Bluebird reveals positive gene therapy data – PharmaTimes

Bluebirdbio has announced updated results from a clinical development programmeevaluatingits investigational Lenti-D gene therapy inpatients with cerebraladrenoleukodystrophy (CALD).

The primary efficacy endpoint in the study isthe proportion of patients who are alive and free of major functionaldisabilities at month 24.Of those patients who have or would have reached 24months of follow-up and completed the study, 88% continue to be alive and majorfunctional disabilities-free in a long-term follow-up study.

Further data show that three out of the 32treated patients did not or will not meet the primary efficacy endpoint; twopatients withdrewfrom the study at investigator discretion, and oneexperienced rapid disease progression early on-study resulting in MFDs anddeath.

The company also announced that the 14 patientscurrently on-study have less than 24 months of follow-up and have shown noevidence of MFDs.

With the longest follow-up from the Phase II/IIIStarbeam study now up to five years, the data show that all boys with CALD whoweretreated with Lenti-D and were free of major functional disabilities at 24months continued to be major functional disabilities -free,explained DavidDavidson, chief medical officer of the company.

He continued, Importantly, there were noreports of graft failure or treatment-related mortality, and adverse eventswere generallyconsistent with myeloablative conditioning. These resultssupport the potential of Lenti-D as a treatment for CALD, which we hope maybecome an option for the boys and their families affected by this devastatingdisease.

CALD is the most severe manifestation ofadrenoleukodystrophy (ALD), a rare metabolic disorder. Approximately 35-40% ofboysdiagnosed with ALD will progress to CALD, typically between the ages ofthree and 12 years. It is characterised by a rapidly progressiveneurologicdecline leading to severe loss of neurologic function and death, in mostuntreated patients.

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Bluebird reveals positive gene therapy data - PharmaTimes