BLUEBIRD'S GENE THERAPY SHOWED PROMISE FOR LORENZO'S OIL DISEASE

Oct 6, 2017

Published in New England Journal of Medicine, a Phase II/III clinical study showed promising data for Bluebird Bio's gene therapy.

At the time of the interim analysis, 15 of the 17 patients (88%) were alive and free of major functional disability from the devastating disease of cerebral adrenoleukodystrophy (CALD), also known as Lorenzo's Oil Disease.

The early results suggest Lenti-D, Bluebird Bio's gene therapy might be a safe and effective alternative to current treatment of stem-cell transplantation. Additional follow-up is needed to fully assess the duration of response and long-term safety of Lenti-D.

Cerebral adrenoleukodystrophy (CALD) is a potentially fatal form of adrenoleukodystrophy (ALD), an X-linked genetic disorder. ALD affects just one in 21,000 male births worldwide, and destroys the protective myelin sheath around the neurons in the brain. It leads to deafness, blindness, seizures, loss of muscle control and dementia, resulting in permanent disability or death. The only current effective treatment is allogeneic hematopoietic stem cell transplant (HSCT).

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