We have the largest, deepest,

ex-vivo gene therapy
data set in the world.

pipeline

Product Candidates
Severe Genetic Diseases
  • Pre-Clinical
  • Phase 1
  • Phase 2
  • Phase 3
elivaldogene autotemcel gene therapy (eli-cel, Lenti-DTM)*
  • PH 2/3 
     
    Cerebral Adrenoleukodystrophy (Starbeam ALD-102)
  • PH 3 
    Cerebral Adrenoleukodystrophy (ALD-104)
betibeglogene autotemcel gene therapy (beti-cel; formerly LentiGlobinTM for β-thalassemia)
  • PH 3 
     
    TDT Non-β0/β0 genotypes (Northstar-2, HGB-207)
  • PH 3 
    TDT β0/β0 and non-β0/β0 genotypes (Northstar-3, HGB-212)
lovotibeglogene autotemcel gene therapy (lovo-cel; formerly LentiGlobinTM for sickle cell disease)†
  • PH 3 

    SCD (HGB-210)

  • PH 1/2 

    SCD (HGB-206)

BCL11A shRNA (mir)1
  • PH 1 

    Sickle Cell Disease

MULTIPLE UNDISCLOSED

  • PRE-C 

    Undisclosed

* Eli-cel clinical studies are currently on hold following a Suspected Unexpected Serious Adverse Reaction (SUSAR) of myelodysplastic syndrome (MDS) in a patient who was treated with eli-cel in the Phase 3 ALD-104 study. Consistent with this known risk, two additional cases of MDS have subsequently been reported and details have been shared with the FDA and study investigators. bluebird bio, Inc. continues to work with the FDA to resolve the hold.

† The lovo-cel clinical program is currently on a partial clinical hold pertaining to pediatric patients (under 18) related to an adolescent patient with persistent, non-transfusion-dependent anemia. bluebird bio, Inc. continues to work with the FDA to resolve the partial hold.

bluebird bio, Inc. gene therapies are investigational and not FDA-approved. Safety and efficacy have not been established.