Transfusion-Dependent β-Thalassemia - Healing Genes

Transfusion-Dependent β-Thalassemia

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A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

Phase 3


Doctors at research locations in the US and abroad seek adult patients with transfusion-dependent β-thalassemia, who do not have a β0/β0 genotype to trial a gene therapy of their own stem cells, which is performed outside the body in a lab and then the cells are returned to the patient with a functional human beta-globin gene. This hematopoetic stem cell transplant therapy uses a disabled virus to transfer a functional copy of the gene, and it requires that the patient receive leukapheresis to collect the stem cells, then chemotherapy to prepare their immune system before they are re-infused.

Follow up after the studies involved lab assessments and lasts 24 months to confirm the gene is producing  βA-T87Q-globin and that the patients require fewer transfusions.


  • Be up to 50 years of age
  • Diagnosis of TDT with a history of at least 100 mL/kg/year of packed red blood cells (pRBCs) in the 2 years preceding enrollment (all subjects), or be managed under standard thalassemia guidelines with ≥8 transfusions of pRBCs per year in the 2 years preceding enrollment (subjects ≥12 years)
  • Not have received hematopoetic stem cell transplant


  1. Screening before the treatment.
  2. Leukapheresis to collect blood stem cells.
  3. No treatment while the stem cells are gene-modified in the lab.
  4. Chemotherapy to prepare the immune system to receive the stem cells.
  5. Infusion of the gene-modified stem cells.
  6. Follow-up for a period of 24 months from the treatment.


Study site locations in both the US and abroad are listed here.

Contact:     bluebird bio  |  (339) 499-9300  |  [email protected]



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