Thalassemia Gene Therapy Study Begins Treating Participants
Summary
New York, NY, January 13, 2013 --( The Phase 1 study, the official title of which is “ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene,” is being conducted at Memorial Sloan-Kettering Cancer Center in Manhattan.“We provided scientific proof-of principle for this approach over a decade ago, and have since focused on resolving numerous clinical, biological and manufacturing challenges to open this trial under the best possible conditions.Sadelain, Boulad and Riviere are significant factors in the progress of the study, which is responding to a demonstrated need for a potential curative treatment for thalassemia.For more information, go to New York, NY, January 13, 2013 --( PR.com )-- The first U.S.-based clinical trial in gene therapy for beta-thalassemia has begun treating trial participants and the Cooley’s Anemia Foundation (CAF) congratulates the researchers involved in the project on the reaching this important stage.The Phase 1 study, the official title of which is “ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene,” is being conducted at Memorial Sloan-Kettering Cancer Center in Manhattan.Sadelain, Boulad and Riviere are significant factors in the progress of the study, which is responding to a demonstrated need for a potential curative treatment for thalassemia.