Bluebird Bio ALD Gene Therapy Update

Update from Bluebird Bio regarding their ALD Gene Therapy Clinical Trial

In August 2021, Bluebird Bio announced that they planned to discontinue their operations in Europe. They announced that “they received a report of myelodysplastic syndrome (MDS) in a patient treated with elivaldogene autotemcel (eli-cel, or Lenti-D™), their investigational gene therapy for early cerebral adrenoleukodystrophy (cALD). The U.S. Food and Drug Administration (FDA) has put the clinical studies on ALD on hold in order to independently evaluate the benefit/risk of Lenti-D™”.


Bluebird Bio are “committed to the long-term follow-up of patients previously enrolled within the EU clinical trial programs as planned, but we will not initiate any new clinical trials in Europe for our therapies for betathalassemia, cerebral adrenoleukodystrophy or sickle cell disease. While we continue to investigate ways to make these therapies available to patients outside of the U.S., the path forward is unclear”.


Bluebird Bio have released an open letter on 21st October which states “Since our last update, despite our best efforts, we have been unable to identify a partner to continue the commercialization of our gene therapies in Europe. Therefore, as part of the winding down of operations, we made the difficult decision to withdraw the regulatory marketing authorization for SKYSONA™ (elivaldogene autotemcel) from the European Union, and our marketing application for SKYSONA from the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom (UK). We anticipate withdrawing marketing authorizations for ZYNTEGLO from both the EU and the UK by early 2022”.


Please follow the link to read the full open letter: bbbcom-community-update-letter.pdf (


We wanted to wait until they gave an update on whether another provider would be continuing their gene therapy programme in Europe before sharing with our community. We are saddened by the Bluebird Bio announcement, they have always been supportive of our work to improve outcomes for individuals with ALD.


If you have any questions, please contact the hospital where you were treated who will be able to advise you further. You can also contact Karen (Support Services Manager) on

Alex - The Leukodystrophy Charity