A Phase I Study of the Treatment of Beta-Thalassemia with a Gene Transfer Approach

Full Title

A Phase I Clinical Trial for the Treatment of ß-Thalassemia Major with Autologous CD34+ Hematopoietic Progenitor Cells Transduced with TNS9.3.55, a Lentiviral Vector Encoding the Normal Human ß-Globin Gene


Beta-thalassemia is an inherited disorder in which the body is unable to make normal hemoglobin and red blood cells. Hemoglobin is the protein in red blood cells that carries oxygen. Patients typically need repeated transfusions during their lifetimes, but this treatment is not a cure for the disease.

In this study, researchers are evaluating a new method for treating beta-thalassemia which relies on an approach called “gene transfer.” They will collect from the blood some of a patient’s own stem cells (blood-forming cells made in the bone marrow). The cells are then treated so they contain the corrected gene for making normal hemoglobin. The treated cells are returned to the patient through an intravenous infusion (by vein), and the patient also receives a drug called busulfan — before the infusion — to prepare the body to receive the new stem cells.

The goal of this phase I study is to assess the safety of this approach and to see if the treated stem cells with the new gene make their way back to the bone marrow without causing side effects. The hope is that this treatment can make a patient’s body produce normal hemoglobin and red blood cells.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must be age 18 or older.
  • Patients must have beta-thalassemia and have been receiving transfusions.
  • Patients may not have an HLA-matched sibling bone marrow donor.
  • Patients may not have active infections with hepatitis B or C, HIV, or HTLV.

For more information about this study and to inquire about eligibility, please contact Dr. Farid Boulad at 212-639-6684 or via e-mail.