Gene Therapy & Adrenoleukodystrophy (ALD)

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.

Latest Updates on Gene Therapy:

The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) unanimously endorsed bluebird bio’s gene therapy treatment for cerebral ALD on June 9, 2022.  The committee’s recommendation is based on the Biologics License Application (BLA) currently under priority review by the FDA with a PDUFA goal date set for September 16, 2022.  To learn more about the meeting and expected timeline for review, please see bluebird bio’s press release linked below.  

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