ex-vivo gene therapy
data set in the world.
our clinical trials
We are committed to diverse and inclusive access in our clinical trials because we believe the best way to deliver potentially life-altering therapies is to ensure clinical research accurately, and fully, represents all those it’s intended to help. We recognize we have work to do on this journey of equity and inclusion, and we intend to set the standard in improving diversity, as well as for gene therapy. We’re confident we can get there together. And we invite you to join the effort.
bluebird bio is conducting clinical studies of investigational gene therapies in three diseases: cerebral adrenoleukodystrophy, sickle cell disease, and β-thalassemia. Safety and efficacy have not been established.
For more information, follow the links below to the study listings on clinicaltrials.gov. Medical professionals may also email us at clinicaltrials@bluebirdbio.com for more information about study participation.
bluebird bio, Inc. gene therapies are investigational and not FDA-approved. Safety and efficacy have not been established.
Lenti-D
PHASE 3 STUDY
ALD-104
Evaluating the efficacy and safety of autologous CD34+ hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of CALD
STATUS: ACTIVE: NOT RECRUITING
PHASE 2/3 STUDY
Starbeam ALD-102
Evaluating safety and efficacy of Lenti-D gene therapy in patients with CALD
STATUS: COMPLETED
LONG-TERM FOLLOW-UP
LTF-304
Monitoring long-term safety and efficacy of Lenti-D gene therapy in patients with CALD treated in bluebird-sponsored clinical trials
STATUS: ENROLLING BY INVITATION
OBSERVATIONAL
ALD-103
Evaluating safety and efficacy of allogeneic hematopoietic stem cell transplantation in patients with CALD aged 17 or younger
STATUS: TERMINATED
Lovotibeglogene autotemcel (lovo-cel)
PHASE 1/2 STUDIES
HGB-206
Evaluating safety and efficacy of lovo-cel gene therapy in patients with SCD
STATUS: ACTIVE, NOT RECRUITING
HGB-205
Evaluating safety and efficacy of lovo-cel gene therapy in patients with TDT or SCD
STATUS: COMPLETE
PHASE 3 STUDY
HGB-210
Evaluating safety and efficacy of lovo-cel gene therapy in patients with SCD
STATUS: RECRUITING
LONG-TERM FOLLOW-UP
LTF-307
Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
STATUS: ENROLLING BY INVITATION
Betibeglogene autotemcel (beti-cel)
PHASE 3 STUDIES
Northstar-2 HGB-207
Evaluating safety and efficacy of beti-cel gene therapy in patients with TDT and non-β0/β0 genotypes
STATUS: ACTIVE, NOT RECRUITING
Northstar-3 HGB-212
Evaluating safety and efficacy of beti-cel gene therapy in patients with TDT and a β0/β0 and non-β0/β0 genotype
STATUS: ACTIVE, NOT RECRUITING
PHASE 1/2 STUDIES
Northstar HGB-204
Evaluating safety and efficacy of beti-cel gene therapy in patients with TDT
STATUS: COMPLETED
LONG-TERM FOLLOW-UP
LTF-303
Monitoring long-term safety and efficacy of beti-cel gene therapy in patients with TDT treated in bluebird-sponsored clinical trials
STATUS: ENROLLING BY INVITATION
* 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.