Patients & Families

what is gene therapy?

The goal of gene therapy is to address a disease at the genetic level. Many different approaches are being studied, such as:

  • Turning off genes that are causing problems
  • Replacing a defective gene with a functional gene
  • Adding functional genes to help do the work of a defective gene

bluebird bio is studying an approach to gene therapy that adds functional copies of a faulty gene to a patient’s own blood stem cells. The functional copies of the gene are delivered into the patient’s blood stem cells outside of the patient’s body. This makes the gene therapy.

The gene therapy is then given to the patient via a bone marrow transplant (BMT), also called stem cell transplant or hematopoietic stem cell transplant.  This takes place in an in-patient hospital setting. As part of the transplant process, the patient receives myeloablative chemotherapy to make room in their bone marrow for the gene therapy. After the gene therapy has been infused, the patient’s cells will need time to multiply and produce enough new cells with the functional gene. This process is called engraftment. The patient remains in the hospital until their immune system cells have recovered and their doctor determines that it is safe for the patient to be discharged.

bluebird bio is currently investigating gene therapy in ongoing clinical trials in patients living with severe sickle cell disease, transfusion-dependent β-thalassemia (also known as β-thalassemia major) and cerebral adrenoleukodystrophy.

The following is a representation of organizations that offer resources and information on gene therapy, severe genetic diseases, cancer, and clinical studies: