San Rocco Therapeutics
Product Pipeline > TNS9
The patient has inherited ß-thalassemia major through the genes. These genes have mistakes in them, so the body cannot make normal red blood cells. Stem cells are made in the bone marrow. They are the earliest form of blood cells.
This study is being done to see if the investigators can make the stem cells produce normal red blood cells and hemoglobin. The investigators do this by collecting the stem cells. The genes with mistakes are removed from the cells. These cells are then treated so they have the corrected gene for making normal hemoglobin. These treated cells are given back to the patient through an injection (shot) in the vein. This is also known as gene transfer. In order for the body to accept these cells, the patient will need to receive a low dose of a drug called busulfan. It is a drug that will prepare the body to receive the new stem cells.
This study will let the investigators know:
If it is safe to give the patient the treated stem cells
If the treated stem cells will go into the bone marrow without causing side effects.
Gene transfer has been successful in treating many blood disorders.
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.
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.
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