Sangamo BioSciences, Inc. announced that the California Institute for Regenerative Medicine (CIRM) has granted the Company a $6.4 million Strategic Partnership Award to develop a potentially curative ZFP Therapeutic for beta-thalassemia based on the application of its zinc finger nuclease (ZFN) gene-editing technology in hematopoietic stem cells (HSCs).

The four-year grant provides matching funds for preclinical work that will support an Investigational New Drug application and a phase 1 clinical trial for a ZFP Therapeutic. The goal of the Therapeutic is to utilize genome-editing technology to raise levels of fetal hemoglobin in red blood cells to a sufficient degree to have a positive clinical effect.

More information: Sangamo Biosciences press release, CIRM funding

The primary aim of this study is to evaluate the effect of the drug N-Acetylcysteine on the frequency of pain in daily life in patients with Sickle Cell Disease (SCD).

Pain is an invalidating hallmark of this disease and has a considerable impact on the Quality of Life of patients and the medical health care system. Oxidative stress is hypothesized to play a central role in its pathophysiology. In pilot studies the administration of N-Acetylcysteine (NAC) resulted in a reduction of oxidative stress. Moreover, administration of NAC seemed to decrease hospitalization for painful crises in a small pilot study in patients with SCD.

This study will be performed as a multicenter, randomized, controlled trial where patients will be treated with either NAC or placebo for a period of 6 months. The investigators expect that NAC can reduce the frequency of pain in patients with SCD, thereby improving their quality of life and participation in society.

More information: clinicaltrials.gov, ITHANET Clinical Trials

Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia. Even when it is clear that alloBMT can give to these patients an improvement in their disease, myeloablative transplants have important toxicities and mortalities associated. The lack of suitable donors continues to be a limit to access to transplantation. Substantial progress has been made recently in the development of pre-treatment regimens that facilitate the sustained engraftment of donor marrow with reduced toxicity. Most of these regimens incorporate highly immunosuppressive drugs, which allow the reduction or elimination of myeloablative agents or total body irradiation without endangering the sustained engraftment of HLA-identical allogeneic stem cells. Preliminary results of non-myeloablative allogeneic stem cell transplantation suggest that the procedure can be performed in patients who are ineligible for myeloablative alloBMT, and that sustained remissions of several hematologic malignancies can be obtained.

More information: clinicaltrials.gov, ITHANET Clinical Trials

A new article was added in IthaPedia under the category tag "Educational resources". The article, entitled "Preimplantation Genetic Diagnosis: general principles and focus on haemoglobinopathies", was written by Jan Traeger-Synodinos, Christina Vrettou, Aspasia Destouni, Georgia Kakourou and Emmanuel Kanavakis.

The ITHANET Team would like to thank the authors for their valuable contribution.

More information: IthaPedia