Gene Therapy

bluebird bio, a company located in Boston, MA  has conducted a clinical trial for ALD gene therapy. 17 boys with ALD have been treated with gene therapy with promising results. The first results have been published and the gene therapy treatment is very promising to be the next, safer method of treatment for ALD. Gene Therapy for ALD has yet to go through FDA approval. Gene Therapy would be the preferred method of treatment for ALD (once FDA approved) as there is no need to find a donor match in the bone marrow registry. Due to using the boys’ own cells there is no chance of rejection, as there may be with bone marrow transplantation.


For more information:

bluebird bio: [email protected]

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy (The New England Journal of Medicine)

bluebird bio receives EC approval for SKYSONA™ (elivaldogene autotemcel, Lenti-D™) gene therapy for patients less than 18 years of age with early cerebral adrenoleukodystrophy (CALD) without matched sibling donor