study, two withdrew on the discretion of the investigator, while one suffered rapid disease progression, experiencing MFDs and ultimately death.

“With the longest follow-up from the Phase 2/3 Starbeam study now up to five years, the data show that all boys with CALD who were treated with Lenti-D and were free of MFDs at 24 months continued to be MFD-free. Importantly, there were no reports of graft failure or treatment-related mortality, and adverse events were generally consistent with myeloablative conditioning,” explained Dr David Davidson, Chief Medical Officer at bluebird bio. “These results support the potential of Lenti-D as a treatment for CALD, which we hope may become an option for the boys and their families affected by this devastating disease.”

Matt Fellows


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