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Gene Therapy Therapeutics for Hemoglobinopathies

SRT 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

SRT identified, licensed and developed the emergent TNS9.3 vector technologies invented by Michel Sadelain, MD, PhD for genetically targeting beta-Thalassemias and other hemoglobinopathies. SRT 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, SRT 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: Juno transaction) track records. The clinical trial is currently based at MSKCC in New York under principal investigator Farid Boulad, MD.

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