Gene Therapy

bluebird bio, a company located in Boston, MA  has conducted clinical trials for ALD gene therapy.  Results have been published (see below) and gene therapy treatment is promising to be an option for treatment for cerebral ALD. Gene therapy for ALD has yet to go through FDA approval.  If approved, it will be a treatment option for children with cerebral ALD that offers an alternative to allogeneic hematopoietic stem cell transplant, eliminating the need for a bone marrow match.



For more information:

bluebird bio: [email protected]

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

bluebird bio Announces FDA Priority Review of Biologics License Application for eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) in Patients Without a Matched Sibling Donor

bluebird Provides Update on FDA Review Timelines for Betibeglogene Autotemcel (beti-cel) for Beta-Thalassemia and Elivaldogene Autotemcel (eli-cel) for Cerebral Adrenoleukodystrophy (CALD)