Gene Therapy Update
October 7, 2014 – Gene therapy and gene editing continue to hold promise as a potential curative approach for people with thalassemia. In recent years, genetic-based approaches to thalassemia have begun moving from animal studies into early stage clinical trials involving human subjects.
Memorial Sloan-Kettering
One of these trials, a Phase 1 study entitled “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. The trial received FDA approval in the summer of 2012 and is currently treating subjects.
Update from bluebird bio
bluebird bio is involved in two thalassemia-based projects about which it recently provided updates. Regarding the LG001 clinical study, bluebird reported information on two subjects treated in France as part of this study, one of which continues to be transfusion independent 72 months after transplantation. For the more recently begun HGB-205 study, information on two patients was presented. One subject recorded a hemoglobin of 10.1 post-transplant and a second patient recorded a hemoglobin of 11.6 subsequent to transplantation; subject one was transplanted 4.5 months prior to the report and subject 2 was transplanted two months prior to the report. Subject 1 last received a transfusion on day 10 post-transplant and subject 2 on day 12 post-transplant.
bluebird anticipates reporting information on another study, the Northstar study, later in 2014.
Sangamo gets RAC approval
On September 9, Sangamo BioSciences presented information to the Recombinant DNA Advisory Committee (RAC) on its proposed clinical trial, “A Phase I, Multicenter, Open-Label, Single-Dose Study to Assess the Safety and Tolerability of Autologous Hematopoietic Stem Progenitor Cells (HSPC) Modified at the BCL11A Gene by Zinc Finger Nucleases (SB-BCLmR-HSPC) to Increase Fetal Hemoglobin Production in Subjects with Transfusion-Dependent Beta-Thalassemia.”
This study is intended to use a gene editing technique to enable a person with thalassemia to increase the production of fetal hemoglobin, ideally to therapeutic levels which would decrease or eliminate the need for transfusions. Fetal hemoglobin production is high prior to birth but rapidly diminishes during the first year of life.
The RAC gave its approval of the protocol at the September 9 meeting. Sangamo subsequently confirmed that it is on track to file an IND (Investigational New Drug) application with the U.S. Food and Drug Administration before the end of the year.
The Cooley’s Anemia Foundation is encouraged at the activity in the gene-based treatment field and will continue to report on results as they become available.