Gene Therapy Trial for ALD Halted

August 9, 2021 – bluebird bio has announced that a patient in their gene therapy trial for early cerebral adrenoleukodystrophy (ALD) has developed MDS (myelodysplastic syndrome).  In line with their protocols, bluebird has shared relevant data and sought advice about this report with the regulatory authorities, trial investigators, and the independent Data Monitoring Committee for the ALD-104 study. The U.S. Food and Drug Administration (FDA) has put the company’s clinical studies on ALD on hold in order to independently evaluate the benefit/risk.

Because the ALD trial uses a different vector than either the thalassemia or sickle cell trials, bluebird bio does not believe that this result will have any impact on either of these trials.  The company has stated that each of these gene therapies is custom-designed and has specific manufacturing requirements to address a specific severe genetic disease and therefore events related to one do not extrapolate to others.

CAF will keep the community informed of any other developments if they arise.

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