Thalagen™:
Gene Therapy Treatment for Thalassemia
Thalagen™ is the brand name for EGT's gene therapy treatment for beta thalassemia, also referred to as Cooley's anemia. To date there is no curative therapy for thalassemia, a disease characterized by the cells of the bone marrow having an inability to produce normal hemoglobin. Currently, patients are treated with transfusion therapy, which aims to correct the anemia, suppress massive erythropoiesis and inhibit gastrointestinal absorption of iron. However, transfusion therapy worsens the iron overload, which over time is lethal if not treated.
Gene therapy is considered by most expert blood specialists as the most promising, long-term and cost-effective treatment of thalassemia. The objective of gene therapy is to insert the "normal" gene for hemoglobin into the DNA of the patient's bone marrow cells. The putative treatment procedure requires the collection of bone marrow hematopoietic stem cells from the thalassemic patient in the hospital followed by the treatment of these cells in the laboratory with the virus vector containing the gene for the production of normal hemoglobin. The treated bone marrow cells are then returned to the patient to begin the production of red blood cells with normal hemoglobin.
The proprietary EGT technology of Thalagen™ is anticipated to be:
- Erythroid-specific for elevated expression of the inserted human β-globin gene
- Long-term, producing sustained expression of the human β-globin gene
June 2007 - Thalagen™ RAC Approval
EGT has secured approval from the The Recombinant DNA Advisory Committee (RAC) to proceed with gene therapy clinical trials for Thalagen™. To view the video from the June 2007 RAC meeting - click here.
April 2007 - Thalagen™ RAC protocol submitted
The Recombinant DNA Advisory Committee (RAC) is a panel of up to 21 national experts in various fields of science, medicine, genetics, ethics, and patient perspectives that considers the current state of knowledge and technology regarding recombinant DNA research. An important RAC function is to review research proposals involving human gene transfer research, or "gene therapy" as it is often called. All human gene transfer trials must be submitted to the Office of Biotechnology Activities (OBA) for review by the RAC.
More information on Thalagen™ - click here.
