The Food and Drug Administration has granted Breakthrough Therapy designation to Lenti-D (bluebird bio) for the treatment of patients with cerebral adrenoleukodystrophy (CALD), also known as Lorenzo’s Oil disease. Currently, the only treatment for CALD is allogeneic hematopoietic stem cell transplant. 

This investigational lentiviral-based gene therapy is currently being studied in the Phase 2/3 Starbeam Study, a 2-year study assessing the safety and efficacy of Lenti-D in boys ≤17 years old with CALD. Findings from preliminary data showed that 88% (15/17) of patients infused with Lenti-D remained alive and free of major functional disabilities at 2-years post-treatment. The safety profile of Lenti-D was also found to be consistent with myeloablative chemotherapy.

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“The founding of bluebird was inspired by the potential to develop a one-time gene therapy for boys suffering from this potentially fatal form of adrenoleukodystrophy,” said David Davidson, MD, chief medical officer, bluebird bio. “With Lenti-D, we hope the modified, autologous hematopoietic stem cells will keep these boys alive and free from major functional disabilities while avoiding many of the safety risks of the current standard of care, allogeneic hematopoietic stem cell transplant.”

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