Economic Partnerships

Gene Therapy Therapeutics for Hemoglobinopathies

EGT has an economic partnership for proceeds generated by the TNS9.3.55 vector technologies. This technology provides for an autologous insertion of missing or errant globin genes into patients? homeopathic stem cells, in order to alleviate chronic hereditary blood disorders.

To date there is no curative therapy for thalassemia, also referred to as Cooley?s anemia, 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 TNS9.3.55 vector technologies are:

  • Erythroid-specific for elevated expression of the inserted human ?-globin gene
  • Long-term, producing sustained expression of the human ?-globin gene

EGT identified, licensed and developed the emergent TNS9.3 vector technologies invented by Michel Sadelain, MD, PhD for genetically targeting beta-Thalassemias and other hemoglobinopathies. EGT completed the pre-clinical milestones (RAC meeting), including a likely world-first multi-patient cGMP batch of the vector for human clinical trials (EGT press release).

Subsequently, EGT repositioned its collaboration with Memorial Sloan Kettering Cancer Center (MSKCC) from exclusive licensee to economic partner for the invention. This arrangement allows the invention to benefit from MSKCC’s clinical (example: NEJM article) and commercialization (example: Juno transaction) track records. The clinical trial is currently based at MSKCC in New York under principal investigator Farid Boulad, MD.

EGT is proud of its work and dedication in progressing the Sadelain/TNS9.3.55 Vector Technology for hereditary hemoglobinopathies.

About The Technology/Patent


December 2013

Presentation of abstract titled ?First US Phase I Clinical Trial Of Globin Gene Transfer For The Treatment Of Beta-Thalassemia Major? at the 2013 American Society of Hematology (ASH) Annual Meeting and Exposition. The abstract states that in the ongoing clinical trial, that as of November 2013 two patients had been safely and effectively treated in the Phase I/II Beta-Thalassemia trial and have reported positive outcomes to date. The full abstract is available here. Furthermore, the details of the open and actively recruiting trial are found here.


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


Visit for full clinical trial description


July 2012 – Thalagen?

Launch of Stem Cell Therapy Trial Offers Hope for Patients with Inherited Blood Disorder – Click here.

?-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ?-Globin Gene – Click here.


July, 2011

EGT Transfers cGMP Vector for Gene Therapy Trial


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.